ATA-200 Gene Therapy Trial in Patients With LGMDR5
NCT05973630 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4
Last updated 2026-01-29
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).
Conditions
- LGMD2C
Interventions
- BIOLOGICAL
-
ATA-200
single intravenous infusion
Sponsors & Collaborators
-
Atamyo Therapeutics
lead INDUSTRY
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 6 Years
- Max Age
- 13 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2025-02-15
- Primary Completion
- 2026-10-30
- Completion
- 2031-01-31
- FDA Drug
- Yes
Countries
- United States
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