A global clinical research data sharing platform
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Vivli

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A global clinical research data sharing platform

Vivli is a global clinical research data sharing platform that aggregates data from more than 8,000 clinical trials contributed by over 55 member organizations, covering approximately 5.2 million participants. The resource contains shareable clinical trial datasets and related research outputs that support secondary analysis, validation, and new scientific investigations, with more than 530 publications associated with its use. Its primary purpose is to enable researchers to access and reuse clinical trial data securely in order to advance medical knowledge, improve transparency, and generate findings that would be difficult or impossible without broad data sharing.

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127

Overall survival with maintenance olaparib in platinum-sensitive relapsed ovarian cancer by somatic or germline BRCA and homologous recombination repair mutation status

Sandro PignataAmit OzaGeoff HallBeatriz PardoRadoslaw Madry20
British Journal Of Cancer
2025
2025/3/17
00 p.1-8
The open-label, single-arm, multicentre ORZORA trial (NCT02476968) evaluated maintenance olaparib in patients with platinum-sensitive relapsed ovarian cancer (PSR OC) with a germline (g) or somatic (s) BRCA1 and/or BRCA2 mutation (BRCAm) or a non-BRCA homologous recombination repair mutation (non-BR...
Medical researchOncology
10.1038/S41416-025-02966-X
ISSN:0007-0920

Feasibility of a digital therapeutic for experiential negative symptoms of schizophrenia: results from an exploratory study

Haig GoenjianAbhishek PratapCassandra SnipesBrendan D. HareJoshua T. Kantrowitz16
Schizophrenia
2025
2025/9/26
Vol.11 No.1 p.1-13
Experiential negative symptoms (ENS) of schizophrenia, such as asociality, anhedonia, and avolition, are associated with poor outcomes, yet no FDA-approved pharmacotherapies currently exist specifically to target these symptoms. With the increasing use of smartphones, evidence-based digital interven...
Human behaviourSchizophrenia
10.1038/S41537-025-00659-1
ISSN:2754-6993

The DNA-PK inhibitor AZD7648 alone or combined with pegylated liposomal doxorubicin in patients with advanced cancer: results of a first-in-human Phase I/IIa study

Timothy A. YapPatricia LoRussoRowan E. MillerRebecca KristeleitAmanda G. Paulovich15
British Journal Of Cancer
2025
2025/5/17
00 p.1-10
Upregulation of DNA-dependent protein kinase (DNA-PK) is associated with poor prognosis and decreased response to DNA-damaging agents across cancer types. A Phase I/IIa study (NCT03907969) investigated the highly potent, selective DNA-PK inhibitor AZD7648 as monotherapy or combined with pegylated li...
Cancer
10.1038/S41416-025-03053-X
ISSN:0007-0920

Antibody-mediated LOX-1 inhibition in patients with residual inflammation after myocardial infarction: a randomized phase 2 trial

Michelle L. O’DonoghueDavid A. MorrowAndrea L. VavereDimitris KardassisFilipe A. Moura28
Nature Medicine
2025
2025/9/25
00 p.1-7
The lectin-like oxidized low-density lipoprotein (LDL) receptor-1 (LOX-1) is a key scavenger receptor for oxidized low-density lipoprotein cholesterol (oxLDL), which promotes inflammation and atherosclerosis. Here we evaluated MEDI6570, an antibody that acts as a LOX-1 antagonist, in a randomized, d...
Myocardial infarctionOutcomes research
10.1038/S41591-025-03951-W
ISSN:1078-8956

Immune correlates analysis of a phase 3 trial of the AZD1222 (ChAdOx1 nCoV-19) vaccine

Benkeser DavidFong YouyiJanes Holly E.Kelly Elizabeth J.Hirsch Ian40
Npj Vaccines
2023
2023/3/11
Vol.8 No.1 p.1-13
In the phase 3 trial of the AZD1222 (ChAdOx1 nCoV-19) vaccine conducted in the U.S., Chile, and Peru, anti-spike binding IgG concentration (spike IgG) and pseudovirus 50% neutralizing antibody titer (nAb ID50) measured four weeks after two doses were assessed as correlates of risk and protection aga...
Adaptive immunityInfectious diseasesVaccinesViral infection
10.1038/S41541-023-00630-0
ISSN:2059-0105

Quadrivalent meningococcal tetanus toxoid-conjugate booster vaccination in adolescents and adults: phase III randomized study

Zambrano BetzanaPeterson JamesDeseda CarmenJulien KatieSpiegel Craig A.18
Pediatric Research
2023
2023/3/10
00 p.1-9
The immunogenicity and safety of a booster dose of tetanus toxoid-conjugate quadrivalent meningococcal vaccine (MenACYW-TT), alone or co-administered with MenB vaccine, were assessed in healthy 13–25-year olds who received MenACYW-TT or a CRM-conjugate vaccine (MCV4-CRM) 3–6 years earlier. This phas...
Medicine/Public Health, generalPediatricsPediatric Surgery
10.1038/S41390-023-02478-5
ISSN:0031-3998

Triple hormone receptor agonist retatrutide for metabolic dysfunction-associated steatotic liver disease: a randomized phase 2a trial

Arun J. SanyalLee M. KaplanJuan P. FriasBram BrouwersQiwei Wu12
Nature Medicine
2024
2024/6/10
00 p.1-12
Retatrutide is a novel triple agonist of the glucose-dependent insulinotropic polypeptide, glucagon-like peptide 1 and glucagon receptors. A 48-week phase 2 obesity study demonstrated weight reductions of 22.8% and 24.2% with retatrutide 8 and 12 mg, respectively. The primary objective of this subst...
Non-alcoholic fatty liver diseaseNon-alcoholic steatohepatitisObesity
10.1038/S41591-024-03018-2
ISSN:1078-8956

AMEERA-1 phase 1/2 study of amcenestrant, SAR439859, in postmenopausal women with ER-positive/HER2-negative advanced breast cancer

Bardia AdityaChandarlapaty SaratLinden Hannah M.Ulaner Gary A.Gosselin Alice17
Nature Communications
2022
2022/7/15
Vol.13 No.1 p.1-15
AMEERA-1 is a Phase 1/2 open-label single-arm study evaluating once-daily (QD) amcenestrant, an orally bioavailable selective estrogen receptor (ER) degrader, in postmenopausal women with ER+/HER2− advanced breast cancer (NCT03284957), who were mostly heavily pretreated (including targeted therapies...
Breast cancerCancer genomicsCancer therapeutic resistance
10.1038/S41467-022-31668-8
ISSN:2041-1723

Efficacy of empagliflozin in heart failure with preserved versus mid-range ejection fraction: a pre-specified analysis of EMPEROR-Preserved

Anker Stefan D.Butler JavedUsman Muhammad ShariqFilippatos GerasimosFerreira João Pedro35
Nature Medicine
2022
2022/12/5
Vol.28 No.12 p.2512-2520
The EMPEROR-Preserved trial showed that the sodium–glucose co-transporter 2 inhibitor empagliflozin significantly reduces the risk of cardiovascular death or hospitalization for heart failure (HHF) in heart failure patients with left ventricular ejection fraction (LVEF) > 40%. Here, we report the...
Heart failureRisk factors
10.1038/S41591-022-02041-5
ISSN:1078-8956

Perioperative durvalumab plus chemotherapy plus new agents for resectable non-small-cell lung cancer: the platform phase II NeoCOAST-2 trial

Tina CasconeLaura BonannoFlorian GuisierAmelia InsaMoishe Liberman25
Nature Medicine
2025
2025/5/31
00 p.1-9
In the phase II NeoCOAST-2 platform study, 202 patients with untreated, resectable stage IIA–IIIB non-small-cell lung cancer (NSCLC) were randomized to receive neoadjuvant durvalumab plus platinum-doublet chemotherapy with oleclumab, a CD73 inhibitor (Arm 1), or with monalizumab, a NKG2A inhibitor (...
ImmunotherapyNon-small-cell lung cancer
10.1038/S41591-025-03746-Z
ISSN:1078-8956

Organ-metastatic landscape delineates overall and site-specific immune-related adverse events of PD-L1 blockade in advanced NSCLC

Si-Heng WangNing GaoYu-Ze WangJiamin HuangZhen Gan16
Npj Precision Oncology
2026
2026/3/18
0
It remains unknown whether metastases to different organs confer varying susceptibility to immune-related adverse events (irAEs) within a specific tumor type. We conducted a retrospective study included 708 advanced patients with non-small cell lung cancer (NSCLC) who received atezolizumab in two ra...
CancerOncology
10.1038/S41698-026-01375-4
ISSN:2397-768X

Functional outcomes of sustained improvement on Diabetic Retinopathy Severity Scale with intravitreal aflibercept in the VISTA and VIVID trials

Dhoot Dilsher S.Moini HadiReed KimberlyDu WeimingVitti Robert7
Eye
2022
2022/4/19
00 p.1-6
To assess time to, cumulative incidence of, and functional benefit of achieving sustained ≥2-step Diabetic Retinopathy Severity Scale (DRSS) improvement in diabetic macular oedema (DMO). Post hoc analysis of VISTA/VIVID including eyes with DMO treated with intravitreal aflibercept injections (IAI), ...
Eye abnormalitiesRetinal diseases
10.1038/S41433-022-02058-7
ISSN:0950-222X

Molecular correlates of clinical response and resistance to atezolizumab in combination with bevacizumab in advanced hepatocellular carcinoma

Zhu Andrew X.Abbas Alexander R.de Galarreta Marina RuizGuan YinghuiLu Shan20
Nature Medicine
2022
2022/6/23
00 p.1-13
Atezolizumab (anti-programmed death-ligand 1 (PD-L1)) and bevacizumab (anti-vascular endothelial growth factor (VEGF)) combination therapy has become the new standard of care in patients with unresectable hepatocellular carcinoma. However, potential predictive biomarkers and mechanisms of response a...
Liver cancerTumour biomarkers
10.1038/S41591-022-01868-2
ISSN:1078-8956

Immunogenicity and efficacy of an mRNA vaccine expressing a virus-like particle spike antigen against SARS-CoV-2

Jason P. LaliberteYingyun CaiTara KennyYi ZhangKuishu Ren29
Npj Vaccines
2025
2025/12/13
0
The COVID-19 pandemic spurred mRNA vaccine innovation, but new SARS-CoV-2 variants highlight the need for vaccines with improved potency and durability. This report presents a novel mRNA vaccine platform encoding virus-like particle antigens (mRNA-VLPs) that mimic native virus structures, aiming to ...
BiotechnologyImmunologyMicrobiology
10.1038/S41541-025-01303-W
ISSN:2059-0105

Tirzepatide cardiovascular event risk assessment: a pre-specified meta-analysis

Sattar NaveedMcGuire Darren K.Pavo ImreWeerakkody Govinda J.Nishiyama Hiroshi7
Nature Medicine
2022
2022/2/24
00 p.1-8
Tirzepatide is a novel, once weekly, dual GIP/GLP-1 receptor agonist and is under development for the treatment of type 2 diabetes (T2D) and obesity. Its association with cardiovascular outcomes requires evaluation. This pre-specified cardiovascular meta-analysis included all seven randomized contro...
Cardiovascular diseasesDiabetes complications
10.1038/S41591-022-01707-4
ISSN:1078-8956

Ucenprubart is an agonistic antibody to CD200R with the potential to treat inflammatory skin disease: preclinical development and a phase 1 clinical study

Anja KoesterDerrick R. WitcherMark LeeStephen J. DemarestScott Potter20
Nature Communications
2025
2025/5/1
Vol.16 No.1 p.1-15
CD200R is a checkpoint inhibitory receptor central to the pathogenesis of inflammatory skin disease. Here we describe the development and phase 1 clinical study (NCT03750643) of ucenprubart, a CD200R agonist antibody to downregulate immune system inflammation. Preclinical studies find ucenprubart in...
AntibodiesPsoriasisRandomized controlled trialsTranslational immunology
10.1038/S41467-025-59147-W
ISSN:2041-1723

Joint AI-driven event prediction and longitudinal modeling in newly diagnosed and relapsed multiple myeloma

Zeshan HussainEdward De BrouwerRebecca BoiarskySama SettyNeeraj Gupta11
Npj Digital Medicine
2024
2024/7/29
Vol.7 No.1 p.1-13
Multiple myeloma management requires a balance between maximizing survival, minimizing adverse events to therapy, and monitoring disease progression. While previous work has proposed data-driven models for individual tasks, these approaches fail to provide a holistic view of a patient’s disease stat...
CancerComputer scienceHealth care
10.1038/S41746-024-01189-3
ISSN:2398-6352

Fenebrutinib in H1 antihistamine-refractory chronic spontaneous urticaria: a randomized phase 2 trial

Metz MartinSussman GordonGagnon RémiStaubach PetraTanus Tonny16
Nature Medicine
2021
2021/11/8
00 p.1-9
Bruton’s tyrosine kinase (BTK) is crucial for FcεRI-mediated mast cell activation and essential for autoantibody production by B cells in chronic spontaneous urticaria (CSU). Fenebrutinib, an orally administered, potent, highly selective, reversible BTK inhibitor, may be effective in CSU. This doubl...
Immunological disordersMast cellsRandomized controlled trialsSkin manifestations
10.1038/S41591-021-01537-W
ISSN:1078-8956

Effectiveness of linvoseltamab versus real-world standard-of-care in triple-class-exposed relapsed/refractory multiple myeloma in the United States

Shaji KumarKatja C. WeiselPaul SpinNicolle BonarMuhaimen Siddiqui21
Blood Cancer Journal
2026
2026/4/1
0
LINKER-MM1 (NCT03761108) is a Phase 1/2 study of linvoseltamab, a human BCMA×CD3 bispecific antibody for patients with relapsed/refractory multiple myeloma (RRMM) who are triple-class exposed (TCE) with ≥3 prior lines of therapy (3L+), or triple-class refractory (TCR). The Phase 2 linvoseltamab 200 ...
Cancer immunotherapyHaematological cancerMyeloma
10.1038/S41408-026-01470-6
ISSN:2044-5385

Peri-operative atezolizumab in early-stage triple-negative breast cancer: final results and ctDNA analyses from the randomized phase 3 IMpassion031 trial

Elizabeth A. MittendorfZoe June AssafNadia HarbeckHong ZhangShigehira Saji21
Nature Medicine
2025
2025/6/4
00 p.1-8
Previously published results demonstrated that the randomized phase 3 IMpassion031 trial met its primary objective: adding atezolizumab to neoadjuvant chemotherapy significantly improved pathologic complete response (pCR) rate in patients with stage II/III triple-negative breast cancer (TNBC). Here ...
Breast cancerPrognostic markers
10.1038/S41591-025-03725-4
ISSN:1078-8956

TANGO: a placebo-controlled randomized phase 2 study of efficacy and safety of the anti-tau monoclonal antibody gosuranemab in early Alzheimer’s disease

Melanie ShulmanJessica KongJohn O’GormanElena RattiRajasimhan Rajagovindan14
Nature Aging
2023
2023/11/27
00 p.1-11
In Alzheimer’s disease, the spread of aberrantly phosphorylated tau is an important criterion in the Braak staging of disease severity and correlates with disease symptomatology. Here, we report the results of TANGO ( NCT03352557 ), a randomized, double-blind, placebo-controlled, parallel-group and ...
Alzheimer's disease
10.1038/S43587-023-00523-W
ISSN:2662-8465

Effects of ulotaront on brain circuits of reward, working memory, and emotion processing in healthy volunteers with high or low schizotypy

Francesca PeriniJadwiga Maria NazimekShane MckieLiliana P. CapitãoJessica Scaife16
Schizophrenia
2023
2023/8/7
Vol.9 No.1 p.1-11
Ulotaront, a trace amine-associated receptor 1 (TAAR1) and serotonin 5-HT1A receptor agonist without antagonist activity at dopamine D2 or the serotonin 5-HT2A receptors, has demonstrated efficacy in the treatment of schizophrenia. Here we report the phase 1 translational studies that profiled the e...
PharmacologySchizophrenia
10.1038/S41537-023-00385-6
ISSN:2754-6993

Modifying muscle metabolic dysregulation in inclusion body myositis with pioglitazone: a single-arm trial

Brittany L. AdlerMichael R. BeneCissy ZhangConrad SayPratik Khare18
Nature Communications
2026
2026/3/15
0
This single-arm, open-label phase 1 trial evaluated the PPARγ agonist pioglitazone in patients with inclusion body myositis (IBM). After a 16-week observation (lead-in) period, participants received pioglitazone 45 mg daily for 32 weeks. The primary outcome was the change in PPARGC1A expression and ...
Mechanisms of diseaseRheumatic diseasesTranslational research
10.1038/S41467-026-70262-0
ISSN:2041-1723

FcRH5×CD3 bispecific antibody cevostamab in relapsed or refractory multiple myeloma: a phase 1 trial

Adam D. CohenJoshua RichterSuzanne TrudelAlexander LesokhinJacob P. Laubach24
Nature Medicine
2026
2026/7/22
00 p.1-9
Fc receptor-homolog 5 (FcRH5) is a membrane protein that is ubiquitously expressed on myeloma cells. Cevostamab is a first-in-class, FcRH5×CD3 bispecific antibody. GO39775 is a phase 1 dose-escalation and dose-expansion study evaluating fixed-duration cevostamab in relapsed or refractory multiple my...
Drug developmentMolecularly targeted therapyMyeloma
10.1038/S41591-026-04522-3
ISSN:1078-8956

Effects of volenrelaxin in worsening heart failure with preserved ejection fraction: a phase 2 randomized trial

Barry A. BorlaugJeffrey M. TestaniMark C. PetrieZhenzhong WangJonathan Cunningham19
Nature Medicine
2025
2025/8/31
00 p.1-9
Relaxin is a peptide hormone that may decrease circulatory congestion and improve kidney function. In this study, we conducted a double-blind, international, multicenter trial to test whether volenrelaxin, a long-acting form of human relaxin, can improve left atrial (LA) function, reduce congestion ...
Medical researchOutcomes research
10.1038/S41591-025-03939-6
ISSN:1078-8956

Infographic: Study design for ELARA, an open-label, phase 3b trial of aflibercept 8 mg in previously treated patients with neovascular age-related macular degeneration or diabetic macular edema

David M. BrownAlyson J. BerlinerRohini Rao
Eye
2026
2026/6/5
00 p.1-2
Macular degenerationRetinal diseases
10.1038/S41433-026-04588-W
ISSN:0950-222X

ctDNA guiding adjuvant immunotherapy in urothelial carcinoma

Thomas PowlesZoe June AssafNicole DavarpanahRomain BanchereauBernadett E. Szabados27
Nature
2021
2021/6/16
00 p.1-6
Minimally invasive approaches to detect residual disease after surgery are needed to identify patients with cancer who are at risk for metastatic relapse. Circulating tumour DNA (ctDNA) holds promise as a biomarker for molecular residual disease and relapse1. We evaluated outcomes in 581 patients wh...
Bladder cancerPredictive markers
10.1038/S41586-021-03642-9
ISSN:0028-0836

Atezolizumab with enzalutamide versus enzalutamide alone in metastatic castration-resistant prostate cancer: a randomized phase 3 trial

Powles ThomasYuen Kobe C.Gillessen SilkeKadel Edward E.Rathkopf Dana20
Nature Medicine
2022
2022/1/10
00 p.1-10
Early clinical data indicate that some patients with castration-resistant prostate cancer may benefit from program death ligand-1 (PD-L1) inhibition, especially with enzalutamide. The IMbassador250 trial (no. NCT03016312) enrolled 759 men with metastatic castration-resistant prostate cancer whose di...
Prostate cancerTumour biomarkers
10.1038/S41591-021-01600-6
ISSN:1078-8956

Correlates of severe and delta COVID-19 in a phase 3 trial of the AZD1222 vaccine

Holly JanesYouyi FongYing HuangDavid BenkeserElizabeth J. Kelly31
Npj Vaccines
2026
2026/4/18
0
In the phase 3 AZD1222 COVID-19 vaccine trial, anti-Spike (vaccine-matched and Delta) binding IgG antibody concentration and neutralizing antibody (nAb) titer (vaccine-matched+D614G and Delta), measured four weeks post-dose two (D57), were assessed as correlates of risk of severe COVID-19 and Delta ...
DiseasesImmunologyMedical researchMicrobiology
10.1038/S41541-026-01411-1
ISSN:2059-0105

Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial

Maja Di RoccoEduardo Forleo-NetoRobert J. PignoloRichard KeenPhilippe Orcel36
Nature Medicine
2023
2023/9/28
00 p.1-10
Fibrodysplasia ossificans progressiva (FOP) is a rare disease characterized by heterotopic ossification (HO) in connective tissues and painful flare-ups. In the phase 2 LUMINA-1 trial, adult patients with FOP were randomized to garetosmab, an activin A-blocking antibody (n = 20) or placebo (n = 24) ...
Clinical trialsConnective tissue diseasesDrug developmentMusculoskeletal abnormalitiesMusculoskeletal system
10.1038/S41591-023-02561-8
ISSN:1078-8956

A longitudinal circulating tumor DNA-based model associated with survival in metastatic non-small-cell lung cancer

Assaf Zoe June F.Zou WeiFine Alexander D.Socinski Mark A.Young Amanda19
Nature Medicine
2023
2023/3/16
00 p.1-10
One of the great challenges in therapeutic oncology is determining who might achieve survival benefits from a particular therapy. Studies on longitudinal circulating tumor DNA (ctDNA) dynamics for the prediction of survival have generally been small or nonrandomized. We assessed ctDNA across 5 time ...
Machine learningNon-small-cell lung cancerClinical trial designTumour biomarkers
10.1038/S41591-023-02226-6
ISSN:1078-8956

Dupilumab in patients with prurigo nodularis: two randomized, double-blind, placebo-controlled phase 3 trials

Yosipovitch GilMollanazar NicholasStänder SonjaKwatra Shawn G.Kim Brian S.17
Nature Medicine
2023
2023/5/4
00 p.1-11
Prurigo nodularis (PN) is a chronic inflammatory skin disease with intensely pruritic nodules. The LIBERTY-PN PRIME and PRIME2 phase 3 trials enrolled adults with PN with ≥20 nodules and severe itch uncontrolled with topical therapies. Dupilumab, a fully human monoclonal antibody, blocks the shared ...
Antibody therapyDrug developmentInflammatory diseases
10.1038/S41591-023-02320-9
ISSN:1078-8956

Benralizumab versus placebo for hypereosinophilic syndrome: a randomized, placebo-controlled phase 3 trial

Princess U. OgboguFlorence RoufossePraveen AkuthotaPiotr KunaMatthieu Groh22
Nature Medicine
2026
2026/3/31
00 p.1-9
Benralizumab, an eosinophil-depleting anti-IL-5 receptor α antibody, has demonstrated efficacy in severe eosinophilic asthma and eosinophilic granulomatosis with polyangiitis and shown promising results in hypereosinophilic syndrome (HES). NATRON was a randomized, double-blind placebo-controlled pha...
ImmunologyMedical research
10.1038/S41591-026-04315-8
ISSN:1078-8956

Development and validation of a predictive model to guide the use of plerixafor in pediatric population

Sebastien BernardCheverton PeterMagnin CatherineAouni JihaneCastan Remi
Bone Marrow Transplantation
2022
2022/9/26
Vol.57 No.12 p.1827-1832
Plerixafor, a CXCR4 receptor antagonist, reduces the binding and chemotaxis of hematopoietic stem cells to the bone marrow stroma, resulting in predictable peak of cluster of differentiation 34+ (CD34+) cells in the peripheral blood (PB) approximately 10 h after its administration. We developed a mo...
Haematopoietic stem cellsStem-cell research
10.1038/S41409-022-01831-2
ISSN:0268-3369

Addressing challenges with real-world synthetic control arms to demonstrate the comparative effectiveness of Pralsetinib in non-small cell lung cancer

Popat SanjayLiu Stephen V.Scheuer NicolasHsu Grace G.Lockhart Alexandre8
Nature Communications
2022
2022/6/17
Vol.13 No.1 p.1-10
As advanced non-small cell lung cancer (aNSCLC) is being increasingly divided into rare oncogene-driven subsets, conducting randomised trials becomes challenging. Using real-world data (RWD) to construct control arms for single-arm trials provides an option for comparative data. However, non-randomi...
Lung cancerNon-small-cell lung cancerOutcomes research
10.1038/S41467-022-30908-1
ISSN:2041-1723

A hierarchical kidney outcome using win statistics in patients with heart failure from the DAPA-HF and DELIVER trials

Toru KondoPardeep S. JhundSamvel B. GasparyanMingming YangBrian L. Claggett11
Nature Medicine
2024
2024/5/6
00 p.1-8
Win statistics offer a new approach to the analysis of outcomes in clinical trials, allowing the combination of time-to-event and longitudinal measurements and taking into account the clinical importance of the components of composite outcomes, as well as their relative timing. We examined this appr...
Heart failureRandomized controlled trials
10.1038/S41591-024-02941-8
ISSN:1078-8956

Inflammation and mutational burden differentially associated with nivolumab or ipilimumab combination efficacy in colorectal cancer

Ming LeiMichael J. OvermanJin YaoThierry AndréSara Lonardi24
Nature Communications
2025
2025/10/6
Vol.16 No.1 p.1-12
Nivolumab alone and in combination with ipilimumab demonstrated durable clinical benefit in patients with previously treated microsatellite instability-high/mismatch repair-deficient metastatic colorectal cancer in the phase 2 CheckMate 142 study. Here, we report exploratory biomarker analyses from ...
Colorectal cancerPredictive markers
10.1038/S41467-025-63960-8
ISSN:2041-1723

Nivolumab plus chemotherapy or ipilimumab in gastroesophageal cancer: exploratory biomarker analyses of a randomized phase 3 trial

Kohei ShitaraYelena Y. JanjigianJaffer AjaniMarkus MoehlerJin Yao26
Nature Medicine
2025
2025/3/7
00 p.1-12
First-line nivolumab-plus-chemotherapy demonstrated superior overall survival (OS) and progression-free survival versus chemotherapy for advanced gastroesophageal adenocarcinoma with programmed death ligand 1 combined positive score ≥ 5, meeting both primary end points of the randomized phase 3 Chec...
Gastrointestinal diseasesPrognostic markers
10.1038/S41591-025-03575-0
ISSN:1078-8956

The obesity paradox in early and advanced HER2 positive breast cancer: pooled analysis of clinical trial data

Natansh D. ModiJin Quan Eugene TanAndrew RowlandBogda KoczwaraAhmad Y. Abuhelwa10
Npj Breast Cancer
2021
2021/3/22
Vol.7 No.1 p.1-6
While many studies have evaluated the relationship between BMI and breast cancer outcomes, it is unclear whether this relationship is consistent between early breast cancer (BC) and advanced BC. The study included 5099 patients with HER2 positive early BC (EBC) and 3496 with HER2 positive advanced B...
Breast cancerCancer epidemiology
10.1038/S41523-021-00241-9
ISSN:2374-4677

Isatuximab, carfilzomib, and dexamethasone in patients with relapsed multiple myeloma: updated results from IKEMA, a randomized Phase 3 study

Thomas MartinMeletios-Athanasios DimopoulosJoseph MikhaelKwee YongMarcelo Capra21
Blood Cancer Journal
2023
2023/5/9
Vol.13 No.1 p.1-9
Longer-term outcomes with the anti-CD38 antibody isatuximab in combination with carfilzomib-dexamethasone (Isa-Kd) were evaluated in the randomized Phase 3 trial IKEMA (NCT03275285), in a prespecified, follow-up analysis of progression-free survival (PFS, primary study endpoint), final complete resp...
Combination drug therapyDrug developmentMyeloma
10.1038/S41408-023-00797-8
ISSN:2044-5385

A machine learning approach to population pharmacokinetic modelling automation

Sam RichardsonItziar Irurzun AranaAndrzej NowojewskiDiansong ZhouJacob Leander8
Communications Medicine
2025
2025/7/31
Vol.5 No.1 p.1-10
Population pharmacokinetic (PopPK) models are crucial for understanding drug behaviour across populations, yet traditional development is often labour-intensive and slow. This study demonstrates an automated, out-of-the-box approach for popPK model development, leveraging optimisation algorithms imp...
Clinical pharmacologyPharmacokinetics
10.1038/S43856-025-01054-8
ISSN:2730-664X

Englumafusp alfa plus glofitamab in B cell non-Hodgkin lymphoma: a phase 1 trial

Martin HutchingsMichael DickinsonGiuseppe GrittiCarmelo Carlo-StellaWilliam Townsend25
Nature Medicine
2026
2026/7/16
00 p.1-9
There is an unmet need for effective, off-the-shelf therapies for relapsed or refractory aggressive B cell non-Hodgkin lymphoma (B-NHL). Part 2 of the current study was an open-label, nonrandomized, phase 1 study of escalating doses of the CD19–4-1BBL co-stimulatory molecule, englumafusp alfa, in co...
Drug developmentNon-hodgkin lymphoma
10.1038/S41591-026-04533-0
ISSN:1078-8956

HER2-related biomarkers predict clinical outcomes with trastuzumab deruxtecan treatment in patients with HER2-expressing metastatic colorectal cancer: biomarker analyses of DESTINY-CRC01

Salvatore SienaKanwal RaghavToshiki MasuishiKensei YamaguchiTomohiro Nishina19
Nature Communications
2024
2024/11/25
Vol.15 No.1 p.1-12
DESTINY-CRC01 (NCT03384940) was a multicentre, open-label, phase 2 study that investigated the safety and efficacy of trastuzumab deruxtecan (T-DXd) in patients with human epidermal growth factor receptor 2 (HER2)-expressing metastatic colorectal cancer (CRC). The present exploratory biomarker analy...
CancerPredictive markersTargeted therapies
10.1038/S41467-024-53223-3
ISSN:2041-1723

Validation of a liquid biopsy assay with molecular and clinical profiling of circulating tumor DNA

Justin D. FinkleHala BoulosTerri M. DriessenChristine LoRichard A. Blidner15
Npj Precision Oncology
2021
2021/7/2
Vol.5 No.1 p.1-12
Liquid biopsy is a valuable precision oncology tool that is increasingly used as a non-invasive approach to identify biomarkers, detect resistance mutations, monitor disease burden, and identify early recurrence. The Tempus xF liquid biopsy assay is a 105-gene, hybrid-capture, next-generation sequen...
Cancer genomics
10.1038/S41698-021-00202-2
ISSN:2397-768X

Bimagrumab plus semaglutide alone or in combination for the treatment of obesity: a randomized phase 2 trial

Steven B. HeymsfieldLouis J. AronnePenelope MontgomeryLloyd B. KlicksteinLaura A. Coleman10
Nature Medicine
2026
2026/3/2
Vol.32 No.3 p.869-882
Bimagrumab is an investigational antibody targeting type II activin receptors, intended to reduce total body and visceral fat mass and promote muscle growth. In this double-blind, placebo-controlled phase 2, trial, 507 adults with obesity (body mass index ≥30 kg m−2 or ≥27 kg m−2 with at least one o...
ObesityQuality of life
10.1038/S41591-026-04204-0
ISSN:1078-8956

Pertuzumab plus high-dose trastuzumab for HER2-positive breast cancer with brain metastases: PATRICIA final efficacy data

Nancy U. LinPriya KumthekarSolmaz SahebjamNuhad IbrahimAnita Fung9
Npj Breast Cancer
2023
2023/11/17
Vol.9 No.1 p.1-10
The PATRICIA study (NCT02536339) examined the efficacy and safety of pertuzumab plus high-dose trastuzumab in patients with HER2-positive metastatic breast cancer (MBC) with progressive central nervous system (CNS) metastases following radiotherapy. Primary analysis confirmed CNS objective response ...
Breast cancer
10.1038/S41523-023-00587-2
ISSN:2374-4677

Efficacy and safety of isatuximab plus bortezomib, lenalidomide, and dexamethasone in patients with newly diagnosed multiple myeloma ineligible/with no immediate intent for autologous stem cell transplantation

Enrique M. OcioAurore PerrotPierre BoriesJesus F. San-MiguelIgor W. Blau21
Leukemia
2023
2023/6/14
00 p.1-9
Patients with newly diagnosed multiple myeloma (NDMM) ineligible for autologous stem cell transplantation (ASCT) have lower survival rates and may benefit from frontline regimens that include novel agents. This Phase 1b study (NCT02513186) evaluated preliminary efficacy, safety, and pharmacokinetics...
Combination drug therapyMyeloma
10.1038/S41375-023-01936-7
ISSN:0887-6924

Molecular and phenotypic characteristics of RSV infections in infants during two nirsevimab randomized clinical trials

Bahar AhaniKevin M. TuffyAnastasia A. AksyukDeidre WilkinsMichael E. Abram21
Nature Communications
2023
2023/7/19
Vol.14 No.1 p.1-10
Nirsevimab is a monoclonal antibody that binds to the respiratory syncytial virus (RSV) fusion protein. During the Phase 2b (NCT02878330) and MELODY (NCT03979313) clinical trials, infants received one dose of nirsevimab or placebo before their first RSV season. In this pre-specified analysis, isolat...
AntibodiesBiological therapyPaediatric researchViral infection
10.1038/S41467-023-40057-8
ISSN:2041-1723

Over-the-counter short-acting β2-agonist purchase and asthma-related health outcomes: a post hoc analysis of the SABINA III study

David PriceMaarten J. H. I. BeekmanWalter Javier MattaruccoRocio Martina Barriga-AcevedoHao-Chien Wang11
Npj Primary Care Respiratory Medicine
2024
2024/11/1
Vol.34 No.1 p.1-10
This post-hoc analysis of the SABINA III study evaluated the association of short-acting β2-agonist (SABA) prescriptions and self-reported over-the-counter (OTC) SABA purchase in the previous 12 months with asthma-related outcomes using multivariable regression models in 4556 patients (mean age, 48....
AsthmaHealth policy
10.1038/S41533-024-00397-4
ISSN:2055-1010

Molecular residual disease analysis of adjuvant osimertinib in resected EGFR-mutated stage IB–IIIA non-small-cell lung cancer

Roy S. HerbstThomas JohnChristian GrohéJonathan W. GoldmanTerufumi Kato23
Nature Medicine
2025
2025/3/17
00 p.1-11
Osimertinib—a third-generation epidermal growth factor receptor-tyrosine kinase inhibitor—is recommended as adjuvant therapy for resected stage IB–IIIA epidermal growth factor receptor-mutated non-small-cell lung cancer, based on significant disease-free survival (DFS) and overall survival improveme...
Lung cancerNon-small-cell lung cancer
10.1038/S41591-025-03577-Y
ISSN:1078-8956

The renoprotective effect of esaxerenone independent of blood pressure lowering: a post hoc mediation analysis of the ESAX-DN trial

Okuda YasuyukiIto SadayoshiKashihara NaokiShikata KenichiNangaku Masaomi8
Hypertension Research
2022
2022/9/13
Vol.46 No.2 p.437-444
Angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs) are recommended as first-line drugs for hypertension with diabetic nephropathy owing to their renoprotective effect; however, their effect beyond lowering blood pressure (BP) has not been confirmed. Recent stud...
Medicine/Public Health, generalInternal MedicinePublic HealthGeriatrics/GerontologyObstetrics/Perinatology/MidwiferyHealth Promotion and Disease Prevention
10.1038/S41440-022-01008-W
ISSN:0916-9636

A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance

Tien DamLili YangColin GillisYumeng LiJohn O’Gorman9
Nature Communications
2025
2025/1/6
Vol.16 No.1 p.1-9
Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder characterized by physical, cognitive, and behavioral impairments. The PSP Rating Scale (PSPRS) is a widely used and validated, clinical scale to monitor disease progression. Here we show the modification of PSPRS to improve cl...
Movement disordersParkinson's disease
10.1038/S41467-024-55442-0
ISSN:2041-1723

Meningococcal quadrivalent ACYW-TT conjugated vaccine at 6–23 months: phase III study (US/Puerto Rico)

Cheryl DuffyOlga LyabisMandeep Singh DhingraBetzana ZambranoJulie Chaix9
Pediatric Research
2026
2026/3/6
00 p.1-11
A quadrivalent meningococcal tetanus toxoid-conjugate vaccine, MenACYW-TT, was developed to provide broad protection against invasive meningococcal disease across all age groups. We evaluated the immunogenicity and safety of MenACYW-TT as a two-dose series (1 + 1 schedule) co-administered with routi...
Medicine/Public Health, generalPediatricsPediatric Surgery
10.1038/S41390-026-04833-8
ISSN:0031-3998

Survodutide in adults with obesity and metabolic dysfunction-associated steatotic liver disease: SYNCHRONIZE-MASLD, a randomized, double-blind, placebo-controlled phase 3 trial

Lee M. KaplanElena StartsevaCarel W. le RouxSean WhartonBiykem Bozkurt14
Nature Medicine
2026
2026/6/7
00 p.1-11
Survodutide is a glucagon receptor/glucagon-like peptide-1 receptor dual agonist under investigation for treating obesity and related diseases. The SYNCHRONIZE-MASLD phase 3, randomized, double-blind, placebo-controlled trial included 216 adults (131 female and 85 male) with obesity (defined as a bo...
Diagnostic markersOutcomes research
10.1038/S41591-026-04479-3
ISSN:1078-8956

Agonist antibody to guanylate cyclase receptor NPR1 regulates vascular tone

Michael E. DunnAaron KithcartJee Hae KimAndre Jo-Hao HoMatthew C. Franklin43
Nature
2024
2024/9/11
Vol.633 No.8030 p.654-661
Heart failure is a leading cause of morbidity and mortality1,2. Elevated intracardiac pressures and myocyte stretch in heart failure trigger the release of counter-regulatory natriuretic peptides, which act through their receptor (NPR1) to affect vasodilation, diuresis and natriuresis, lowering veno...
Antibody therapyClinical pharmacology
10.1038/S41586-024-07903-1
ISSN:0028-0836

Prasinezumab slows motor progression in rapidly progressing early-stage Parkinson’s disease

Gennaro PaganoKirsten I. TaylorJudith Anzures CabreraTanya SimuniKenneth Marek18
Nature Medicine
2024
2024/4/15
Vol.30 No.4 p.1096-1103
Prasinezumab, a monoclonal antibody that binds aggregated α-synuclein, is being investigated as a potential disease-modifying therapy in early-stage Parkinson’s disease. Although in the PASADENA phase 2 study, the primary endpoint (Movement Disorder Society Unified Parkinson’s Disease Rating Scale (...
Drug developmentParkinson's disease
10.1038/S41591-024-02886-Y
ISSN:1078-8956

Examining protective effects of SARS-CoV-2 neutralizing antibodies after vaccination or monoclonal antibody administration

Dean FollmannMeagan P. O’BrienJonathan FintziMichael P. FayDavid Montefiori24
Nature Communications
2023
2023/6/17
Vol.14 No.1 p.1-9
While new vaccines for SARS-CoV-2 are authorized based on neutralizing antibody (nAb) titer against emerging variants of concern, an analogous pathway does not exist for preventative monoclonal antibodies. In this work, nAb titers were assessed as correlates of protection against COVID-19 in the cas...
AntibodiesRNA vaccinesSARS-CoV-2Viral infection
10.1038/S41467-023-39292-W
ISSN:2041-1723

Odronextamab monotherapy in patients with relapsed/refractory diffuse large B cell lymphoma: primary efficacy and safety analysis in phase 2 ELM-2 trial

Won Seog KimTae Min KimSeok-Goo ChoIsidro JarqueElżbieta Iskierka-Jażdżewska25
Nature Cancer
2025
2025/3/17
00 p.1-12
The phase 2, multicohort, ongoing ELM-2 study evaluates odronextamab, a CD20×CD3 bispecific antibody, in patients with relapsed/refractory (R/R) B cell non-Hodgkin lymphoma after ≥2 lines of therapy. Here primary analysis of the diffuse large B cell lymphoma (DLBCL) cohort is reported. Patients rece...
Antibody therapyCancerDrug discoveryClinical trial design
10.1038/S43018-025-00921-6
ISSN:2662-1347

Disease progression patterns and association of prostate-specific antigen level with risk of progression in nonmetastatic castration-resistant prostate cancer

Alicia K. MorgansChristopher J. D. WallisSusan HalabiAndrew J. ArmstrongPatrick Adorjan8
Prostate Cancer And Prostatic Diseases
2026
2026/2/12
00 p.1-7
In ARAMIS, darolutamide statistically significantly prolonged metastasis-free survival by 2 years and reduced the risk of death by 31% in patients with nonmetastatic castration-resistant prostate cancer (nmCRPC). We report post hoc analyses of ARAMIS evaluating patterns of disease progression overal...
Cancer therapyOutcomes researchProstate cancerProstatic diseases
10.1038/S41391-026-01076-W
ISSN:1365-7852

Pan-tumor activity of olomorasib, a next-generation KRAS G12C inhibitor in KRAS G12C-mutant advanced solid tumors: a first-in-human study

Yonina R. Murciano-GoroffAntoine HollebecqueRebecca S. HeistPhilippe A. CassierJi-Youn Han27
Nature Communications
2026
2026/3/12
0
This multicenter, first-in-human Phase 1 study (NCT04956640) evaluated olomorasib (LY3537982), a next-generation KRAS G12C inhibitor designed to enhance target occupancy at low absolute exposures. In total, data from 195 patients are reported: Phase 1a dose escalation (n = 112) assessed olomora...
Cancer therapyDrug delivery
10.1038/S41467-026-69943-7
ISSN:2041-1723

Secondary analysis of the EMPACT-MI trial reveals cardiovascular–kidney efficacy and safety of empagliflozin after acute myocardial infarction

Rahul AggarwalDeepak L. BhattAdrian F. HernandezStefan D. AnkerJosephine Harrington13
Nature Cardiovascular Research
2025
2025/6/13
00 p.1-14
Data on the cardiovascular–kidney effects and safety of empagliflozin among patients with acute myocardial infarction are limited. EMPACT-MI (Study to Evaluate the Effect of Empagliflozin on Hospitalization for Heart Failure and Mortality in Patients with Acute Myocardial Infarction) was a double-bl...
CardiologyMedical research
10.1038/S44161-025-00657-7
ISSN:2731-0590

Efficacy and safety of isatuximab subcutaneous plus carfilzomib and dexamethasone in patients with relapsed/refractory multiple myeloma: results of the Phase 2 study IZALCO

Gurdeep ParmarMarcelo CapraFernanda SeguroVania HungriaMeletios-Athanasios Dimopoulos23
Blood Cancer Journal
2025
2025/12/27
0
In the Phase 2 IZALCO study, we evaluated efficacy, patient preference, safety and pharmacokinetics for isatuximab administered SC by an innovative on-body injector (OBI) or manual injection, plus carfilzomib-dexamethasone (Kd) in relapsed/refractory multiple myeloma (RRMM) patients. In Part 1, isat...
Drug developmentMyeloma
10.1038/S41408-025-01436-0
ISSN:2044-5385

Tirzepatide after intensive lifestyle intervention in adults with overweight or obesity: the SURMOUNT-3 phase 3 trial

Thomas A. WaddenAriana M. ChaoSriram MachineniRobert KushnerJamy Ard12
Nature Medicine
2023
2023/10/15
00 p.1-10
The effects of tirzepatide, a glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist, on weight reduction after successful intensive lifestyle intervention are unknown. This double-blind, placebo-controlled trial randomized (1:1) adults with body mass index ≥30 or ...
Metabolic disordersObesity
10.1038/S41591-023-02597-W
ISSN:1078-8956

High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial

Richard S. FinkelThomas O. CrawfordEugenio MercuriCharlotte J. SumnerMaria del Mar Garcia Romero19
Nature Medicine
2026
2026/2/3
Vol.32 No.3 p.1095-1104
Despite the remarkable benefits of nusinersen and other disease-modifying therapies in spinal muscular atrophy (SMA), patients may still experience clinical manifestations of the disease. Here we assessed the potential for high-dose nusinersen to rapidly slow neurodegeneration and lead to improved o...
BiomarkersNeuromuscular disease
10.1038/S41591-025-04193-6
ISSN:1078-8956

DARolutamide ObservationaL (DAROL) study in patients with nonmetastatic castration-resistant prostate cancer: prespecified third interim analysis

Evan Y. YuHiroyoshi SuzukiChristopher M. PieczonkaGeoffrey GottoAlberto Briganti24
Prostate Cancer And Prostatic Diseases
2025
2025/11/1
00 p.1-4
DAROL is an ongoing study of real-world safety and effectiveness of darolutamide in patients with nonmetastatic castration-resistant prostate cancer (nmCRPC). This prespecified interim analysis included 550 patients with nmCRPC who completed ≥6 months of treatment with darolutamide 600 mg twice dail...
Cancer therapyOutcomes researchProstate cancerProstatic diseases
10.1038/S41391-025-01047-7
ISSN:1365-7852

Cemiplimab plus chemotherapy versus chemotherapy alone in non-small cell lung cancer: a randomized, controlled, double-blind phase 3 trial

Gogishvili MirandaMelkadze TamarMakharadze TamtaGiorgadze DavitDvorkin Mikhail26
Nature Medicine
2022
2022/8/25
Vol.28 No.11 p.2374-2380
First-line cemiplimab (anti-programmed cell death-1 (PD-1)) monotherapy has previously shown significant improvement in overall survival (OS) and progression-free survival (PFS) versus chemotherapy in patients with advanced non-small cell lung cancer (aNSCLC) and PD-ligand 1 (PD-L1) expression ≥50%....
Cancer immunotherapyNon-small-cell lung cancer
10.1038/S41591-022-01977-Y
ISSN:1078-8956

AI-guided patient stratification improves outcomes and efficiency in the AMARANTH Alzheimer’s Disease clinical trial

Delshad VaghariGayathri MohankumarKeith TanAndrew LoweCraig Shering7
Nature Communications
2025
2025/7/17
Vol.16 No.1 p.1-12
Alzheimer’s Disease (AD) drug discovery has been hampered by patient heterogeneity, and the lack of sensitive tools for precise stratification. Here, we demonstrate that our robust and interpretable AI-guided tool (predictive prognostic model, PPM) enhances precision in patient stratification, impro...
DementiaNeuroscience
10.1038/S41467-025-61355-3
ISSN:2041-1723

Time-to-event analysis mitigates the impact of symptomatic therapy on therapeutic benefit in Parkinson’s disease trials

Gennaro PaganoDylan TrundellTanya SimuniNicola PaveseKenneth Marek14
Npj Parkinson's Disease
2025
2025/7/1
Vol.11 No.1 p.1-8
The use of symptomatic medications represents a challenge for clinical trials of novel medicines designed to slow Parkinson’s disease progression. A time-to-event (TTE) approach using a defined motor progression milestone may mitigate the confounding effect of symptomatic therapy on the Movement Dis...
Parkinson's disease
10.1038/S41531-025-01041-9
ISSN:2373-8057

First-line zolbetuximab plus mFOLFOX6 and nivolumab in unresectable CLDN18.2-positive gastric or gastroesophageal junction adenocarcinoma: a phase 2 trial

Kohei ShitaraHirokazu ShojiNicola FazioSara LonardiKeun-Wook Lee19
Nature Medicine
2026
2026/3/16
00 p.1-9
There is an unmet need for effective and safe treatments for patients with metastatic gastric/gastroesophageal junction (mG/GEJ) adenocarcinoma. Targeting claudin 18 isoform 2 (CLDN18.2) and programmed death ligand 1 (PD-L1), represents a promising strategy. Zolbetuximab, a CLDN18.2-targeting antibo...
Gastric cancerTumour biomarkers
10.1038/S41591-026-04306-9
ISSN:1078-8956

Effects of tirzepatide on circulatory overload and end-organ damage in heart failure with preserved ejection fraction and obesity: a secondary analysis of the SUMMIT trial

Barry A. BorlaugMichael R. ZileChristopher M. KramerSeth J. BaumKarla Hurt10
Nature Medicine
2024
2024/11/17
00 p.1-8
Patients with obesity-related heart failure with preserved ejection fraction (HFpEF) display circulatory volume expansion and pressure overload contributing to cardiovascular–kidney end-organ damage. In the SUMMIT trial, patients with HFpEF and obesity were randomized to the long-acting glucose-depe...
Outcomes researchTranslational research
10.1038/S41591-024-03374-Z
ISSN:1078-8956

Exploratory analyses of clinical outcomes from the BIIB080 phase 1b study in mild Alzheimer’s disease

Melanie ShulmanShuang WuNick ZiogasAmanda EdwardsJessica Collins17
Nature Aging
2026
2026/2/11
Vol.6 No.2 p.445-453
This study conducted exploratory analyses of the effects of BIIB080, a MAPT (microtubule-associated protein tau)-targeting antisense oligonucleotide, in participants with mild Alzheimer’s disease. A multicenter, randomized, double-blind, phase 1b trial was conducted as a placebo-controlled, multiple...
AgeingAlzheimer's disease
10.1038/S43587-025-01031-9
ISSN:2662-8465

Effect of esaxerenone on nocturnal blood pressure and natriuretic peptide in different dipping phenotypes

Kario KazuomiIto SadayoshiItoh HiroshiRakugi HiromiOkuda Yasuyuki6
Hypertension Research
2021
2021/10/15
00 p.1-9
There are limited data on the nighttime blood pressure (BP)-lowering effect of esaxerenone and its effect on N-terminal pro b-type natriuretic peptide (NT-proBNP), a predictor of cardiovascular risk, according to different dipping patterns of nocturnal BP. This was a post hoc analysis of a multicent...
Medicine/Public Health, generalInternal MedicinePublic HealthGeriatrics/GerontologyObstetrics/Perinatology/MidwiferyHealth Promotion and Disease Prevention
10.1038/S41440-021-00756-5
ISSN:0916-9636

Efficacy and safety of darolutamide plus androgen-deprivation therapy in Black patients with metastatic hormone-sensitive prostate cancer from the phase 3 ARANOTE trial

Quoc-Dien TrinhDaniel J. GeorgeNeal ShoreEgils VjatersDavid Olmos12
Prostate Cancer And Prostatic Diseases
2026
2026/6/20
00 p.1-4
This analysis reports efficacy and safety data in the subgroup of Black patients from the ARANOTE phase 3 trial. Patients starting on androgen-deprivation therapy (ADT) were randomised 2:1 to receive darolutamide 600 mg twice daily or placebo. Of the total ARANOTE population (N = 669), 65 patients (...
Urological cancer
10.1038/S41391-026-01131-6
ISSN:1365-7852

Anticoagulation with osocimab in patients with kidney failure undergoing hemodialysis: a randomized phase 2 trial

Jeffrey I. WeitzLászló B. TankóJürgen FloegeKeith A. A. FoxDeepak L. Bhatt10
Nature Medicine
2024
2024/2/16
00 p.1-8
Individuals with kidney failure undergoing hemodialysis are at elevated risk for thromboembolic events. Factor (F) XI, which is in the intrinsic pathway of coagulation, is emerging as an attractive target for new anticoagulants that may be safer than existing agents. Osocimab—an inhibitory FXIa anti...
Drug developmentEnd-stage renal disease
10.1038/S41591-023-02794-7
ISSN:1078-8956

Tirzepatide versus insulin glargine as second-line or third-line therapy in type 2 diabetes in the Asia-Pacific region: the SURPASS-AP-Combo trial

Leili GaoByung Wan LeeManoj ChawlaJoshua KimLi Huo8
Nature Medicine
2023
2023/5/25
00 p.1-11
Tirzepatide is a once-weekly GIP/GLP-1 receptor agonist. In this phase 3, randomized, open-label trial, insulin-naive adults (≥18 years of age) with type 2 diabetes (T2D) uncontrolled on metformin (with or without a sulphonylurea) were randomized 1:1:1:1 to weekly tirzepatide 5 mg, 10 mg or 15 mg or...
DiseasesMedical researchType 2 diabetes
10.1038/S41591-023-02344-1
ISSN:1078-8956

Prior human endemic coronavirus exposure does not affect humoral responses to SARS-CoV-2 protein vaccines

Guy de BruynHaritha AdhikarlaCaroline K. BrackettYichen JiaPremkumar Lakshmanane12
Npj Vaccines
2025
2025/7/13
Vol.10 No.1 p.1-6
We included measurement of pre-existing immunity to human endemic coronaviruses (HCoV) in a Phase I/II study of SARS-CoV-2 spike protein vaccine candidates. A Binding Antibody Multiplex Assay measured HCoV-specific IgG to the receptor binding domain or full-length spike of human coronaviruses HKU1, ...
Drug developmentPredictive markersProtein vaccinesViral infection
10.1038/S41541-025-01203-Z
ISSN:2059-0105

Phase II trial of afatinib in patients with advanced urothelial carcinoma with genetic alterations in ERBB1-3 (LUX-Bladder 1)

Albert FontBegona MelladoMiguel A. ClimentJuan Antonio VirizuelaStephane Oudard21
British Journal Of Cancer
2023
2023/12/15
00 p.1-8
Preclinical and early clinical data suggest that the irreversible ErbB family blocker afatinib may be effective in urothelial cancers harbouring ERBB mutations. This open-label, phase II, single-arm trial (LUX-Bladder 1, NCT02780687) assessed the efficacy and safety of second-line afatinib 40 mg/d i...
Bladder cancerOncology
10.1038/S41416-023-02513-6
ISSN:0007-0920

Assessing the inter-rater reliability of the Schizophrenia Cognition Rating Scale: a non-interventional quantitative study

Sebastien TulliezStella KarantzoulisJames C. MarcusMontserrat CasamayorCassie Blanchard12
Schizophrenia
2025
2025/4/28
Vol.11 No.1 p.1-8
Background: Cognitive impairment is a core feature of schizophrenia, profoundly impacting patients’ functional abilities. As such, evaluating cognition-related functional activity/impairment is essential for identifying effective treatments. This study presents findings from a non-interventional qua...
PsychosisSchizophrenia
10.1038/S41537-025-00619-9
ISSN:2754-6993

Trastuzumab deruxtecan versus trastuzumab emtansine in HER2-positive metastatic breast cancer: long-term survival analysis of the DESTINY-Breast03 trial

Javier CortésSara A. HurvitzSeock-Ah ImHiroji IwataGiuseppe Curigliano20
Nature Medicine
2024
2024/6/2
00 p.1-8
Trastuzumab deruxtecan (T-DXd) demonstrated significantly improved efficacy over trastuzumab emtansine (T-DM1) in DESTINY-Breast03 (median follow-up, 28 months). We report updated efficacy and safety analyses, including secondary and exploratory efficacy endpoints (median follow-up, 41 months) of DE...
Breast cancerTargeted therapies
10.1038/S41591-024-03021-7
ISSN:1078-8956

Treatment monitoring by biomarker analysis in a Phase I dose-expansion study of AZD2811 for relapsed/refractory small-cell lung cancer

Melissa L. JohnsonGiulia FabbriCarmela CiardulloJudy S. WangGerald S. Falchook25
British Journal Of Cancer
2026
2026/4/3
00 p.1-13
Aurora kinase B (AURKB) is overexpressed in lung cancer and is associated with poor prognosis. AZD2811 is an AURKB inhibitor that demonstrated tolerability during a Phase I dose-escalation study in patients with advanced solid tumours, including small-cell lung cancer (SCLC). Here we report the dose...
Small-cell lung cancerTumour biomarkers
10.1038/S41416-026-03414-0
ISSN:0007-0920

Allocation and validation of the second revision of the International Staging System in the ICARIA-MM and IKEMA studies

Paul G. RichardsonAurore PerrotJoseph MikhaelThomas MartinMeral Beksac18
Blood Cancer Journal
2024
2024/11/28
Vol.14 No.1 p.1-11
The International Staging System for multiple myeloma recently underwent a second revision (R2-ISS) to include gain/amplification of 1q21 and account for the additive prognostic significance of multiple high-risk features. The phase 3 ICARIA-MM (isatuximab–pomalidomide–dexamethasone vs. pomalidomide...
Cancer immunotherapyTherapeutics
10.1038/S41408-024-01149-W
ISSN:2044-5385

Effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy: secondary outcomes of the HELIOS-B trial

Karola S. JeringMarianna FontanaOlivier LairezSimone LonghiOlga Azevedo13
Nature Medicine
2025
2025/8/6
00 p.1-9
In the HELIOS-B randomized clinical trial, the RNA interference therapeutic agent vutrisiran reduced the risk of all-cause mortality and recurrent cardiovascular events among patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). In this secondary analysis of HELIOS-B, we evaluated v...
Heart failureMedical researchOutcomes research
10.1038/S41591-025-03851-Z
ISSN:1078-8956

Data-driven prioritization of high-risk individuals for weight loss interventions

Kamil DemircanJulia Carrasco-ZaniniAlice WilliamsonCarl BeuchelLinsey Jackson15
Nature Medicine
2026
2026/4/30
00 p.1-11
New obesity medications have demonstrated efficacy in trials, but their real-world deployment is partly limited by the absence of approaches that identify individuals for treatment based on risks for obesity-related complications. Here we present a risk prediction model to guide prioritization of hi...
Predictive markersPredictive medicinePrognostic markers
10.1038/S41591-026-04353-2
ISSN:1078-8956

LRRK2-targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial

Omar S. MabroukBen TichlerH. Moore ArnoldEva C. ThomaSara M. Alexanian36
Nature Medicine
2026
2026/3/24
00 p.1-11
LRRK2 (encoding leucine-rich repeat kinase 2) variants are the most common genetic cause of Parkinson’s disease (PD). Lowering LRRK2 levels and/or inhibiting LRRK2 activity may modify PD-associated neuropathology. BIIB094 (ION859), an antisense oligonucleotide, targets LRRK2 mRNA for degradation. RE...
Drug developmentParkinson's disease
10.1038/S41591-026-04262-4
ISSN:1078-8956

Trastuzumab deruxtecan in HER2-positive advanced gastric cancer: exploratory biomarker analysis of the randomized, phase 2 DESTINY-Gastric01 trial

Kohei ShitaraYung-Jue BangSatoru IwasaNaotoshi SugimotoMin-Hee Ryu21
Nature Medicine
2024
2024/5/14
00 p.1-10
Trastuzumab deruxtecan (T-DXd) showed statistically significant clinical improvement in patients with human epidermal growth factor receptor 2-positive (HER2+) gastric cancer in the DESTINY-Gastric01 trial. Exploratory results from DESTINY-Gastric01 suggested a potential benefit in patients with HER...
Gastric cancerPredictive markers
10.1038/S41591-024-02992-X
ISSN:1078-8956

Infographic: Efficacy and safety of high-dose intravitreal aflibercept 8 mg in patients with diabetic macular oedema: week 96 results from the phase 2/3 PHOTON trial

Diana V. DoSergio LealAlyson J. Berliner
Eye
2026
2026/8/7
00 p.1-2
Retinal diseasesVision disorders
10.1038/S41433-026-04597-9
ISSN:0950-222X

The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial

Jörg F. HippCarlos A. BacinoLynne M. BirdIna Bruenig-TraebertDaniel Chan21
Nature Medicine
2025
2025/7/11
00 p.1-10
Angelman syndrome (AS) is a severe genetic neurodevelopmental disorder with no disease-modifying treatments. AS is caused by deletion or mutation of the neuronally imprinted gene encoding the ubiquitin-protein ligase E3A (UBE3A). Rugonersen (RO7248824) is an antisense oligonucleotide that reinstates...
Development of the nervous systemDevelopmental disordersDrug developmentNeurodevelopmental disorders
10.1038/S41591-025-03784-7
ISSN:1078-8956

Tirzepatide on ingestive behavior in adults with overweight or obesity: a randomized 6-week phase 1 trial

Corby K. MartinOwen T. CarmichaelSusan CarnellRobert V. ConsidineDavid A. Kareken19
Nature Medicine
2025
2025/6/24
00 p.1-10
Tirzepatide induces weight reduction but the underlying mechanisms are unknown. This 6-week phase 1 study investigated early effects of tirzepatide on energy intake. Male and female adults without diabetes (n = 114) and a body mass index from 27 to 50 kg per m2 were randomized 1:1:1 to blinded once-...
Obesity
10.1038/S41591-025-03774-9
ISSN:1078-8956

Final results of DESTINY-CRC01 investigating trastuzumab deruxtecan in patients with HER2-expressing metastatic colorectal cancer

Takayuki YoshinoMaria Di BartolomeoKanwal RaghavToshiki MasuishiFotios Loupakis20
Nature Communications
2023
2023/6/7
Vol.14 No.1 p.1-13
DESTINY-CRC01 (NCT03384940) was a multicenter, open-label, phase 2 trial assessing the efficacy and safety of trastuzumab deruxtecan (T-DXd) in patients with HER2-expressing metastatic colorectal cancer (mCRC) that progressed after ≥2 prior regimens; results of the primary analysis are published. Pa...
Colorectal cancerMetastasisTargeted therapies
10.1038/S41467-023-38032-4
ISSN:2041-1723

A SARS-CoV-2 variant‑adjusted threshold of protection model for monoclonal antibody pre-exposure prophylaxis against COVID-19

Rhiannon EdgeSam MatthewsBahar AhaniAnastasia A. AksyukLindsay Clegg18
Nature Communications
2025
2025/10/14
Vol.16 No.1 p.1-14
Clinical development of monoclonal antibodies (mAbs) against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is challenging due to rapid changes in the variant landscape. This study identified a threshold model for neutralising antibody (nAb) titres associated with clinically relevant p...
AntibodiesTranslational researchViral infection
10.1038/S41467-025-63972-4
ISSN:2041-1723

Tirzepatide on obstructive sleep apnea-related cardiometabolic risk: secondary outcomes of the SURMOUNT-OSA randomized trial

Atul MalhotraRonald GrunsteinAli AzarbarzinScott SandsVirend K. Somers12
Nature Medicine
2026
2026/1/15
Vol.32 No.2 p.653-659
Obstructive sleep apnea (OSA) is associated with obesity and cardiovascular risk. The SURMOUNT-OSA master protocol comprised two, 52-week, randomized, double-blind, placebo-controlled phase 3 studies (study 1 and study 2) and demonstrated a significant reduction of a number of cardiometabolic risk m...
Cardiovascular diseasesMetabolic disordersPhase III trials
10.1038/S41591-025-04071-1
ISSN:1078-8956

Abemaciclib in combination with pembrolizumab for HR+, HER2− metastatic breast cancer: Phase 1b study

Rugo Hope S.Kabos PeterBeck J. ThadJerusalem GuyWildiers Hans12
Npj Breast Cancer
2022
2022/11/5
Vol.8 No.1 p.1-8
This nonrandomized, open-label, multi-cohort Phase 1b study (NCT02779751) investigated the safety and efficacy of abemaciclib plus pembrolizumab with/without anastrozole in patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2−) metastatic breast canc...
Breast cancerCancer immunotherapyTargeted therapies
10.1038/S41523-022-00482-2
ISSN:2374-4677

A Phase Ib/II study of IGF-neutralising antibody xentuzumab with enzalutamide in metastatic castration-resistant prostate cancer

Valentine M. MacaulaySimon LordSyed HussainJosé Pablo MarotoRobert Hugh Jones14
British Journal Of Cancer
2023
2023/8/3
00 p.1-9
This multicentre, open-label, Phase Ib/II trial evaluated the insulin-like growth factor (IGF) 1/2 neutralising antibody xentuzumab plus enzalutamide in metastatic castrate-resistant prostate cancer (mCRPC). The trial included Phase Ib escalation and expansion parts and a randomised Phase II part ve...
Drug discoveryProstate cancer
10.1038/S41416-023-02380-1
ISSN:0007-0920

COLUMBIA-1: a randomised study of durvalumab plus oleclumab in combination with chemotherapy and bevacizumab in metastatic microsatellite-stable colorectal cancer

Neil H. SegalJeanne TieScott KopetzMichel DucreuxEric Chen16
British Journal Of Cancer
2024
2024/7/25
00 p.1-9
To determine whether the addition of durvalumab (anti-PD-L1) and oleclumab (anti-CD73) to standard-of-care treatment (FOLFOX and bevacizumab) enhances the anti-tumour effect in patients with metastatic colorectal cancer (mCRC). COLUMBIA-1 (NCT04068610) was a Phase Ib (feasibility; Part 1)/Phase II (...
Cancer immunotherapyColon cancerTumour biomarkers
10.1038/S41416-024-02796-3
ISSN:0007-0920

Phase 2 multicenter maintenance study of golidocitinib, A JAK1 selective inhibitor, in patients with peripheral T cell lymphomas after first-line systemic therapy (JACKPOT26)

Juying WeiQingqing CaiLiling ZhangLiqun ZouZengjun Li24
Blood Cancer Journal
2026
2026/3/17
Vol.16 No.1 p.360
Patients with peripheral T cell lymphoma (PTCL) who achieved tumor response with first-line standard therapy were at high risk of disease relapse. We explored golidocitinib (150 mg once daily) as maintenance therapy for this group of patients (JACKPOT26, NCT06511869). This study included two cohorts...
Drug developmentMolecularly targeted therapyPhase II trialsT-cell lymphomaTranslational research
10.1038/S41408-026-01452-8
ISSN:2044-5385

Mosunetuzumab with polatuzumab vedotin in relapsed or refractory aggressive large B cell lymphoma: a phase 1b/2 trial

Lihua E. BuddeAdam J. OlszewskiSarit AssoulineIzidore S. LossosCatherine Diefenbach24
Nature Medicine
2023
2023/12/10
00 p.1-11
Relapsed/refractory aggressive large B cell lymphoma (LBCL) remains an area of unmet need. Here we report the primary analysis of a phase 1b/2 trial of outpatient mosunetuzumab (a CD20xCD3 T-cell-engaging bispecific antibody) plus polatuzumab vedotin (an anti-CD79B antibody–drug conjugate) in relaps...
B-cell lymphomaCancer immunotherapyImmunotherapyPhase II trials
10.1038/S41591-023-02726-5
ISSN:1078-8956

The SGLT2 inhibitor empagliflozin in patients hospitalized for acute heart failure: a multinational randomized trial

Voors Adriaan A.Angermann Christiane E.Teerlink John R.Collins Sean P.Kosiborod Mikhail30
Nature Medicine
2022
2022/2/28
00 p.1-7
The sodium–glucose cotransporter 2 inhibitor empagliflozin reduces the risk of cardiovascular death or heart failure hospitalization in patients with chronic heart failure, but whether empagliflozin also improves clinical outcomes when initiated in patients who are hospitalized for acute heart failu...
Drug therapyHeart failure
10.1038/S41591-021-01659-1
ISSN:1078-8956

First-line durvalumab in combination with trastuzumab deruxtecan in women with locally advanced unresectable or metastatic, hormone-receptor-negative, HER2-low breast cancer: multicenter, open-label, phase 1b/2 BEGONIA platform trial

Peter SchmidSeock-Ah ImZbigniew NoweckiPiotr J. WysockiJacek Jassem12
Nature Cancer
2026
2026/6/8
00 p.1-10
More effective therapies are required for advanced breast cancer. We report results from 58 women with locally advanced unresectable or metastatic hormone-receptor (HR)-negative, human epidermal growth factor receptor 2 (HER2)-low breast cancer enrolled in arm 6 of the multicenter, open-label phase ...
Breast cancerCancerDrug developmentTumour biomarkers
10.1038/S43018-026-01181-8
ISSN:2662-1347

Targeting the angiopoietin-like protein 3/8 complex with a monoclonal antibody in patients with mixed hyperlipidemia: a phase 1 trial

Daniel GaudetMalgorzata GonciarzXi ShenJennifer K. LeohrThomas P. Beyer13
Nature Medicine
2025
2025/7/10
00 p.1-8
The angiopoietin-like protein 3/8 complex (ANGPTL3/8) inhibits lipoprotein lipase (LPL) activity, primarily in oxidative tissues, and does so more potently than ANGPTL3, making ANPTL3/8 an attractive target for treating dyslipidemia. This study enrolled 48 adults (36 men, 12 women) with mixed hyperl...
Metabolic disordersOutcomes research
10.1038/S41591-025-03830-4
ISSN:1078-8956

Linvoseltamab versus real-world International Myeloma Working Group standard-of-care in triple-class exposed relapsed/refractory multiple myeloma

Shaji KumarSundar JagannathKatja C. WeiselLaura RosiñolXavier Leleu43
Blood Cancer Journal
2026
2026/3/25
Vol.16 No.1 p.440
LINKER-MM1 (NCT03761108) is a Phase 1/2 study of linvoseltamab, a human BCMA×CD3 bispecific antibody for patients with relapsed/refractory multiple myeloma (RRMM) who are triple-class exposed (TCE) with ≥ 3 prior lines of therapy (3L+), or triple-class refractory (TCR). To contextualize efficacy dat...
Cancer immunotherapyHaematological cancerMyeloma
10.1038/S41408-026-01466-2
ISSN:2044-5385

Predictive biomarkers for survival benefit with ramucirumab in urothelial cancer in the RANGE trial

van der Heijden Michiel S.Powles ThomasPetrylak Danielde Wit RonaldNecchi Andrea25
Nature Communications
2022
2022/4/6
Vol.13 No.1 p.1-15
The RANGE study (NCT02426125) evaluated ramucirumab (an anti-VEGFR2 monoclonal antibody) in patients with platinum-refractory advanced urothelial carcinoma (UC). Here, we use programmed cell death-ligand 1 (PD-L1) immunohistochemistry (IHC) and transcriptome analysis to evaluate the association of i...
Bladder cancerPredictive markersTumour biomarkers
10.1038/S41467-022-29441-Y
ISSN:2041-1723

Effectiveness, immunogenicity and safety of monovalent DS-5670a and bivalent DS-5670a/b SARS-CoV-2 vaccines: results from two randomized, active-comparator, non-inferiority trials

Shunsuke HamadaMiharu SudaKatsuyasu IshidaKei FurihataYoshitaka Sogawa12
Communications Medicine
2026
2026/3/31
0
Although the emergency phase of the COVID-19 pandemic is over, attention has remained focused towards ongoing vaccination programs and the protection of high risk populations. The DS-5670 lipid nanoparticle-mRNA vaccine platform contains SARS-CoV-2 spike protein receptor-binding domain (RBD)-derived...
Viral infection
10.1038/S43856-025-01215-9
ISSN:2730-664X

Neoadjuvant PD-1 and LAG-3-targeting bispecific antibody and other immune checkpoint inhibitor combinations in resectable melanoma: the randomized phase 1b/2 Morpheus-Melanoma trial

Georgina V. LongNitya NairDaniel MarbachRichard A. ScolyerSabine Wilson26
Nature Medicine
2025
2025/9/24
00 p.1-13
Patients with stage III melanoma are at high risk of relapse. The NADINA trial evaluating neoadjuvant nivolumab plus ipilimumab and the SWOG-1801 trial evaluating neoadjuvant pembrolizumab have demonstrated superior clinical outcomes with neoadjuvant versus adjuvant checkpoint inhibition. Morpheus-M...
Cancer immunotherapyMelanomaTranslational researchTumour biomarkers
10.1038/S41591-025-03967-2
ISSN:1078-8956

Building a gene editing lexicon: a model for rare and inherited disorders

Leonard A. ValentinoCedric HermansDonna CoffinWolfgang MiesbachMaria Elisa Mancuso10
Gene Therapy
2026
2026/3/13
00 p.1-6
As more advanced cell and gene therapies, including gene editing technologies, progress through drug development, there is increased emphasis on the importance of stakeholders, including people living with disease, caregivers, and healthcare professionals, to communicate using clear, accurate, and c...
DiseasesHealth sciences
10.1038/S41434-026-00596-3
ISSN:0969-7128

Wild-type KRAS activation drives evasion of interferon-mediated immunity and resistance to immunotherapy in hepatocellular carcinoma

Martina Mang Leng LeiCarmen Oi Ning LeungRainbow Wing Hei LeungXue Qian WuKatherine Po Sin Chung21
Nature Communications
2025
2025/11/11
Vol.16 No.1 p.99130
Increasing evidence indicates that activation of oncogenic pathways contributes to an unfavourable tumour immune microenvironment (TIME), ultimately resulting in resistance to immunotherapy. Here, we aim to identify a critical oncogenic pathway involved in an antigen-expressing c-MYC-lucOSOE/Tp53KO ...
Hepatocellular carcinomaImmunoediting
10.1038/S41467-025-64860-7
ISSN:2041-1723

Pretrained transformers applied to clinical studies improve predictions of treatment efficacy and associated biomarkers

Gustavo Arango-ArgotyElly KipkogeiRoss StewartGerald J. SunArijit Patra7
Nature Communications
2025
2025/3/1
Vol.16 No.1 p.1-18
Cancer treatment has made significant advancements in recent decades, however many patients still experience treatment failure or resistance. Attempts to identify determinants of response have been hampered by a lack of tools that simultaneously accommodate smaller datasets, sparse or missing measur...
BiomarkersCancerComputational modelsMachine learning
10.1038/S41467-025-57181-2
ISSN:2041-1723

AI-driven reclassification of multiple sclerosis progression

Habib GanjgahiDieter A. HäringPiet AardenGordon GrahamYang Sun22
Nature Medicine
2025
2025/8/20
00 p.1-11
Multiple sclerosis (MS) affects 2.9 million people. Traditional classification of MS into distinct subtypes poorly reflects its pathobiology and has limited value for prognosticating disease evolution and treatment response, thereby hampering drug discovery. Here we report a data-driven classificati...
Multiple sclerosis
10.1038/S41591-025-03901-6
ISSN:1078-8956

Durability of neutralizing RSV antibodies following nirsevimab administration and elicitation of the natural immune response to RSV infection in infants

Wilkins DeidreYuan YuanChang YueAksyuk Anastasia A.Núñez Beatriz Seoane11
Nature Medicine
2023
2023/4/24
00 p.1-8
Nirsevimab is an extended half-life monoclonal antibody specific for the prefusion conformation of the respiratory syncytial virus (RSV) F protein, which has been studied in preterm and full-term infants in the phase 2b and phase 3 MELODY trials. We analyzed serum samples collected from 2,143 infant...
ImmunizationVaccinesViral infection
10.1038/S41591-023-02316-5
ISSN:1078-8956

Trastuzumab deruxtecan in HER2-positive advanced breast cancer with or without brain metastases: a phase 3b/4 trial

Nadia HarbeckEva CiruelosGuy JerusalemVolkmar MüllerNaoki Niikura22
Nature Medicine
2024
2024/9/13
00 p.1-10
Trastuzumab deruxtecan (T-DXd) intracranial activity has been observed in small or retrospective patient cohorts with human epidermal growth factor receptor 2–positive (HER2+) advanced/metastatic breast cancer (mBC) and stable or active (untreated/previously treated and progressing) brain metastases...
Breast cancer
10.1038/S41591-024-03261-7
ISSN:1078-8956

Analysis of acquired resistance mechanisms to osimertinib in patients with EGFR-mutated advanced non-small cell lung cancer from the AURA3 trial

Chmielecki JuliannMok TonyWu Yi-LongHan Ji-YounAhn Myung-Ju16
Nature Communications
2023
2023/2/27
Vol.14 No.1 p.1-8
Osimertinib, an epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI), potently and selectively inhibits EGFR-TKI-sensitizing and EGFR T790M resistance mutations. This analysis evaluates acquired resistance mechanisms to second-line osimertinib (n = 78) in patients with EGFR T790M ad...
CancerLung cancerTargeted therapies
10.1038/S41467-023-35962-X
ISSN:2041-1723

Sustained effect of prasinezumab on Parkinson’s disease motor progression in the open-label extension of the PASADENA trial

Gennaro PaganoAnnabelle MonnetAdriana ReyesBenjamin RibbaHanno Svoboda20
Nature Medicine
2024
2024/10/8
00 p.1-7
The Phase II trial of Anti-alpha-Synuclein Antibody in Early Parkinson’s Disease (PASADENA) is an ongoing double-blind, placebo-controlled trial evaluating the safety and efficacy of prasinezumab in early-stage Parkinson’s disease (PD). During the double-blind period, prasinezumab-treated individual...
Motor controlParkinson's disease
10.1038/S41591-024-03270-6
ISSN:1078-8956

Trastuzumab deruxtecan in HER2-low metastatic breast cancer: long-term survival analysis of the randomized, phase 3 DESTINY-Breast04 trial

Shanu ModiWilliam JacotHiroji IwataYeon Hee ParkMaria Vidal Losada25
Nature Medicine
2025
2025/10/8
00 p.1-9
In DESTINY-Breast04 ( NCT03734029 ), trastuzumab deruxtecan (T-DXd) significantly improved overall survival (OS) and progression-free survival compared with treatment of physician’s choice of chemotherapy (TPC) for patients with human epidermal growth factor receptor 2-low (HER2-low) (immunohistoche...
Breast cancerTargeted therapies
10.1038/S41591-025-03981-4
ISSN:1078-8956

Hybrid-control arm construction using historical trial data for an early-phase, randomized controlled trial in metastatic colorectal cancer

Li ChenFerro AnaMhatre Shivani K.Lu DannyLawrance Marcus16
Communications Medicine
2022
2022/7/15
Vol.2 No.1 p.1-8
Treatment for metastatic colorectal cancer patients beyond the second line remains challenging, highlighting the need for early phase trials of combination therapies for patients who had disease progression during or following two prior lines of therapy. Leveraging hybrid control design in these tri...
Colorectal cancerOncologyPhase I trials
10.1038/S43856-022-00155-Y
ISSN:2730-664X

Systematic review of evidence on feasibility of administering intranasal live attenuated influenza vaccine at home

Ravi JhaveriNeil SkolnikAllyn Bandell
Npj Vaccines
2025
2025/11/23
0
In the United States, influenza vaccination coverage in adults and children remains below target, with disparities across racial and ethnic groups and setting urbanicity. Reducing barriers to influenza vaccination may improve coverage. Live attenuated influenza vaccine (LAIV) recently gained United ...
Drug regulationLive attenuated vaccines
10.1038/S41541-025-01324-5
ISSN:2059-0105

Primary and secondary caregiver burden of cognitive impairment associated with schizophrenia: a qualitative study based on caregiver interviews

Sébastien TulliezAlessandra GirardiMatthew RidleyKatja RudellChristoph U. Correll9
Schizophrenia
2025
2025/10/17
Vol.11 No.1 p.1270
Cognitive impairment associated with schizophrenia (CIAS) places a significant burden on patients and caregivers. This study explored the burden experienced by primary (informal) and secondary (formal) caregivers. A secondary qualitative analysis was performed on interviews from a Schizophrenia Cogn...
PsychosisSchizophrenia
10.1038/S41537-025-00675-1
ISSN:2754-6993

Candidate mechanisms of acquired resistance to first-line osimertinib in EGFR-mutated advanced non-small cell lung cancer

Chmielecki JuliannGray Jhanelle E.Cheng YingOhe YuichiroImamura Fumio15
Nature Communications
2023
2023/2/27
Vol.14 No.1 p.1-9
Osimertinib, an epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI), potently and selectively inhibits EGFR-TKI-sensitizing and EGFR T790M resistance mutations. In the Phase III FLAURA study (NCT02296125), first-line osimertinib improved outcomes vs comparator EGFR-TKIs in EGFRm ad...
CancerLung cancerTargeted therapies
10.1038/S41467-023-35961-Y
ISSN:2041-1723

ROBIN: a randomised, double-masked, placebo-controlled Phase IIa study of the AOC3 inhibitor BI 1467335 in diabetic retinopathy

Quan Dong NguyenJustis P. EhlersDavid S. BoyerXidong JinAndrea Giani6
Eye
2024
2024/5/28
00 p.1-9
To evaluate the safety and efficacy of BI 1467335 in patients with non-proliferative diabetic retinopathy (NPDR). ROBIN is a Phase IIa, double-masked, randomised, placebo-controlled study (NCT03238963). Patients with NPDR and without centre-involved diabetic macular oedema were included; all had a b...
Diabetes complicationsEye diseases
10.1038/S41433-024-03017-0
ISSN:0950-222X

Precision targeting of β-catenin induces tumor reprogramming and immunity in hepatocellular cancers

Brandon M. LehrichEvan R. DelgadoTyler M. YasakaSilvia LiuCatherine Cao35
Nature Communications
2025
2025/5/30
Vol.16 No.1 p.1-26
First-line immune checkpoint inhibitor (ICI) combinations show responses in subsets of hepatocellular carcinoma (HCC) patients. Nearly half of HCCs are Wnt-active with mutations in CTNNB1 (encoding for β-catenin), AXIN1/2, or APC, and demonstrate heterogeneous and limited benefit to ICI due to an im...
Cancer microenvironmentHepatocellular carcinomaTargeted therapies
10.1038/S41467-025-60457-2
ISSN:2041-1723

Longitudinal genomic profiling using liquid biopsies in metastatic nonsquamous NSCLC following first line immunotherapy

Haolun DingMin YuanYaning YangXu Steven Xu
Npj Precision Oncology
2025
2025/1/8
Vol.9 No.1 p.1-8
Tumor genomic profiling is often limited to one or two timepoints due to the invasiveness of tissue biopsies, but longitudinal profiling may provide deeper clinical insights. Using ctDNA data from IMpower150 study, we examined genetic changes in metastatic non-squamous NSCLC post-first-line immunoth...
Cancer genomicsNon-small-cell lung cancer
10.1038/S41698-024-00797-2
ISSN:2397-768X

Evinacumab in severe hypertriglyceridemia with or without lipoprotein lipase pathway mutations: a phase 2 randomized trial

Rosenson Robert S.Gaudet DanielBallantyne Christie M.Baum Seth J.Bergeron Jean17
Nature Medicine
2023
2023/3/6
Vol.29 No.3 p.729-737
Severe hypertriglyceridemia (sHTG) is an established risk factor for acute pancreatitis. Current therapeutic approaches for sHTG are often insufficient to reduce triglycerides and prevent acute pancreatitis. This phase 2 trial ( NCT03452228 ) evaluated evinacumab (angiopoietin-like 3 inhibitor) in t...
DyslipidaemiasFat metabolism
10.1038/S41591-023-02222-W
ISSN:1078-8956

Safety and tolerability of long-term treatment with darolutamide in patients with metastatic castration-resistant prostate cancer

Robert Hugh JonesKarim FizaziNicholas D. JamesTeuvo L. TammelaNobuaki Matsubara18
Prostate Cancer And Prostatic Diseases
2023
2023/10/26
00 p.1-4
In patients with metastatic castration-resistant prostate cancer, darolutamide was well tolerated for 25 months, but minimal long-term safety data are available. Treatment-emergent adverse events (TEAEs) for patients receiving darolutamide for a median of 38 months (n = 13) are described in this poo...
Cancer therapyProstate cancer
10.1038/S41391-023-00740-9
ISSN:1365-7852

GDF8 and activin A are the key negative regulators of muscle mass in postmenopausal females: a randomized phase I trial

Dinko Gonzalez TrotterStephen DonahueChris WynneShazia AliProdromos Parasoglou19
Nature Communications
2025
2025/5/13
Vol.16 No.1 p.1-14
Evolutionary pressures to protect against food scarcity likely resulted in highly-conserved pathways designed to minimize energy expenditure, one of which involves the minimization of muscle mass; these mechanisms may be counter-productive in a modern world suffering from obesity and sarcopenia. Gro...
Endocrine system and metabolic diseasesMetabolic disordersObesity
10.1038/S41467-025-59380-3
ISSN:2041-1723

Trajectories of α-fetoprotein and unresectable hepatocellular carcinoma outcomes receiving atezolizumab plus bevacizumab: a secondary analysis of IMbrave150 study

Linbin LuPeichan ZhengYan PanShanshan HuangErqian Shao14
British Journal Of Cancer
2023
2023/7/8
00 p.1-6
α-fetoprotein (AFP) response has been demonstrated as a biomarker for unresectable hepatocellular carcinoma (uHCC) patients receiving immunotherapy, but its definition is still unclear. This exploratory study investigated the AFP trajectory and clinical outcomes of receiving atezolizumab plus bevaci...
Hepatocellular carcinomaPrognostic markersTumour biomarkers
10.1038/S41416-023-02334-7
ISSN:0007-0920

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

Robert E. MacLarenM. Dominik FischerJames A. GowByron L. LamEeva-Marja K. Sankila10
Nature Medicine
2023
2023/10/9
Vol.29 No.10 p.2464-2472
Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 1011 vector genomes (vg);...
Drug developmentPharmacogenetics
10.1038/S41591-023-02520-3
ISSN:1078-8956

Orforglipron for maintenance of body weight reduction: the double-blind, randomized phase 3b ATTAIN-MAINTAIN trial

Louis J. AronneDeborah B. HornCarel W. le RouxAriana M. ChaoWayne Ho14
Nature Medicine
2026
2026/5/13
00 p.1-9
Incretins have improved the management of obesity and its related complications, but maintaining these health benefits requires ongoing administration, which can be challenging. Orforglipron, a once-daily oral nonpeptide glucagon-like peptide-1 (GLP-1) receptor agonist, has demonstrated weight loss ...
ObesityPatient education
10.1038/S41591-026-04386-7
ISSN:1078-8956

Biomarker-directed targeted therapy plus durvalumab in advanced non-small-cell lung cancer: a phase 2 umbrella trial

Benjamin BesseElvire Pons-TostivintKeunchil ParkSylvia HartlPatrick M. Forde38
Nature Medicine
2024
2024/2/13
00 p.1-14
For patients with non-small-cell lung cancer (NSCLC) tumors without currently targetable molecular alterations, standard-of-care treatment is immunotherapy with anti-PD-(L)1 checkpoint inhibitors, alone or with platinum-doublet therapy. However, not all patients derive durable benefit and resistance...
Non-small-cell lung cancerTumour biomarkers
10.1038/S41591-024-02808-Y
ISSN:1078-8956

Prediction of checkpoint inhibitor immunotherapy efficacy for cancer using routine blood tests and clinical data

Seong-Keun YooConall W. FitzgeraldByuri Angela ChoBailey G. FitzgeraldCatherine Han37
Nature Medicine
2025
2025/1/6
00 p.1-12
Predicting whether a patient with cancer will benefit from immune checkpoint inhibitors (ICIs) without resorting to advanced genomic or immunologic assays is an important clinical need. To address this, we developed and evaluated SCORPIO, a machine learning system that utilizes routine blood tests (...
Cancer immunotherapyMachine learningOutcomes researchPredictive markersPrognostic markers
10.1038/S41591-024-03398-5
ISSN:1078-8956