← Back to Search

Gene Therapy

LYS-GM101 for GM1 Gangliosidosis

Phase 1 & 2
Waitlist Available
Research Sponsored by LYSOGENE
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years (multiple visits)
Awards & highlights

Summary

This trial is testing a new gene therapy called LYS-GM101 for babies with a severe genetic disorder called GM1 gangliosidosis. The therapy aims to deliver a healthy gene to the brain to help prevent damage. The study will first check if the treatment is safe and then see if it works over several years. LYS-GM101 is designed to deliver a healthy GLB1 gene to treat GM1 gangliosidosis, a disorder caused by mutations in the GLB1 gene leading to β-galactosidase deficiency.

Eligible Conditions
  • GM1 Gangliosidosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years (multiple visits)
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years (multiple visits) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Stage 1: Assessment of humoral immune response by measurement of antibodies anti-AAV and anti-beta-galactosidase (ELISA) and cellular immune response by beta-galactosidase-specific T-cell proliferation assay
Stage 1: Change from baseline in biochemistry laboratory parameters
Stage 1: Change from baseline in coagulation and hematology laboratory parameters
+7 more
Secondary study objectives
Blood and cerebrospinal fluid (CSF) biomarkers (GM1 ganglioside)
Blood and cerebrospinal fluid (CSF) biomarkers (beta-galactosidase)
Brain MRI
+3 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: 8x10^12 vg/Kg LYS-GM101Experimental Treatment1 Intervention
Subjects will receive a single infusion: 8x10\^12 vg/Kg LYS-GM101

Find a Location

Who is running the clinical trial?

LYSOGENELead Sponsor
5 Previous Clinical Trials
76 Total Patients Enrolled
Clinical OperationsStudy DirectorLYSOGENE
19 Previous Clinical Trials
2,306 Total Patients Enrolled
~1 spots leftby Sep 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top