Gene therapy for children with limb girdle muscular dystrophy type 2c
A Phase 1-2, Open-label, Dose Escalation Study to Evaluate the Safety of 2 Doses of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients with Gamma-sarcoglycanopathy (LGMDR5)
This study is testing a new gene therapy to see if it can safely help children with limb girdle muscular dystrophy type 2c improve their condition.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 6 (estimated) |
| Ages | 6 Years to 13 Years |
| Sex | All |
| Sponsor | Atamyo Therapeutics Industry-sponsored |
| Locations | 3 sites (Gainsville, Florida and 2 other locations) |
| Trial ID | NCT05973630 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients diagnosed with limb girdle muscular dystrophy type 2c (LGMDR5). The trial is designed as a multicenter Phase 1b study, where patients will be treated in two sequential dose cohorts. The first cohort will consist of three patients receiving a potentially effective dose, followed by a second cohort with a higher dose after safety data review. Participants will be monitored for safety and efficacy over a period of 4.5 years following treatment.
Who should consider this trial
Good fit: Ideal candidates are ambulant children aged 6 to less than 12 years with a confirmed diagnosis of LGMDR5.
Not a fit: Patients with detectable neutralizing antibodies against AAV8 or significant comorbid conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a new treatment option for children suffering from LGMDR5, potentially improving their mobility and quality of life.
How similar studies have performed: While gene therapy approaches for muscular dystrophies are being explored, this specific trial represents a novel application targeting LGMDR5.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping * Ambulant male or female patients aged 6 to less than 12 years of age at screening * Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support Exclusion Criteria: * Detectable neutralizing antibodies against AAV8 * Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50% * Respiratory assistance * Concomitant medical condition that might interfere with LGMDR5 evolution * Acute illness within 4 weeks of anticipated IMP administration * Current participation in another clinical trial with investigational medicinal product * Previous participation in gene and cell therapy trials * Any condition that would contraindicate immunosuppressant treatment * Presence of any permanent items (e.g., metal braces) precluding undergoing MRI * Any vaccination 1 month prior to planned IMP administration * Serology consistent with HIV exposure or active hepatitis B or C infection * Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation
Where this trial is running
Gainsville, Florida and 2 other locations
- Child Health Research Institute — Gainsville, Florida, United States (Recruiting)
- Hopital Trousseau — Paris, France (Not_yet_recruiting)
- Ospedale Maggiore Policlinico — Milano, Italy (Not_yet_recruiting)
Study contacts
- Study coordinator: Sophie Olivier
- Email: s.olivier@atamyo.com
- Phone: +33638682337
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.