Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
STEER
A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory
2 other identifiers
interventional
126
13 countries
42
Brief Summary
This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Feb 2022
Typical duration for phase_3
42 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 11, 2021
CompletedFirst Posted
Study publicly available on registry
October 22, 2021
CompletedStudy Start
First participant enrolled
February 1, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 12, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
April 29, 2025
CompletedResults Posted
Study results publicly available
December 8, 2025
CompletedJanuary 13, 2026
December 1, 2025
2.8 years
October 11, 2021
October 27, 2025
December 18, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline at the End of Period 1 in the Hammersmith Functional Motor Scale Expanded - Total Score - in the ≥ 2 to < 18 Years Age Group
The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
Baseline, Week 52 (or Week 48)
Secondary Outcomes (9)
Change From Baseline in HFMSE Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 5 Years Age Group
Baseline, Week 52 (or Week 48)
Change From Baseline in Revised Upper Limb Module (RULM) Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 18 Years Age Group
Baseline, Week 52 (or Week 48)
Change From Baseline in the RULM Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 5 Years Age Group
Baseline, Week 52 (or Week 48)
% of Participants Who Achieved at Least a 3-point Improvement From Baseline in HFMSE Total Score at the End of Follow-up Period 1 in the ≥ 2 to < 18 Years Age Group
Baseline, Week 52 (or Week 48) (end of Period 1)
% of Participants Who Achieved at Least a 3-point Improvement From Baseline in HFMSE Total Score at the End of Follow-up Period 1 for Participants Aged ≥ 2 to < 5 Years
Baseline, Week 52 (or Week 48)(end of Period 1)
- +4 more secondary outcomes
Study Arms (2)
OAV101 in Treatment Period 1; Sham Control in Treatment Period 2
EXPERIMENTALOAV101 administered as a single, one-time intrathecal dose of 1.2 x 10\^14 vector genomes (vg) in Treatment Period 1; Sham Control in Treatment Period 2 (Week 52 +1 day).
Sham control in Treatment Period 1; OAV101 in Treatment Period 2
SHAM COMPARATORA skin prick in the lumbar region in Treat Period 1; OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10\^14 vector genomes (vg) in Treatment Period 2 (Week 52 +1 day)
Interventions
Gene therapy
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
You may qualify if:
- 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.
You may not qualify if:
- Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to \> 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab.
- Infectious process (e.g., viral, bacterial) or febrile illness within 30 days prior 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 in a sitting position
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (42)
Connecticut Children's Medical Center
Farmington, Connecticut, 06032, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Clinic for Special Children
Strasburg, Pennsylvania, 17579, United States
St Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507, United States
Children's Specialty Group/CHKD
Norfolk, Virginia, 23507, United States
Novartis Investigative Site
Curitiba, Paraná, 81520-060, Brazil
Novartis Investigative Site
São Paulo, São Paulo, 05403 000, Brazil
Novartis Investigative Site
Beijing, Beijing Municipality, 100034, China
Novartis Investigative Site
Chongqing, Chongqing Municipality, 400010, China
Novartis Investigative Site
Guangzhou, Guangdong, 510623, China
Novartis Investigative Site
Shenzhen, Guangdong, 518034, China
Novartis Investigative Site
Chengdu, Sichuan, 610041, China
Novartis Investigative Site
Hangzhou, Zhejiang, 310052, China
Peking University First Hospital
Beijing, 100034, China
Novartis Investigative Site
Beijing, 100069, China
Novartis Investigative Site
Beijing, 100730, China
Novartis Investigative Site
Copenhagen, 2100 O, Denmark
Paediatric Neurology
Copenhagen, 2100 O, Denmark
Sir Ganga Ram Hospital
New Delhi, National Capital Territory of Delhi, 110 060, India
Novartis Investigative Site
New Delhi, National Capital Territory of Delhi, 110029, India
Novartis Investigative Site
New Delhi, National Capital Territory of Delhi, 110060, India
AIIMS, Ansari Nagar
New Delhi, New Delhi, 110029, India
Novartis Investigative Site
Kolkata, West Bengal, 700094, India
Novartis Investigative Site
Hyderabad, 500034, India
Novartis Investigative Site
Mumbai, 400016, India
P.D. Hinduja National Hospital & MRC
Mumbai, 400016, India
Novartis Investigative Site
Kuala Lumpur, 50300, Malaysia
Novartis Investigative Site
Kuala Lumpur, 59100, Malaysia
Hospital Civil De Guadalajara Fray Antonio Alcalde
Guadalajara, Jalisco, 44280, Mexico
Novartis Investigative Site
Guadalajara, Jalisco, 44280, Mexico
Novartis Investigative Site
Mexico City, Mexico City, 06720, Mexico
Novartis Investigative Site
Riyadh, 11211, Saudi Arabia
Novartis Investigative Site
Singapore, 119074, Singapore
Novartis Investigative Site
Cape Town, 7925, South Africa
Red Cross War Memorial Childrens Hospital
Cape Town, 7925, South Africa
Kaohsiung Medical University Hospital
Kaohsiung City, 80756, Taiwan
Novartis Investigative Site
Kaohsiung City, 80756, Taiwan
Novartis Investigative Site
Bangkok, 10700, Thailand
Siriraj Hospital
Bangkok, 10700, Thailand
National Children's Hospital
Hanoi, 100000, Vietnam
Novartis Investigative Site
Hanoi, 100000, Vietnam
Related Publications (1)
Proud CM, Vu DC, Wilmshurst JM, Sanmaneechai O, Gulati S, Xiong H, Moreno HC, Tay SKH, Thong MK, Born AP, Banzzatto Ortega A, Jong YJ, Al-Muhaizea MA, Lee AW, Visootsak J, Tauscher-Wisniewski S, Alecu I, Parlikar R, Finkel RS; STEER Study Group. Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial. Nat Med. 2026 Feb;32(2):481-487. doi: 10.1038/s41591-025-04103-w. Epub 2025 Dec 8.
PMID: 41360993DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 11, 2021
First Posted
October 22, 2021
Study Start
February 1, 2022
Primary Completion
November 12, 2024
Study Completion
April 29, 2025
Last Updated
January 13, 2026
Results First Posted
December 8, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.