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NCT04713475 · ClinicalTrials.gov registry record · Phase 1
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
A Phase 1 study of GM1 Gangliosidosis and GM1 Gangliosidosis, Type I, sponsored by Gemma Biotherapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 26
- Enrollment target
- 7
- Study locations
NCT04713475 is a Phase 1 study of GM1 Gangliosidosis and GM1 Gangliosidosis, Type I that is active but no longer recruiting, run by Gemma Biotherapeutics. The registered enrollment target is 26 participants, below the 103-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (75% lower). The trial reports 7 study locations across 5 states.
The verdict
NCT04713475, a Phase 1 study of GM1 Gangliosidosis and GM1 Gangliosidosis, Type I, is active but no longer recruiting, sponsored by Gemma Biotherapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 26 participants
- Enrollment target
- 7
- Study locations
Study Summary
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis
Conditions Studied
Interventions
- BIOLOGICAL PBGM01
Study Locations (7)
Other
- Hospital de Clínicas de Porto Alegre (HCPA) - Porto Alegre
- Gazi University - Ankara
- Great Ormond Street Hospital - London
California
- Benioff Children's Hospital - Oakland
Minnesota
- University of Minnesota - Minneapolis
New Jersey
- Children's Hospital at St. Peter's University Hospital - New Brunswick
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2021-03-17 |
| Est. Completion | 2029-02 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04713475
The ClinicalTrials.gov registry entry for NCT04713475 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 103-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (75% lower). The listed sponsor is Gemma Biotherapeutics, which has 1 total studies on file at ClinicalTrials.gov.
The record links to 4 conditions, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 1 intervention - of which PBGM01 is the first listed.
NCT04713475 reports 7 study locations spanning 5 distinct geographic areas - top geographies include Other, California, Minnesota.
Frequently Asked Questions
What is clinical trial NCT04713475 about?
NCT04713475 is a clinical study titled "Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis". PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infanti...
What is the current status of trial NCT04713475?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 26 participants. The study started on 2021-03-17. Estimated completion is 2029-02.
What conditions does trial NCT04713475 study?
This clinical trial studies the following conditions: GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency.
What interventions are being tested in trial NCT04713475?
The interventions under investigation include: PBGM01 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04713475?
This trial is sponsored by Gemma Biotherapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04713475 being conducted?
This trial has 7 study locations across California, Minnesota, New Jersey, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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