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drugset / Trial / NCT05973630

ATA-200 Gene Therapy Trial in Patients With LGMDR5

NCT05973630 ↗

Phase 1 Active not recruiting 4 enrolled Atamyo Therapeutics
Non-randomizedSingle-groupOpen-labelTreatment

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).

Timeline

Start
2025-02-15
Primary completion
2026-10-30
Completion
2031-01-31

Drugs

EvaluationDrugModalityDoseRoute
Subject ATA-200 Gene therapy (AAV / viral vector) 1e+14 vg/kg Intravenous

Indications

autosomal recessive limb-girdle muscular dystrophy type 2C