Phase 2 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 2 years of age or older at the time of signing the consent form
- You must have a confirmed genetic and clinical diagnosis of Type 3 Spinal Muscular Atrophy (SMA), caused by a specific gene change on chromosome 5
- Your score on a standard motor skills test (called the HFMSE) must fall between 10 and 54 at the initial check-up
- Female participants who are pregnant, breastfeeding, or could become pregnant, as well as all other participants (male and female), must use effective birth control for 6 months after treatment
- You (or your legal guardian) must be able to understand what the study involves and willingly sign and date a consent form before any study procedures begin
Who may not be able to join:
- You have previously taken part in any gene therapy research trial
- You have a high level of a certain antibody (called AAV9 neutralizing antibody) in your blood, at a level of 1:200 or higher (confirm with trial site)
- You have taken the SMA medication Nusinersen within the last 120 days, or Risdiplam within the last 15 days before treatment
- You currently need a breathing machine or ventilator support for an average of 16 or more hours per day
- Your genetic test shows more than 4 copies of the SMN2 gene
- You require a feeding tube through the nose or stomach to eat
- You have tested positive for HIV, Hepatitis B, Hepatitis C, or syphilis
- You have a known allergy or bad reaction to prednisolone, other steroid medications, or their ingredients
- You have severe joint stiffness or tightening that would prevent you from completing movement tests or receiving the treatment by injection into the spine
- You have other serious health conditions affecting the heart, brain, blood vessels, digestive system, kidneys, hormones, blood, immune system, or nervous system (including conditions like epilepsy, meningitis, seizures, or mental illness)
- You have a previous injury (such as a broken bone) or have had surgery that has not yet fully healed or stabilised
- You have received any vaccine within 2 weeks before treatment
- The trial doctor believes, for any other reason, that the trial would not be suitable for you
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
Phone: +8613501380583
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
Incidence of Treatment-Emergent Adverse Events; Change from baseline on Hammersmith Functional Motor Scale - Expanded (HFMSE) scores at Month 12
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.