🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

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

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Showing 5 of 5 recruiting trials for “gm2-gangliosidosis-ab-variant

Phase 2RecruitingNCT07399704

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

🏥 Azafaros A.G.📍 3 sites📅 Started Feb 2026View details ↗
Phase 3RecruitingNCT07082543

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

🏥 Azafaros A.G.📍 36 sites📅 Started Jun 2025View details ↗
RecruitingNCT05007990

Caregiving Networks Across Disease Context and the Life Course

👨‍⚕️ Laura M Koehly, Ph.D., National Human Genome Research Institute (NHGRI)📍 1 site📅 Started Sep 2022View details ↗
Phase 1, PHASE2RecruitingNCT03952637

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

👨‍⚕️ Cynthia J Tifft, M.D., National Human Genome Research Institute (NHGRI)📍 1 site📅 Started Aug 2019View details ↗
RecruitingNCT00668187

A Natural History Study of the Gangliosidoses

👨‍⚕️ Jeanine R. Jarnes, PharmD, University of Minnesota - Fairview📍 1 site📅 Started Dec 2010View details ↗

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