Phase 1/2 Study in Pediatric Subjects with GM1 Gangliosidosis
Research type
Research Study
Full title
A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered into the Cisterna Magna of Pediatric Participants with Type 1 (Early Onset Infantile) and Type 2a (Late Onset Infantile) GM1 Gangliosidosis (Imagine-1 Study)
IRAS ID
284305
Contact name
Julien Baruteau
Contact email
Sponsor organisation
GEMMA Biotherapeutics
Eudract number
2020-001109-22
Clinicaltrials.gov Identifier
Duration of Study in the UK
7 years, 6 months, 31 days
Research summary
GM1 Gangliosidosis (GM1), an inherited disease, is one of a family of diseases referred to as lysosomal storage diseases. The child affected has mutations in the human galactosidase beta 1 gene (GLB1), which codes for a specific enzyme called beta-galactosidase (β‑gal). β‑gal assists in breaking down chemicals. Because GLB1 does not work properly, specific chemicals cannot be broken down, leading to a build-up in the body. GM1 manifests as a spectrum of disease, where the earlier the onset the more rapid and severe the disease. There are no approved medical or curative therapies for the infantile or late infantile child with GM1.
Passage Bio has manufactured a gene therapy called PBGM01, which carries a normal copy of the GLB1 gene. It is thought that if a copy of the normal gene is given to patients with GM1, then the disease may be treated. This main purpose of this study is to test if PBGM01 will be safe in subjects aged 4-24 months with Type 1 (Early Onset Infantile) and Type 2a (Late Onset Infantile) GM1. In addition, the study will look to see if PBGM01 provides benefit by slowing or stopping the worsening condition usually seen in patients with GM1.
This study will test two dose levels of PBGM01 (a low dose and a high dose). A total of up to 28 children with GM1 worldwide will receive one of these two doses of PBGM01. All subjects will receive a single dose of PBGM01 administered by intra-cisterna magna (ICM injection), which is at the back of the neck at the top of the subject’s spine. All subjects will be assessed for safety, tolerability, efficacy and durability of effect for 5 years post administration of PBGM01.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
21/LO/0039
Date of REC Opinion
5 Mar 2021
REC opinion
Further Information Favourable Opinion