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 have been confirmed through genetic testing to have SMA (Spinal Muscular Atrophy), with a specific change or deletion in the SMN1 gene
- You have two, three, or four copies of the SMN2 gene
- You are between 5 and 20 years old
- If you cannot walk independently: you can sit on your own for at least 5 seconds, have never walked independently, and your best level of movement is sitting or standing with some support — and you score between 10 and 45 on a standard muscle function test (HFMSE)
- If you can walk independently: you are able to walk at least 100 meters on your own and score between 40 and 60 on the HFMSE muscle function test
- In both cases, you must have enough ankle strength to perform a repeated pushing-down foot movement for 2 minutes (confirm with trial site)
- You fall into one of these treatment situations:
- You have been prescribed Evrysdi by your doctor but have not yet started taking it, OR
- You have been taking Spinraza for at least 12 months (with the correct number of doses) or received Zolgensma at least one year ago, OR
- You are switching from Spinraza or Zolgensma to Evrysdi, have been on your current treatment long enough (as above), and have made this decision with your doctor but not yet started Evrysdi, OR
- You have never received any SMA treatment
Who may not be able to join:
- Your blood or urine test results at the start of the study are significantly abnormal, including liver enzyme levels more than twice the normal upper limit or significant protein in the urine (confirm with trial site)
- You are not able to reliably complete the movement tests or the exercise testing inside an MRI scanner
- You are currently taking certain medications or supplements that may affect SMA or muscle energy — such as oral albuterol, hydroxyurea, valproic acid, creatine, or similar supplements — unless you stopped taking them at least 3 months before joining (note: a daily multivitamin, Vitamin D, and occasional inhaled albuterol may be allowed if the dose stays the same)
- You regularly need a breathing machine (non-invasive ventilation) to support your breathing
- You require feeding through a tube (such as a stomach tube) rather than eating by mouth
- You have any metal implants in your body (such as metal spinal rods) that would prevent you from safely having an MRI scan
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: Richard Finkel, MD, St. Jude Children's Research Hospital
Phone: 901-595-1693
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
Feasibility of performing MR functional imaging in SMA patients; Reliability of performing MR functional imaging in SMA patients; Compare skeletal muscle oxidative phosphorylation bioenergetics in patients with SMA types 2 and 3 (phosphocreatine); Compare skeletal muscle oxidative phosphorylation bioenergetics in patients with SMA types 2 and 3 (creatine concentrations); Measure intramuscular fat fraction in major muscle extremity in patients with SMA types 2 and 3; Measure electrophysiological tests of motor neuron function to repetitive nerve stimulation in patients with SMA types 2 and 3
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.