NCT06419322

Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).

A Monocentric, Prospective and Longitudinal Study Investigating the Acceptability, Feasibility, Safety and Efficacy of an Optimized Rehabilitation Program for Treated Patients With SMA Compared to the Current Rehabilitation Program in United Kingdom: ACE SMA.

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: University of Oxford · Academic / Other
• Sex: All
• Age: 1 Year – 10 Years
• Healthy volunteers: Not accepted

Summary

The goal of this study is to investigate the acceptability, feasibility, safety and efficacy of an optimized rehabilitation program for treated patients with spinal muscular atrophy (SMA) compared to the current rehabilitation program in the United Kingdom. The aim is to provide patients with more hands on physiotherapy and access to rehabilitation devices at home to support parents currently providing rehabilitation on their own.

Detailed description

The main study objective is to investigate whether an optimized rehabilitation program would be accepted and is feasible within the SMA patient community. The optimized program will comprise of goal orientated rehabilitation with more frequent hands on physiotherapy (every 2 weeks) as well as a SMA approved rehabilitation device used at home on a weekly basis, to provide the most appropriate therapy for each individual patient. All study participants will receive the optimized rehabilitation program over 12 months. An external control group will be used to determine the potential efficacy. Research study visits will take place at baseline, month 6 and month 12. At these visits a general physical exam will be undertaken alongside collecting the participant and carer perception, satisfaction and compliancy of the rehabilitation program. Motor function assessments according to age and ambulatory status will be assessed to help evaluate potential therapeutic benefits.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
OTHER	Optimized rehabilitation program	Consisting of: goal-oriented rehabilitation, hands on physiotherapy sessions and home use of SMA approved rehabilitation device.

Timeline

• Start date: 2024-06-01
• Primary completion: 2026-06-01
• Completion: 2026-12-01
• First posted: 2024-05-17
• Last updated: 2025-02-03

Locations

1 site across 1 country: United Kingdom