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10288+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

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Showing 20 of 10288 recruiting trials

Enrolling by InvitationNCT06885424

A Long-Term Follow-Up Study of Participants Treated With A2 Biotherapeutics (A2 Bio) Gene Therapy (GT) Products

👨‍⚕️ Eric Ng, MD, A2 Biotherapeutics📍 2 sites📅 Started Aug 2025View details ↗
NARecruitingNCT03727555

IT and IV Lentiviral Gene Therapy for X-ALD

🏥 Shenzhen Geno-Immune Medical Institute📍 1 site📅 Started Aug 2025View details ↗
Phase 4RecruitingNCT07150624

The Treatment of Liver Injury After Liver Resection With Polyene Phosphatidylcholine

🏥 Anhui Provincial Hospital📍 1 site📅 Started Aug 2025View details ↗
Phase 1RecruitingNCT07038824

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

👨‍⚕️ Entrada Therapeutics Clinical Trials, Entrada Therapeutics, Inc.📍 15 sites📅 Started Aug 2025View details ↗
Phase 3RecruitingNCT07443397

Anlotinib Plus Whole-Brain Radiotherapy for Brain Metastases in SCLC Patients.

👨‍⚕️ Lei Deng, MD, National Cancer Center/Cancer Hospital, Chinese Academy of Medical Science and Peking Union Medical College📍 1 site📅 Started Aug 2025View details ↗
Enrolling by InvitationNCT07050160

Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

👨‍⚕️ Lexeo Clinical Trials, Lexeo Therapeutics📍 1 site📅 Started Aug 2025View details ↗
Phase 3RecruitingNCT07377032

TAP-GRIN: Interventional Study on Patients With GRIN-related Neurodevelopmental Disorders

🏥 Meyer Children's Hospital IRCCS📍 3 sites📅 Started Aug 2025View details ↗
Phase 2RecruitingNCT06628947

A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa

👨‍⚕️ Doron Hickey, MBChB, The Centre for Eye Research Australia (CERA)📍 5 sites📅 Started Aug 2025View details ↗
Phase 2RecruitingNCT06989359

Phase 2 Study of ADX-038 in Complement-Mediated Kidney Disease

👨‍⚕️ Aditya Patel, MD, ADARx Pharmaceuticals, Inc.📍 12 sites📅 Started Aug 2025View details ↗
NARecruitingNCT06864351

Prospective Evaluation of OptiThyDose

👨‍⚕️ Gabor Szinnai, Prof. MD, PhD, Paediatric Endocrinology, UKBB📍 2 sites📅 Started Aug 2025View details ↗
Phase 3RecruitingNCT06960213

STOP-HAE: A Phase 3 Study of ADX-324 in HAE

👨‍⚕️ Lauge Farnaes, MD, ADARx Pharmaceuticals, Inc.📍 14 sites📅 Started Aug 2025View details ↗
NARecruitingNCT07399353

Comparative Study: Fistula Rerouting vs. LIFT for High Perianal Fistula

🏥 Cairo University📍 1 site📅 Started Aug 2025View details ↗
Phase 2RecruitingNCT06866964

A Single-arm, Phase II Clinical Trial of ASPIRin to prEvent Venous Thromboembolism in Patients With Advanced Germ Cell Tumors Receiving Chemotherapy

👨‍⚕️ Landon Brown, MD, Atrium Health Wake Forest Baptist Comprehensive Cancer Center📍 3 sites📅 Started Aug 2025View details ↗
NARecruitingNCT07159035

Flexible Ureteroscopy With a Flexible and Navigable Suction Ureteral Access Sheath Versus Mini-Percutaneous Nephrolithotomy for 1-2 cm Lower Pole Kidney Stones

🏥 The First Affiliated Hospital of Guangzhou Medical University📍 1 site📅 Started Aug 2025View details ↗
Phase 2Enrolling by InvitationNCT07188844

An Open-Label Extension Study of Batoclimab in Adult Participants With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

🏥 Immunovant Sciences GmbH📍 30 sites📅 Started Aug 2025View details ↗
Phase 1RecruitingNCT07087002

GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults

👨‍⚕️ Katherine Ryan, DO, Stanford University📍 1 site📅 Started Aug 2025View details ↗
RecruitingNCT07152119

A Study to Learn About Litfulo Capsule in People With Severe Alopecia Areata in Routine Clinical Practice.

👨‍⚕️ Pfizer CT.gov Call Center, Pfizer📍 1 site📅 Started Aug 2025View details ↗
Phase 1RecruitingNCT06712810

Q702 for the Treatment of Patients With Hematologic Malignancies

👨‍⚕️ Jithma P. Abeykoon, MD, Mayo Clinic in Rochester📍 2 sites📅 Started Aug 2025View details ↗
Phase 3RecruitingNCT07200908

A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2)

🏥 Mirum Pharmaceuticals, Inc.📍 44 sites📅 Started Aug 2025View details ↗
RecruitingNCT07154524

Immunoadsorption for Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

👨‍⚕️ Johannes Dorst, University of Ulm📍 1 site📅 Started Aug 2025View details ↗
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