CompletedNCT04041102
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Studying Gangliosidosis
Last synced from ClinicalTrials.gov
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- University of Pennsylvania
- Principal Investigator
- Can Ficicioglu, MD, PhD, M.DChildren's Hospital of Philadelphia
- Enrollment
- 31 enrolled
- Eligibility
- All sexes
- Timeline
- 2020 – 2024
Study locations (6)
- UCSF Benioff Children's Hospital Oakland, Oakland, California, United States
- Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
- Montreal Children's Hospital Research Institute - McGill University, Montreal, Quebec, Canada
- Gazi University, Ankara, Turkey (Türkiye)
- UCL Great Ormond Street Institute of Child Health, London, United Kingdom
Collaborators
Passage Bio, Inc.
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT04041102 on ClinicalTrials.govOther trials for Gangliosidosis
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- RECRUITINGPHASE1, PHASE2NCT03952637A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 GangliosidosisNational Human Genome Research Institute (NHGRI)
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