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 (NCT02594124) enrolled a total of 292 participants across nine groups. The participants had either infantile-onset or later-onset Spinal Muscular Atrophy (SMA) and came from several earlier related studies. The groups included people who had previously received a placebo-like "sham" procedure, people who had previously received the study drug nusinersen (also referred to as ISIS 396443), and people entering from other studies. The trial was measuring a range of monitored observations — including unwanted medical events (called adverse events), abnormalities in vital signs (such as blood pressure and heart rate), body weight changes, neurological (nerve and muscle) examination findings, and blood test results. The reported data shows that across the nine groups, the number of participants who experienced any treatment-emergent adverse event (that is, a new or worsening medical event that appeared after dosing began) ranged from 8 out of 8 participants in one group up to 80 out of 83 participants in another group. Serious adverse events — defined as events involving death, being life-threatening, requiring hospitalisation, or causing significant long-term disability — were also counted, though the full breakdown across all nine groups was not completely reported in the data provided. For the other monitored areas: abnormalities in vital signs recorded as adverse events were reported in very small numbers across most groups (ranging from 0 to 6 participants per group); weight changes of 7% or more were reported in 0 to 3 participants per group; neurological examination abnormalities recorded as adverse events were reported in 0 to 2 participants per group; laboratory test abnormalities were reported in 0 to 6 participants per group; and abnormalities in blood clotting measures were reported in 0 to 2 participants per group. 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, 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:
- A parent or guardian has given their signed permission for the child to take part, and the participant themselves has also agreed to join if appropriate for their age.
- The participant has recently finished a related ("index") study — either by completing it normally or because the study was ended early by the company — within the past 16 weeks.
Who may not be able to join:
- The participant has a medical condition, or a condition that has gotten worse, that the doctor running the trial believes would make it unsafe or unsuitable for them to take part.
- The participant had significant abnormal results on blood tests, other lab tests, or a heart tracing (ECG) at the screening visit that the doctor believes would make participation unsafe.
- The participant's parent or legal guardian is not willing or able to follow standard care guidelines — including keeping up with vaccinations and a specific type of infection prevention treatment (if available) — or to provide the necessary nutritional and breathing support throughout the study.
- The participant has been treated with another experimental treatment, a biological medicine, or an investigational medical device within the past month before screening, or within a certain timeframe based on how long the previous study drug stays in the body — whichever is longer (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
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs); Number of Participants With Vital Sign Abnormalities Reported as AEs; Number of Participants With Weight Abnormalities Reported as AEs; Number of Participants With Neurological Abnormalities Reported as AEs; Number of Participants With Laboratory Abnormalities Reported as AEs; Number of Participants With Coagulation Parameters Reported as AEs; Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results; Number of Participants Taking Any Concomitant Medi...
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.