Trial results
AI generated results 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
According to the results reported on ClinicalTrials.gov, this trial (NCT05386680) enrolled 27 participants who received a single dose of the gene therapy OAV101 (at a dose level of 1.2×10¹⁴ viral genome particles). Of the 27 who started, 25 completed the study and 2 did not. The trial was primarily focused on tracking unintended medical events (called adverse events) that occurred after treatment, and secondarily on measuring changes in participants' physical abilities over 52 weeks using two standard movement assessment tools used in spinal muscular atrophy (SMA). The reported data shows that, out of 27 participants, 27 experienced at least one treatment-emergent adverse event (an unintended medical occurrence that happened after receiving the treatment). Thirteen participants experienced adverse events considered related to the treatment. The trial also tracked specific categories of concern — including liver-related events, low platelet counts, heart-related events, and nerve-related signs — with small numbers of participants (ranging from 1 to 8 across different categories) recorded under these special-interest groups. The standard deviations and full breakdown labels for many of these sub-counts were not fully reported in the submitted data. The reported data shows that, on the movement assessment scales measured at 52 weeks, participants' scores on the Hammersmith Functional Motor Scale Expanded (HFMSE — a 0–66 motor ability score) changed by an average of 0.17 points from where they started (with a separate statistical estimate of 1.05 points). On the Revised Upper Limb Module (RULM — a 0–37 upper limb ability score), the average change from baseline was 0.29 points. No standard deviation or margin-of-error figures were included in the submitted results for these measures. These numbers describe what was recorded in this group; they do not tell us what the results mean for any individual. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 41360995) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Spinal Muscular Atrophy Trial, Completed
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 diagnosed with Spinal Muscular Atrophy (SMA)
- You are between 2 and 17 years old
- You have already received at least four loading doses of nusinersen (Spinraza®), or have been taking risdiplam (Evrysdi®) for at least 3 months before the screening visit
- You are currently showing symptoms of SMA as described in the trial guidelines (confirm with trial site)
Who may not be able to join:
- Your blood tests show a high level of antibodies against a specific virus called AAV9, which is used to deliver the trial treatment
- Your screening blood tests or other test results show significant abnormalities
- You have a medical reason why a lumbar puncture (a procedure involving a needle in the lower back) would not be safe for you
- You received nusinersen (Spinraza®) or risdiplam (Evrysdi®) within a specific time period before the start of the trial (confirm exact timeframe with trial site)
- You received any vaccinations within 2 weeks before receiving the trial treatment
- You were hospitalised for a breathing problem or for nutritional support within 2 months before screening, or you have a major surgery planned
- You had an infection or illness with fever in the 30 days before receiving the trial treatment
- You currently need a breathing machine (invasive ventilation) to breathe
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
Australian sites
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
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Overview of Treatment-emergent Adverse Events by Age Subgroup; Treatment-emergent Adverse Events Related to Treatment by System Organ Class, Preferred Term, Age Subgroup (>= 10%); Adverse Events of Special Interest by System Organ Class, Preferred Term, Age Subgroup
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.