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 (NCT03505099) involved 29 babies in total, split into two groups based on their genetic profile. Fourteen babies were in Cohort 1 (carrying two copies of a gene called SMN2) and fifteen were in Cohort 2 (carrying three copies of SMN2). All 29 participants completed the study. The trial was measuring whether babies reached certain physical milestones — such as sitting, standing, and walking — that are not typically expected in children with their condition. The reported data shows that for Cohort 1, all 14 participants were reported to have achieved sitting alone for at least 30 seconds without support (the primary goal for that group). For Cohort 2, all 15 participants were reported to have achieved standing alone for at least 3 seconds without support (the primary goal for that group). Turning to the secondary goals, the reported data shows that all 14 babies in Cohort 1 were alive, not on permanent breathing support, and still in the study at 14 months of age. Thirteen out of 14 babies in Cohort 1 were reported to have maintained a healthy weight without needing a feeding tube up to 18 months of age. For Cohort 2, 14 out of 15 participants were reported to have achieved walking alone, defined as taking at least 5 independent steps. 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 35715567) ↗
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:
- Your baby must be 6 weeks old or younger (no more than 42 days old) at the time they receive the study treatment
- Your baby must be able to swallow thin liquids safely, as confirmed by a formal swallowing test
- Your baby must have a nerve signal strength result (called a CMAP) of 2mV or higher, measured at the start of the study
- Your baby must have been born between 35 and 42 weeks of pregnancy
- A parent or legal guardian must be willing and able to sign the consent forms and follow all study visits and procedures
- Your baby must have been diagnosed with pre-symptomatic (no symptoms yet) SMA Type 1, with 2 copies of a gene called SMN2 (confirm with trial site)
- Your baby must have been diagnosed with pre-symptomatic (no symptoms yet) SMA Type 2, with 3 copies of the SMN2 gene (confirm with trial site)
Who may not be able to join:
- Babies who weigh less than 2 kg (about 4.4 lbs) at the screening visit
- Babies whose blood oxygen levels are too low without any breathing support (below 96% when awake or asleep, or below 92% for those living at high altitudes above 1,000 metres)
- Babies who are already showing signs or symptoms that the doctor believes are strongly linked to SMA
- Babies who have had a tracheostomy (a breathing tube in the throat) or who have used or needed any kind of breathing support device at any point before or during screening
- Babies who show signs of difficulty swallowing or who cannot tolerate thin liquids based on a swallowing test, or who are being fed by any method other than by mouth
- Babies with significantly abnormal blood test results related to liver function, kidney function, or blood counts before treatment (confirm with trial site for specific values)
- Babies who have already received any treatment for SMA, including nusinersen, any gene therapy, antisense oligonucleotide treatment, or cell transplantation
- Babies whose weight is very low for their age, falling below the 3rd percentile on World Health Organization growth charts
- Babies whose biological mother has an active HIV infection, or tests positive for hepatitis B or C
- Babies whose biological mother has a suspected or confirmed Zika virus infection (the baby may need to be tested for Zika before being allowed to join)
- Babies who have had a serious illness (not related to breathing) needing treatment or hospitalisation within the 2 weeks before screening
- Babies who have had a respiratory (breathing or lung) infection needing medical attention within 4 weeks before receiving the study treatment
- Babies who have had a severe non-respiratory infection within 4 weeks before treatment, or who have other serious conditions such as major kidney or liver problems, a known seizure disorder, diabetes, a condition called idiopathic hypocalciuria, or heart muscle disease
- Babies who are known to be allergic to prednisolone (a steroid medication) or similar steroid medicines
- Babies who have had, or are expected to have, a major surgery (such as scoliosis repair) during the study period
- Babies currently taking certain medications, including drugs for muscle or nerve conditions, diabetes medicines, or immune-suppressing treatments within 4 weeks before receiving the study treatment
- Babies with a high level of antibodies against a component of the gene therapy (called anti-AAV9 antibodies, above a level of 1:50) — though re-testing may be allowed within the screening period (confirm with trial site)
- Babies whose biological mother (if she is involved in the child's care) refuses to be tested for anti-AAV9 antibodies before treatment
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
Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds; Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds
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.