ATA-200 Gene Therapy Trial in Patients With LGMDR5
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 intravenous ATA-200 for the treatment of LGMDR5.
The study will enroll ambulant patients with LGMDR5. Patients will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. Patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies.
Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months.
All subjects will be followed up for an additional 4.5 years after completion of the initial evaluation period.
official title
A Phase 1-2, Open-label Study to Evaluate the Safety of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)