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Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2025-05-21

No results posted yet for this 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

  • GM1 Gangliosidosis
  • GM1 Gangliosidosis, Type I
  • GM1 Gangliosidosis, Type 2
  • Beta-Galactosidase-1 (GLB1) Deficiency

Interventions

BIOLOGICAL

PBGM01

AAVhu68 viral vector

Sponsors & Collaborators

  • Gemma Biotherapeutics

    lead INDUSTRY

Principal Investigators

  • May Orfali, MD · Gemma Biotherapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Month
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-17
Primary Completion
2026-02-28
Completion
2029-02-28
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04713475 on ClinicalTrials.gov