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

Last updated: January 30, 2025
Sponsor: University of Oxford
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Muscular Dystrophy

Myasthenia Gravis (Chronic Weakness)

Spinal Muscular Atrophy

Treatment

Optimized rehabilitation program

Clinical Study ID

NCT06419322
ACE SMA
PID17165
  • Ages 1-10
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Patient participant Eligibility Criteria:

Inclusion Criteria:

  • Genetically confirmed SMA considered as a non-sitter, sitter or walker

  • Post-symptomatically treated and on stable dose for 12 months with anydisease-modifying market-approved drug

  • Patients from 1-10 years of age at baseline

  • Parent(s)/legal guardian(s) of patients less than 16 years of age must providewritten informed consent prior to the patient's participation in the study

  • Willing and able to comply with all protocol requirements and procedures.

Exclusion

Exclusion Criteria:

The participant may not enter the trial if ANY of the following apply:

  • Any acute or chronic condition which, as assessed by the investigator, significantlyinterferes with the rehabilitation of the patient

  • Need of diurnal and/or invasive ventilation, naps excluded

  • Currently enrolled in a treatment study; or treatment with an experimental therapy

  • Any surgical and/or medical intervention, according to the investigator, 3 monthsbefore baseline and/or during the study participation.

Carer (considered as participant) Eligibility Criteria:

Inclusion Criteria:

  • Willing and able to comply with all protocol requirements and procedures

  • Carer's child has been included in study.

Exclusion Criteria:

• No exclusion criteria.

Study Design

Total Participants: 14
Treatment Group(s): 1
Primary Treatment: Optimized rehabilitation program
Phase:
Study Start date:
June 01, 2024
Estimated Completion Date:
December 31, 2026

Study 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.

Connect with a study center

  • Oxford Brookes University

    Oxford, Oxfordshire OX3 0BP
    United Kingdom

    Site Not Available

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