Clinical Trial

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

Study acronym: Imagine-1
Active, Not Recruiting Phase 1/2
View on ClinicalTrials.gov →
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
Protocol Amendment History 23 amendments
This ClinicalTrials.gov record has been amended 23 times since 2021-01-14; most recent amendment 2026-06-26.
Status change: Recruiting → Active, Not Recruiting 2024-01-16
Status change: Active, Not Recruiting → Recruiting 2023-06-26
Status change: Recruiting → Active, Not Recruiting 2023-06-14
Status change: Active, Not Recruiting → Recruiting 2023-06-12
Status change: Recruiting → Active, Not Recruiting 2023-01-05
Status change: Not Yet Recruiting → Recruiting 2021-02-23
Trial Details
NCT Number NCT04713475
Lead Sponsor Gemma Biotherapeutics
Conditions GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Enrollment 26 participants
Start Date 2021-03-17
Primary Completion 2028-02 (estimated)
Study Completion 2029-02 (estimated)
Updated on ClinicalTrials.gov 2026-06-30