Clinical Research Alert: Clinical Study in Children with SMA

Researchers at Teachers College, Columbia University (New York) are seeking children living with spinal muscular atrophy (SMA) who are receiving disease-modifying therapy to participate in a pilot study exploring the integration of targeted rehabilitation strategies. This intervention aims to strengthen muscles, enhance neuromuscular coordination, and reduce fatigue, ultimately supporting improved movement and functional abilities in children with SMA.

The study

This study involves an intensive rehabilitation intervention for all participants. Enrolled participants will undergo 15 therapist-supervised, in-person intensive rehabilitation day-camp sessions with a schedule of 6 hours/day, 5 days/week over 3 weeks. They will undergo physical evaluations at three timepoints: baseline (immediately before the 3-week camp), post-intervention (immediately after the 3-week camp), and at a 3-month follow-up. The duration of the study will be about 4 months.

The effect of the invention will be evaluated using a number of tests and assessments including, but not limited to: standardized tests of gross motor, upper limb, and trunk function (HFMSE, RULM, TCMS), endurance and fatigability (ESBBT, 6MWT, 10-Meter Walk, Timed Up and Go, 30-Second Sit-to-Stand), patient-reported outcomes (SMAEFFORT, COPM), and neuromuscular function using surface electromyography (sEMG) to measure muscle fatigue and motor unit recruitment.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:

• Have a confirmed SMA diagnosis
• Be 5–17 years old
• Complete all pre- and post-intervention assessments
• Understand and speak English
• Follow instructions without major cognitive or behavioral issues
• Have received a stable disease-modifying therapy (DMT) dose for ≥6 months
• Be classified as a sitter or walker with supported standing ability (score ≥1 on Item 18 of the HFMSE) without KAFOs, AFOs, or external standing devices

Individuals may not be eligible to participate if they meet the following exclusion criteria:

• Orthopedic surgery within the past year
• New pharmaceutical treatment during the study
• Concurrent participation in another clinical trial

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

To learn more or inquire about participation, visit the sponsor’s trial website or contact the Study Coordinator by e-mail: cpresearch@tc.columbia.edu.