Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Active Phase 1 study of GM1 Gangliosidosis and GM1 Gangliosidosis, Type I, sponsored by Gemma Biotherapeutics.
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. According to ClinicalTrials.gov, the official US trial registry.
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
Primary Outcome
Assess the number of treatment-related adverse events (AEs) and serious adverse events (SAEs) as characterized by CTCAEv5.0
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 |
What the registry record for NCT04713475 still lists
NCT04713475 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 103-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (75% lower).
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 lists 7 locations in 5 states (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.
Learn More About Clinical Trials
Similar trials for GM1 Gangliosidosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
RECRUITING · Phase 3
-
A Natural History Study of the Gangliosidoses
RECRUITING
-
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
COMPLETED
-
Interviews and Video Capture in Patients With GM1 Gangliosidosis
COMPLETED
-
Stem Cell Transplant for Inborn Errors of Metabolism
COMPLETED · Phase 2
-
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
COMPLETED
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02646475 · 26 participants · Phase 1
Metabolic Effects of Angiotensin-(1-7)
- NCT03603223 · 26 participants · Phase 2
Pembrolizumab in Treating Participants With Leukoplakia
- NCT04063592 · 26 participants · NA
AMI Construction in Lower Extremity Residual Limbs
- NCT04906460 · 26 participants · Phase 1
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI