Phase 4 Spinal Muscular Atrophy Trial, Recruiting
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 must be under 2 years old at the time of joining the study
- Your child must have a confirmed diagnosis of spinal muscular atrophy (SMA) caused by a specific genetic change affecting the SMN1 gene
- Your child must have exactly two copies of a related gene called SMN2, confirmed by a lab test
- Your child must have already received a gene therapy treatment called onasemnogene abeparvovec (also known as Zolgensma), either before or after symptoms appeared
- Your child must have received that gene therapy at least 13 weeks before joining this study
- If your child was given a medication called risdiplam before the gene therapy, it must have been taken for no more than 3 weeks and stopped at least 1 day before the gene therapy was given
- In the doctor's opinion, your child's abilities (such as swallowing, breathing, or movement) must have stopped improving or gotten worse after gene therapy, based on at least two recorded check-ups over a period of 26 weeks or less
Who may not be able to join:
- Your child is currently in, or has previously been part of, another research study before starting treatment in this trial
- Your child has unresolved abnormal lab results related to the gene therapy medication
- Your child has received a different type of SMA treatment called an antisense oligonucleotide (for example, nusinersen/Spinraza) at any point
- Your child has ever used a type of drug called an anti-myostatin agent
- Your child requires a breathing machine connected through a surgical airway (invasive ventilation or tracheostomy)
- Your child has a feeding tube and scores zero on a specific swallowing assessment (confirm with trial site)
- Your child has been hospitalised for a breathing-related problem in the last 2 months, or has a planned hospitalisation at the time of screening
- Your child has had a serious illness requiring hospitalisation within the past month, or any illness with a fever within the week before screening or before receiving the first dose
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: Clinical Trials, Hoffmann-La Roche
Phone: 888-662-6728 (U.S. Only)
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
Primary endpoints
Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment
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.