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

Stopped early · Phase 1/Phase 2

Conditions studied: GM1 Gangliosidosis

In brief

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.

Key facts

Study ID

NCT04273269

Run by

LYSOGENE

People needed

5

Starts

2021-05-11

Expected to finish

2023-05-22

Last updated by the study team

2023-06-09

Who can join

Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Children's Hospital of Orange County (CHOC) — Orange, California, United States
• Hôpital Armand-Trousseau, Centre de Référence des Maladies Lysosomales (CRML), Service de Neuropédiatrie — Paris, France
• Manchester University NHS Foundation Trust — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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