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 are between 5 and 17 years old (inclusive), a parent or guardian can sign the consent form on your behalf, and you are able to follow the study requirements
- You have been officially diagnosed with 5q Spinal Muscular Atrophy (SMA) confirmed through genetic testing
- You are currently on a stable treatment plan using nusinersen or risdiplam, and/or you have previously received onasemnogene abeparvovec treatment
- You are able to walk at least 50 meters (about 164 feet) without any walking aids during a walking test at the start of the study
Who may not be able to join:
- You have another health condition that could affect the study results or put you at risk, as decided by the study doctor
- You have had a major operation (other than spinal fusion surgery) in the last 3 months, or you plan to have major surgery during the study
- You have received any anti-myostatin therapies (a specific type of muscle-related treatment) in the past 6 months
- You have a severe curve of the spine (greater than 40 degrees) and/or significant joint tightness, OR you have had spinal fusion surgery within the last 6 months, or plan to have it during the study
- You need a breathing machine or ventilation support while you are awake during the day (note: using breathing support only at night or during naps is acceptable)
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: 857-350-4834
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 AEs; Incidence of SAEs; Change in RHS total score from baseline to week 24 of the double blinded treatment period (DBTP)
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.