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

NCT06419322

Active Not RecruitingNA

Sponsor: University of Oxford

Conditions: Spinal Muscular Atrophy

Interventions: Optimized rehabilitation program

Countries: United Kingdom

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 overview

Sex: ALL

Age: 1 Year to 10 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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 any disease-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 provide written informed consent prior to the patient's participation in the study
* Willing and able to comply with all protocol requirements and procedures.

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, significantly interferes 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 months before 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.

Locations (1)

Oxford, Oxfordshire, United Kingdom