OAV101 for Spinal Muscular Atrophy
(STEER Trial)
Recruiting at 41 trial locations
NG
NG
NG
NG
NP
NM
NM
Overseen ByNovartis Medical Information - International
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Waitlist Available
Sponsor: Novartis Pharmaceuticals
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
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.
Eligibility Criteria
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
My condition is confirmed to be 5q spinal muscular atrophy.
I can sit by myself but have never been able to walk on my own.
I have never taken SMN-targeting therapies like Evrysdi or Spinraza.
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Exclusion Criteria
I need a machine to help me breathe, either through a mask for more than 6 hours a day or through a tracheostomy.
You have significant problems with feeling or sensation in your nerves during a medical exam.
My test shows high levels of AAV9 antibodies.
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Treatment Details
Interventions
- OAV101 (Gene Therapy)
- Sham control (Procedure)
Trial OverviewThe trial tests the effectiveness of OAV101 given through the spine compared to a sham control in kids with SMA over 15 months. It aims to see if this treatment improves their condition without causing harm.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: OAV101Experimental Treatment1 Intervention
OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10\^14 vector genomes (vg).
Group II: Sham controlPlacebo Group1 Intervention
A skin prick in the lumbar region without any medication.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Trials
2,963
Recruited
4,275,000+
Founded
1996
Headquarters
Basel, Switzerland
Known For
Precision medicine
Top Products
Gleevec, Cosentyx, Entresto, Kisqali
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