Status:
TERMINATED
A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis
Lead Sponsor:
LYSOGENE
Conditions:
GM1 Gangliosidosis
Eligibility:
All Genders
Up to 3 years
Phase:
PHASE1
PHASE2
Brief 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 safet...
Detailed Description
GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons and progressive neurodegeneration. There are three ...
Eligibility Criteria
Inclusion
- Documented GM1 gangliosidosis diagnosis based on genotyping confirming the β-gal gene mutations and/or documented deficiency of β-gal enzyme by laboratory testing
- Children with early infantile GM1 gangliosidosis less than 12 months of age with ability to swallow
- Children with late infantile GM1 gangliosidosis less than 3 years of age with ability to sit
Exclusion
- Uncontrolled seizure disorder. Patients who are stable on anti-convulsive medications may be included
- More than 40% brain atrophy as measured by MRI total brain volume at screening
- Current participation in a clinical trial of another investigational medicinal product
- Past participation in a gene therapy trial
- History of hematopoietic stem cell transplantation
- Any condition that would contraindicate treatment with immunosuppressant therapy
- Presence of concomitant medical condition or anatomical abnormality precluding lumbar puncture or intracisternal injection
- Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
- History of non-GM1 gangliosidosis medical condition that would confound scientific rigor or interpretation of results
- Rare and unrelated serious comorbidities, e.g., Down syndrome, intraventricular hemorrhage in the new-born period, extreme low birth weight (\<1500 grams) or known bleeding disorders
- Any vaccination 1 month prior to the planned immunosuppressant treatment
- Serology consistent with HIV exposure or consistent with active hepatitis B or C infection
- Grade 2 or higher lab abnormalities for Liver function tests (LFT), bilirubin, creatinine, hemoglobin, white blood cell (WBC) count, platelet count, prothrombin time (PT), and partial thromboplastin time (PTT), according to CTCAE v5.0
Key Trial Info
Start Date :
May 11 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 22 2023
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT04273269
Start Date
May 11 2021
End Date
May 22 2023
Last Update
June 9 2023
Active Locations (3)
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1
Children's Hospital of Orange County (CHOC)
Orange, California, United States, 92868
2
Hôpital Armand-Trousseau, Centre de Référence des Maladies Lysosomales (CRML), Service de Neuropédiatrie
Paris, France, 75012
3
Manchester University NHS Foundation Trust
Manchester, United Kingdom, M13 9WL