Open-Label Adaptive rh.10 treatment of GM1 gangliosidosis (P1-GM-101)
Research type
Research Study
Full title
An open-label adaptive design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human β-galactosidase cDNA for the treatment of GM1 gangliosidosis
IRAS ID
289445
Contact name
Simon Jones
Contact email
Sponsor organisation
Lysogene
Eudract number
2019-004949-32
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
19440, IND Number
Duration of Study in the UK
5 years, 0 months, 5 days
Research summary
GM1 gangliosidosis is a severe disease that starts in early childhood and destroys nerve cells in the brain and spinal cord over time. Patients with GM1 gangliosidosis are missing or do not have enough of a protein called beta-galactosidase. There is no disease-modifying treatment currently available for GM1 gangliosidosis.
The study drug called LYS-GM101 is an experimental gene therapy that contains a normal copy of the gene coding for the beta-galactosidase protein, the gene that is defective in patients with GM1 gangliosidosis. A modified virus will be used to deliver the gene in the CSF via the cisterna magna. The virus has been modified so that it cannot cause disease.
Cerebrospinal fluid (CSF) tap will be collected at the opportunity of each MRI and in visit 2 and analysed for disease biomarkers.
This study will assess, in a 2-stage adaptive-design, the safety and efficacy of treatment in subjects with early and late infantile GM1 gangliosidosis. After the administration of the product, patients will be FU for two years.
One UK Site will be participating in the study.
Safety will be monitored through the entire study period.REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/1167
Date of REC Opinion
6 Jan 2021
REC opinion
Further Information Favourable Opinion