not yet recruiting

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

key information

study id #: NCT05089656

condition: Type 2 Spinal Muscular Atrophy

status: Not yet recruiting


To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age over a 15 month trial duration.

intervention: OAV101, Sham control


last updated: February 04, 2022