YS-GM101 gene therapy trial in patients with GM1 gangliosidosis
- Conditions
- GM1 gangliosidosisMedDRA version: 20.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2019-004949-32-FR
- Lead Sponsor
- ysogene
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 18
Stage 1: Safety phase
1. Confirmed GM1 gangliosidosis diagnosis based on genotyping confirming the ß-gal gene mutations and/or deficiency of ß-gal enzymatic activity
2. Study population:
a. Children with early infantile GM1 gangliosidosis less than 12 months of age at screening and with swallowing ability (presence of feeding tube is permitted)
b. Children with late infantile GM1 gangliosidosis less than 3 years of age at screening and with ability to sit with hand support or props)
3. Signed written informed consent before any study related procedure is performed
4. Patient medical status sufficiently stable and ability of parents/legal guardian, in the opinion of the Investigator, to adhere to the study visit schedule and other protocol requirements.
Stage 2: Confirmatory phase
Following DSMB review of 3-month safety from patients enrolled during the safety phase, enrollment in Stage 2 will be initiated. After the interim analysis on 6-month data of patients enrolled in the safety cohort, the final eligibility criteria for patients with early and late infantile GM1 gangliosidosis will be confirmed or may be revised based on the results of the interim analysis.
Are the trial subjects under 18? yes
Number of subjects for this age range: 18
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Uncontrolled seizure disorder. Patients who are stable on anticonvulsive medications may be included
2. More than 40% brain atrophy as measured by total brain volume on MRI
3. Current participation in a clinical trial of another investigational medicinal product
4. Past participation in gene therapy trials
5. History of hematopoietic stem cell transplantation
6. Any condition that would contraindicate treatment with immunosuppressants
7. Presence of concomitant medical condition or anatomical abnormality precluding lumbar puncture or intracisternal injection
8. Presence of any permanent item (e.g., metal braces) precluding undergoing MRI
9. History of non-GM1 gangliosidosis medical condition that would confound scientific rigor or interpretation of results
10. 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
11. Any vaccination 1 month prior to immunosuppressant treatment
12. Serology consistent with HIV exposure or consistent with active hepatitis B or C infection
13. Grade 2 or higher lab abnormalities for LFT, bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT, according to CTCAE latest version (v5.0, dated 27 Nov 2017).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method