Status:
RECRUITING
ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5
Lead Sponsor:
Atamyo Therapeutics
Conditions:
LGMD2C
Eligibility:
All Genders
6-13 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5)....
Detailed Description
This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200 for the treatment of LGMDR5. The dose escalation phase will enroll ambulant patients with LGMDR5. Two dose c...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
February 15 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 31 2031
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT05973630
Start Date
February 15 2025
End Date
January 31 2031
Last Update
October 6 2025
Active Locations (3)
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1
Child Health Research Institute
Gainesville, Florida, United States, 32610
2
Hopital Trousseau
Paris, France
3
Ospedale Maggiore Policlinico
Milan, Italy