A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age, treatment naive, sitting, and never ambulatory

drug protocol number

COAV101B12301

drug sponsor

Novartis Saudi Limited

drug study

OAV101 (formerly AVXS-101)

drug trial site

King Faisal Specialist Hospital and Research Center (Riyadh),King Fahad Specialist Hospital (Dammam)

drug status

Ongoing

drug phase