Spinal Muscular Atrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been treated with a gene therapy called OAV-101 (also known as Zolgensma) and have been diagnosed with Spinal Muscular Atrophy (SMA) confirmed by genetic testing, no matter when you were diagnosed.
- You (or your parent/guardian, if applicable) have agreed and given permission to take part in this registry.
Who may not be able to join:
- You are currently taking part in another clinical trial that is testing an investigational (not yet fully approved) medicine for SMA.
Important notes about eligibility:
- If you received OAV-101 (Zolgensma) through a special access or compassionate use program — such as a Managed Access Program, Expanded Access Program, Single Patient program, or Named Patient Program — you may still be eligible to join this registry, regardless of when your SMA was diagnosed (confirm with trial site).
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Phone: 1-888-669-6682
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Change in probability of survival of all patients with SMA using Kaplan Meier method to estimate; Change from baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in infants with pre-symptomatic or type I SMA; Change from baseline Hammersmith Infant Neurological Examination (HINE) in infants with pre-symptomatic, type I or type II SMA; Change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) for patients with type II and III SMA; Incidence of treatment emergent adverse events; Incidence of treatment emergent serious adverse events...
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.