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 (NCT02193074) enrolled 121 infants in total — 41 in the control group and 80 in the nusinersen group. The trial was measuring things like whether children showed improvements in physical movement milestones (such as head control, sitting, or rolling), how long it took before children either died or needed permanent breathing support, and changes in muscle function tests. The study was stopped early, with 24 control participants and 65 nusinersen participants recorded as having completed the trial (including those who completed due to early termination). The reported data shows that for the main ("primary") outcome of motor milestone improvement, 0% of children in the control group met the criteria for being a "responder" (meaning they showed meaningful gains in movement categories), compared with 51% of children in the nusinersen group. For the other primary outcome — the proportion of children who died or needed permanent breathing support over time — the reported data shows that at the final measured point, approximately 73.5% of the control group and 44.7% of the nusinersen group had reached that outcome. For the secondary outcomes, 3% of control participants and 71% of nusinersen participants showed a meaningful improvement on a separate muscle movement test (CHOP-INTEND). Regarding permanent ventilation, 68% of the control group and 77% of the nusinersen group did not require it. In a nerve-signal test (CMAP), 5% of the control group and 36% of the nusinersen group met the responder criteria. 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 38705943) ↗
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, Terminated
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 was born after a full-term pregnancy (between 37 and 42 weeks)
- The baby has been officially diagnosed with Spinal Muscular Atrophy (SMA)
- The baby has exactly 2 copies of a specific gene called SMN2
- The baby's weight is at or above the 3rd percentile (a minimum healthy weight range) for their age, based on guidelines used in their country
- The baby is able to take part in and complete all required study procedures
- The family lives within about 9 hours of driving distance from a participating study location, for the entire time the study is running
Who may not be able to join:
- The baby has low blood oxygen levels while awake or asleep (below 96%), without the use of breathing support equipment
- The baby has significant abnormal results in blood tests or heart readings at the initial screening visit, which the study doctor feels would make participation unsafe (confirm with trial site)
- The baby's parent or legal guardian is not willing to follow standard care guidelines, which include keeping up with vaccinations, a preventative treatment for a common respiratory virus (if available), and providing the necessary nutritional and breathing support throughout the study
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
Percentage of Motor Milestones Responders; Time to Death or Permanent Ventilation
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.