Phase 3 Spinal Muscular Atrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You are between 15 and 60 years old (inclusive) at the time you give your consent to participate
• You have a confirmed genetic diagnosis of 5q Spinal Muscular Atrophy (SMA), shown through genetic testing
• You have physical signs and symptoms that match a diagnosis of SMA
• Your genetic test shows you have at least one copy of a gene called SMN2
• Your score on a specific physical ability assessment (a movement test called the HFMSE) falls between 10 and 54 out of a possible higher score (confirm with trial site)
• You are able to sit up without any support for at least 10 seconds
• You have never been treated with a type of drug called a myostatin inhibitor, and you are willing to avoid that type of drug for the whole study
• If you can walk independently, you must be able to walk at least 10 meters on your own and be willing to complete a 6-minute walk test at your first screening visit
• If joining the group with no prior SMA treatment: you have never received any approved or experimental drug specifically for treating SMA
• If joining the group currently on risdiplam: you are currently taking risdiplam (5 mg once daily) and have been doing so for at least 6 months before your screening visit
• If joining the risdiplam group: you are willing to stop taking risdiplam for the entire duration of the study, with your last dose taken the day before your first dose of the study drug
• If joining the risdiplam group: you have not previously been treated with any other approved or experimental SMA therapies apart from risdiplam

Who may not be able to join:

• You need breathing support (such as a ventilator or breathing machine) for more than 6 hours in any 24-hour period, not counting nighttime breathing support through a face mask (CPAP/BiPAP)
• You rely on a feeding tube through your stomach for most of your nutrition, as judged by the trial doctor
• You have a history of brain or spinal cord conditions, or other issues (such as severe spine curvature) that could interfere with a procedure involving the lower back (lumbar puncture), or that could affect how the study assessments work — this includes a history of fluid buildup around the brain (hydrocephalus) or a drainage shunt (confirm with trial site)
• You were hospitalised in the past 2 months for surgery, a breathing problem, or nutritional support, or you plan to have any scheduled (non-emergency) procedure or surgery at any point after signing your consent form through to the end of the study — note that if you previously had surgery for spine curvature, it must have been done at least 1 year before your screening visit
• You currently have an active health issue, such as an infection or a recent bone fracture, that the trial doctor believes makes it unsafe for you to participate
• You are currently enrolled in, or planning to join, another clinical trial involving an experimental or investigational treatment within 90 days (or longer, depending on the treatment) before your screening visit — this also includes treatments such as spinal cord stimulation

Important: Always verify eligibility with the trial site directly before applying.