Active, not recruitingPHASE1, PHASE2NCT04713475
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
Studying GM1 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
- Gemma Biotherapeutics
- Principal Investigator
- May Orfali, MD, M.DGemma Biotherapeutics
- Intervention
- PBGM01(biological)
- Enrollment
- 26 enrolled
- Eligibility
- All sexes
- Timeline
- 2021 – 2029
Study locations (7)
- Benioff Children's Hospital, Oakland, California, United States
- University of Minnesota, Minneapolis, Minnesota, United States
- Children's Hospital at St. Peter's University Hospital, New Brunswick, New Jersey, United States
- The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Hospital de Clínicas de Porto Alegre (HCPA), Porto Alegre, Brazil
- Gazi University, Ankara, Turkey (Türkiye)
- Great Ormond Street Hospital, London, United Kingdom
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 NCT04713475 on ClinicalTrials.gov