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A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

NCT04273269 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2023-06-09

No results posted yet for this study

Summary

LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system. This study will assess, in a 2-stage adaptive-design, the safety and efficacy of treatment in subjects with infantile GM1 gangliosidosis.

Conditions

  • GM1 Gangliosidosis

Interventions

GENETIC

LYS-GM101

LYS-GM101 is an adeno-associated viral vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene, formulated as a suspension for injection

Sponsors & Collaborators

  • LYSOGENE

    lead INDUSTRY

Principal Investigators

  • Clinical Operations · LYSOGENE

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-11
Primary Completion
2023-05-22
Completion
2023-05-22
FDA Drug
Yes

Countries

  • United States
  • France
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04273269 on ClinicalTrials.gov