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Registry for patients with spinal muscular atrophy

A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Observational Novartis · NCT04174157

This study is tracking patients with spinal muscular atrophy over 15 years to see how well the new treatment OAV-101 (Zolgensma) works and how the disease progresses in real life.

Quick facts

Study typeObservational
Enrollment700 (estimated)
SexAll
SponsorNovartis Industry-sponsored
Locations99 sites (Phoenix, Arizona and 98 other locations)
Trial IDNCT04174157 on ClinicalTrials.gov

What this trial studies

This observational registry aims to assess the long-term outcomes of patients diagnosed with spinal muscular atrophy (SMA) in light of new treatment options, particularly focusing on the safety and effectiveness of OAV-101 (Zolgensma). The study will follow patients for up to 15 years, allowing researchers to gather data on the natural progression of the disease and the impact of treatment in a real-world clinical setting. Patients will receive standard clinical care as determined by their healthcare providers, without additional protocol-driven interventions.

Who should consider this trial

Good fit: Ideal candidates include patients with a genetically confirmed diagnosis of SMA who have been treated with OAV-101.

Not a fit: Patients currently enrolled in interventional clinical trials involving investigational treatments for SMA may not benefit from this registry.

Why it matters

Potential benefit: If successful, this registry could provide valuable insights into the long-term effects of SMA treatments, improving patient care and outcomes.

How similar studies have performed: While observational studies have been conducted in similar contexts, this registry specifically focuses on the long-term outcomes of SMA patients treated with OAV-101, making it a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
* Appropriate consent/assent has been obtained for participation in the registry

Exclusion Criteria:

\- Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA.

Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.

Where this trial is running

Phoenix, Arizona and 98 other locations

+49 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Spinal Muscular Atrophy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.