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

Phase 1/2 Active not recruiting 26 enrolled Gemma Biotherapeutics

Non-randomizedSequentialOpen-labelTreatment

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

Timeline

Start: 2021-03-17
Primary completion: 2028-02
Completion: 2029-02

Publications

Zhang X, Brind'Amour K, King KE, Hartmaier S, Harris K, Weinstein DA, Girman CJ. Strategy for Generating Blinded Evidence for Single-Arm Trials with External Controls Using Expert Review of Home Video. Ther Innov Regul Sci. 2023 Nov;57(6):1304-1313. doi: 10.1007/s43441-023-00568-4. Epub 2023 Aug 17.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	PBGM01	Gene therapy (AAV / viral vector)	3.3e+10 unknown	Other
Subject	PBGM01	Gene therapy (AAV / viral vector)	1.1e+11 unknown	Other
Subject	PBGM01	Gene therapy (AAV / viral vector)	2.2e+11 unknown	Other

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

Summary

Timeline

Publications

Drugs

Indications