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

N/ACompletedNCT06419322

University of Oxford14 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Spinal Muscular Atrophy

Interventions

Optimized rehabilitation programOTHER

Consisting of: goal-oriented rehabilitation, hands on physiotherapy sessions and home use of SMA approved rehabilitation device.

Eligibility

Sex: ALLMin age: 1 YearMax age: 10 Years

Medical Language ↔ Plain English

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 Brookes University

Oxford, Oxfordshire, United Kingdom

Outcomes

Primary Outcomes

Acceptability of an optimized rehabilitation program. Measured as number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate. Endpoint: 70% of acceptability.

Number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate.

Time frame: Baseline visit

Feasibility of an optimized rehabilitation program. Measured as the number of patients who complete the study compared to the number of included patients. Endpoint: 60% of feasibility.

Number of patients who complete the study compared to the number of included patients.

Time frame: Month 12 (end of study)

Secondary Outcomes

Safety of an optimized rehabilitation program. Measured as the number of serious adverse events (SAE) compared to the year before the baseline.

Number of Serious Adverse Events (SAEs).

Time frame: From baseline to month 12 (end of study)

Carer perception and satification measured through the Clinical Global Impression scale - Improvement (CGI I). Comparison of CGI-S score with CGI-I score of the treated group.

Potential effects of the optimized rehabilitation program on patients experience.

Time frame: Month 6 and Month 12 (end of study).

Carer perception and satification measured through the Clinical Global Impression scale - Severity (CGI-S). Comparison of CGI-S score with CGI-I score of the treated group.

Potential effects of the optimized rehabilitation program on patients experience.

Time frame: Baseline visit: CGI-S.

Data from ClinicalTrials.gov

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