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 (NCT04851873) enrolled 24 children in total, divided into three groups based on body weight: 7 children weighing 8.5–13 kg, 8 children weighing more than 13–17 kg, and 9 children weighing more than 17–21 kg. All 24 children completed the study. Each child received a single dose of OAV101 (a gene therapy), and the trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) and changes in vital signs such as blood pressure and breathing rate across the different weight groups. The reported data shows that all 24 participants across the three weight groups experienced at least one adverse event (sometimes called an "AE" — an unwanted medical occurrence during the study). When it came to serious adverse events (more significant unwanted medical occurrences), 8 out of 24 participants were reported to have experienced one — 1 in the lightest group, 4 in the middle group, and 3 in the heaviest group. The trial also tracked a specific list of pre-identified medical concerns, including effects on the liver, low platelet counts, heart-related events, nerve-related effects, and a condition affecting small blood vessels. The reported data shows 20 out of 24 participants experienced at least one of these pre-identified concerns, and 3 out of 24 experienced what were categorised as serious versions of these specific concerns. For blood pressure and breathing rate, the reported changes from the start of the study to later measurements were generally small and varied across the groups; the exact figures differed by weight group and time point, and no single consistent direction of change was seen across all groups. 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 39804575) ↗
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) confirmed by a genetic test showing specific changes in the SMN1 gene, along with at least one copy of the SMN2 gene
- You weigh between 8.5 kg (about 18.7 lbs) and 21 kg (about 46.3 lbs) at the time of the screening visit
- You have either never received treatment for SMA, or you have stopped taking an approved SMA medication
Who may not be able to join:
- You have previously received OAV101 or any gene therapy using AAV9 (a specific type of gene delivery method)
- Your body weight is considered very low for your height and age (below the 3rd percentile on a BMI chart)
- You have a history of, or current signs of, accidentally inhaling food or liquid into the lungs (aspiration pneumonia)
- You have high levels of antibodies in your blood that react against AAV9 (confirm with trial site)
- You have previously had gene therapy, a bone marrow/stem cell transplant, or an organ transplant
- You are unable to take steroid medications (corticosteroids)
- You are currently taking medications that suppress your immune system
- You need a breathing machine (ventilator), a breathing tube in your throat (tracheostomy), or breathing support while awake
- You have received any vaccines within 2 weeks before the trial treatment infusion
- You have low oxygen levels in your blood while awake
- You have liver problems or reduced liver function
- You currently have, or are suspected to have, an active infection
- If you have previously taken an SMA medication, a specific waiting period must have passed before joining (confirm with trial site)
- Both of your biological parents are closely related to each other (such as cousins) (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
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
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket; Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket; Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure; Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg); Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg);...
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.