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

A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Patients Type 2 Spinal Muscular Atrophy (SMA) Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

ClinicalTrials.gov Identifier: NCT05089656

Novartis Reference Number: COAV101B12301

Last Update: May 09, 2022

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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.

Type 2 Spinal Muscular Atrophy
Phase 3
Overall status 
Start date 
Feb 01, 2022
Completion date 
Oct 21, 2024
2 Years - 17 Years (Child)


Gene therapy
Sham control
The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur.

Eligibility Criteria

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

Study Locations

United States
Children's Specialty Group/CHKD
Norfolk, 23507 - Virginia
Contact: Crystal Proud, M.D. (757-668-5755) - [email protected] - Crystal Proud, M.D.
United States
Novartis Investigative Site
Singapore, 119074


Novartis Gene Therapies Medical Information (US, Asia-Pacific, Latin America, and Canada)
Novartis Gene Therapies EMEA Medical Information (Europe, Middle East and Africa)

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