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Long-term follow-up for patients with spinal muscular atrophy treated with OAV101

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials

Phase 3 Interventional Novartis · NCT05335876

This study is testing the long-term safety and effectiveness of the OAV101 treatment in patients with spinal muscular atrophy who have already taken part in a previous trial.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment175 (estimated)
Ages0 Years to 100 Years
SexAll
SponsorNovartis Industry-sponsored
Locations32 sites (Norfolk, Virginia and 31 other locations)
Trial IDNCT05335876 on ClinicalTrials.gov

What this trial studies

This global, prospective, multi-center study aims to evaluate the long-term safety and efficacy of OAV101 in patients who previously participated in an OAV101 clinical trial. The study includes a Baseline Visit followed by two follow-up periods over five years, with assessments conducted at investigational sites. For the first two years, follow-up visits will occur every six months, while the last three years will involve annual visits to monitor patient outcomes.

Who should consider this trial

Good fit: Ideal candidates are individuals who have previously participated in an OAV101 clinical trial.

Not a fit: Patients who have not participated in an OAV101 clinical trial will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term effects of OAV101 treatment for spinal muscular atrophy.

How similar studies have performed: Other studies on gene therapies for spinal muscular atrophy have shown promising results, indicating a potential for success in this long-term follow-up.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Participated in an OAV101 clinical trial.
2. Written informed consent must be obtained before any assessment is performed.
3. Patient/Parent/legal guardian willing and able to comply with study procedures.

Exclusion Criteria:

There are no exclusion criteria for this study.

Where this trial is running

Norfolk, Virginia and 31 other locations

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 AtrophyZolgensmaOAV101AVXS 101gene therapyMuscle atrophySBMAspinal and bulbar 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.