Clinical Trials Logo

Gangliosidosis, GM1 clinical trials

View clinical trials related to Gangliosidosis, GM1.

Filter by:
  • Active, not recruiting  
  • Page 1

NCT ID: NCT05109793 Active, not recruiting - Sandhoff Disease Clinical Trials

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

PRONTO
Start date: February 22, 2022
Phase:
Study type: Observational

The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).

NCT ID: NCT04713475 Active, not recruiting - GM1 Gangliosidosis Clinical Trials

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

Imagine-1
Start date: March 17, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

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