← 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

Study Summary

This trial is testing a gene therapy for a disease that causes damage to the nervous system. They will be testing how safe and effective it is.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any patient slots available for this research endeavor?

"According to the records on clinicaltrials.gov, this investigation is no longer seeking new participants as recruitment ended in 8/30/2022 since first posting on 5/11/2021. However, there are still 7 other medical trials that are currently calling for volunteers."

Answered by AI

What is the primary goal of this research endeavor?

"The primary outcome measure for this trial will be observed over a 6-month period, and focuses on the incidence of any adverse events or serious health risks. Secondary measurements include developmental changes (as gauged by VABS-II, BSID-III or KABC-II), plus blood and cerebrospinal fluid biomarkers such as beta galactosidase activity levels."

Answered by AI
~1 spots leftby Mar 2025