OAV101 for Spinal Muscular Atrophy
(STEER Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Are You a Good Fit for This Trial?
This trial is for children aged 2 to <18 with Type 2 spinal muscular atrophy (SMA) who can sit but not walk independently, have had symptoms since ≥6 months old, and haven't used any SMN-targeting therapies. They must pass a motor function test and not have severe contractures, liver issues, recent or planned spine/hip surgery, sensory abnormalities, high AAV9 antibody levels, recent infections or require extensive ventilation.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Period 1
Participants receive OAV101 or sham procedure with in-patient hospitalization on Study Day 1, Day 2, and Day 3 (optional)
Follow-up Period 1
Participants are monitored for safety and efficacy assessments
Treatment Period 2
Eligible participants receive OAV101 or sham procedure with in-patient hospitalization
Follow-up Period 2
Participants are monitored for safety and efficacy assessments
Long-term follow-up
Participants may enroll in a long-term follow-up study to monitor long-term safety and efficacy
What Are the Treatments Tested in This Trial?
Interventions
- OAV101
- Sham control
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD