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

Key Inclusion criteria:

Diagnostic confirmation during screening period of 5q SMA
The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
Onset of clinical signs and symptoms at ≥ 6 months of age
A complete Hammersmith Functional Motor Scale - Expanded (HFMSE) assessment during the screening period for trial eligibility
Able to sit independently at screening, but has never had the ability to walk independently.

Key Exclusion criteria:

Anti-adeno-associated virus serotype 9 (AAV9) antibody titers > 1:50 at screening as determined by sponsor designated lab. NOTE: A negative anti-AAV9 antibody titer is defined as ≤ 1:50.
Infectious process (e.g. viral, bacterial) or febrile illness prior to start of screening, and up to OAV101 treatment or sham procedure
Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), > upper limit of normal (ULN).
Requiring invasive ventilation, awake noninvasive ventilation for > 6 hours during a 24-hour period, noninvasive ventilation for > 12 hours during a 24-hour period or requiring tracheostomy
Complications at screening that would interfere with motor efficacy assessments including but not limited to, severe contractures or Cobb angle > 40
Surgery for scoliosis or hip fixation in the 12 months prior to Screening or planned within the next 64 weeks
Clinically significant sensory abnormalities in the neurological examination at Screening