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

No Placebo-Only Group

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years (multiple visits)

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

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Secondary study objectives

Blood and cerebrospinal fluid (CSF) biomarkers (GM1 ganglioside)

Blood and cerebrospinal fluid (CSF) biomarkers (beta-galactosidase)

Brain MRI

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