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 (NCT02386553) enrolled 25 infants who had been diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms — meaning they were identified through genetic testing rather than because they were already unwell. The babies were divided into two groups based on how many copies they had of a gene called SMN2 (15 babies with 2 copies, and 10 with 3 copies), as this gene affects how the condition may develop. The trial was testing a medicine called ISIS 396443 (also known as nusinersen) and was primarily measuring the time until a child either died or needed significant breathing assistance. Secondary measures included whether children developed signs of SMA, whether they were alive at the end of the study, and whether they reached normal movement milestones such as sitting, standing, and walking. The reported data shows that the primary outcome — time to death or need for breathing support — was listed as "NA" (not available/not reported) in the submitted results, so a specific figure for this measure cannot be described. For the secondary outcomes, the reported data shows that 100% of participants in both groups were alive at the time of reporting. Regarding whether children developed clinically recognised signs of SMA, the reported proportion was 0.67 (meaning about 67 in every 100) in the 2-copy group and 0.20 (20 in every 100) in the 3-copy group. For motor development milestones assessed using standard WHO movement checks (such as sitting without support or walking alone), the reported data shows that between 87% and 100% of children in the 2-copy group, and 100% of children in the 3-copy group, reached each of the six milestones. A muscle strength and function score (rated 0–64, where higher means better) showed a reported change from a starting score of around 47 in the 2-copy group and around 52 in the 3-copy group, with one additional figure of 29 reported, though the data as submitted did not make clear which time point this last figure relates to. 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 37409780) ↗
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 2 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:
- The baby must be 6 weeks old or younger at the time of the first dose.
- The baby must have a confirmed genetic test showing they have the type of SMA caused by a change or deletion on chromosome 5q.
- The baby must have a genetic test confirming they have 2 or 3 copies of a specific gene called SMN2.
- The baby must have a nerve signal reading (a test measuring electrical activity in the arm muscles) of at least 1 mV at the start of the study.
- For single births, the baby must have been born between 37 and 42 weeks of pregnancy; for twins, between 34 and 42 weeks of pregnancy.
- The baby must meet additional study-related requirements (confirm with trial site).
Who may not be able to join:
- Babies who have low oxygen levels in their blood (below 96%) while awake or asleep, without any extra oxygen or breathing support.
- Babies who are already showing clear signs or symptoms of SMA at the screening visit or just before the first dose, as judged by the study doctor.
- Babies who have significant abnormalities in their blood test results at the start of the study.
- Babies who have previously received any experimental drug, biological treatment, or device for SMA, or who have had gene therapy, a specific type of drug treatment called antisense oligonucleotide (ASO) therapy, or a cell transplant at any point.
- Babies who meet additional study-related exclusion requirements (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: Medical Director, Biogen
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
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Time to Death or Respiratory Intervention
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.