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10259+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

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Showing 20 of 10259 recruiting trials

Phase 3Enrolling by InvitationNCT07204938

A Long-Term Study of Navenibart in Participants With Hereditary Angioedema

🏥 Astria Therapeutics, Inc.📍 1 site📅 Started Oct 2025View details ↗
NARecruitingNCT06910306

Alpha Radiation Emitters Device (DaRT) for the Treatment of Recurrent Glioblastoma

🏥 Alpha Tau Medical LTD.📍 1 site📅 Started Oct 2025View details ↗
Phase 1RecruitingNCT07221240

Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Amisodin in Healthy Adult Subjects With Amyotrophic Lateral Sclerosis

🏥 PRG Science & Technology Co., Ltd.📍 1 site📅 Started Oct 2025View details ↗
Phase 2RecruitingNCT07161999

Study of COYA 302 for the Treatment of ALS

🏥 Coya Therapeutics📍 24 sites📅 Started Oct 2025View details ↗
Phase 1Enrolling by InvitationNCT06429007

A Safety and Feasibility Trial Protocol of Metformin in Infants After Perinatal Brain Injury

👨‍⚕️ Brian Kalish, MD, Boston Children's Hospital📍 1 site📅 Started Oct 2025View details ↗
Phase 2RecruitingNCT06790706

IMMUNORARE5: A National Platform of 5 Academic Phase II Trials Coordinated by Lyon University Hospital to Assess the Safety and the Efficacy of the IMMUNOtherapy With Domvanalimab + Zimberelimab Combination in Patients With Advanced RARE Cancers

🏥 Hospices Civils de Lyon📍 15 sites📅 Started Oct 2025View details ↗
RecruitingNCT07352332

Immune Profiling of Refractory cSLE Exposed to CD3×CD19 BiTE

🏥 The Children's Hospital of Zhejiang University School of Medicine📍 1 site📅 Started Sep 2025View details ↗
Phase 2, PHASE3RecruitingNCT07204275

A Study to Evaluate Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN)

🏥 Vertex Pharmaceuticals Incorporated📍 73 sites📅 Started Sep 2025View details ↗
Phase 1RecruitingNCT07140614

A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.

🏥 Novartis Pharmaceuticals📍 3 sites📅 Started Sep 2025View details ↗
Phase 2RecruitingNCT07197866

An Extension Trial to Test if TEV-56286 is Effective in Relieving Multiple System Atrophy

👨‍⚕️ Teva Medical Expert, MD, Teva Branded Pharmaceutical Products R&D LLC📍 5 sites📅 Started Sep 2025View details ↗
Phase 2RecruitingNCT07187375

Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old

🏥 Neurocrine Biosciences📍 3 sites📅 Started Sep 2025View details ↗
Phase 2RecruitingNCT07095127

Safety, Tolerability, and Efficacy of NVG-2089 in Participants With Immune Thrombocytopenia

🏥 Nuvig Therapeutics, Inc.📍 1 site📅 Started Sep 2025View details ↗
RecruitingNCT07234656

Patient-partnered Research in Investigating Fear of Cancer Recurrence in Kidney Cancer

👨‍⚕️ Brigitta R Villumsen, PhD, Gødstrup Hospital📍 3 sites📅 Started Sep 2025View details ↗
RecruitingNCT06865482

Clinical Course Of Disease In Participants With FA-CM

👨‍⚕️ Lexeo Clinical Trials, Lexeo Therapeutics📍 10 sites📅 Started Sep 2025View details ↗
NARecruitingNCT07289100

Novel Sensors and Artificial Intelligence for Detection of Acute Pulmonary Exacerbations in Cystic Fibrosis and Bronchiectasis

👨‍⚕️ Andres Floto, University of Cambridge📍 1 site📅 Started Sep 2025View details ↗
RecruitingNCT07242963

Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study

👨‍⚕️ Murali Chintagumpala, MD, Baylor College of Medicine📍 1 site📅 Started Sep 2025View details ↗
Phase 1, PHASE2RecruitingNCT07179640

A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1

🏥 Alesta Therapeutics📍 2 sites📅 Started Sep 2025View details ↗
Phase 1, PHASE2RecruitingNCT06970106

Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy

🏥 PYC Therapeutics📍 3 sites📅 Started Sep 2025View details ↗
EARLY_Phase 1RecruitingNCT07193810

A Study of CC312 for Relapsed/Refractory Autoimmune Diseases

🏥 CytoCares Inc📍 1 site📅 Started Sep 2025View details ↗
Phase 3RecruitingNCT07187206

Safety and Efficacy of FETO in CDH Phase III

🏥 Children's Hospital Medical Center, Cincinnati📍 1 site📅 Started Sep 2025View details ↗
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