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Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
NCT04713475 · View on ClinicalTrials.gov ↗
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 |
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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. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Gemma Biotherapeutics, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04713475 reports 7 study locations spanning 5 distinct geographic areas — top geographies include Other, California, Minnesota. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04713475?
The interventions under investigation include: PBGM01 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04713475?
This trial is sponsored by Gemma Biotherapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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