ATA-200 Gene Therapy Trial in Patients With LGMDR5

Running, not enrolling · Phase 1

Conditions studied: LGMD2C

In brief

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).

Key facts

Study ID

NCT05973630

Run by

Atamyo Therapeutics

People needed

4

Starts

2025-02-15

Expected to finish

2031-01-31

Last updated by the study team

2026-01-29

Who can join

Age: 6 and older, up to 13. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 it is running

• Child Health Research Institute — Gainsville, Florida, United States

Full record on ClinicalTrials.gov

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