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

NCT ID: NCT06419322

Last Updated: 2025-02-03

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-06-01
• Study Completion Date: 2026-12-31

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

Treated cohort

Group Type: OTHER

Optimized rehabilitation program

Intervention Type: OTHER

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

Interventions

Optimized rehabilitation program

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

Intervention Type: OTHER

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.

• Willing and able to comply with all protocol requirements and procedures
• Carer's child has been included in study.

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:

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Oxford

OTHER

Sponsor Role: lead

Oxford Brookes University

OTHER

Sponsor Role: collaborator

Oxford University Hospitals NHS Trust

OTHER

Sponsor Role: collaborator

ACE SMA Charity

UNKNOWN

Sponsor Role: collaborator

Roche Products Limited

UNKNOWN

Sponsor Role: collaborator

Scholar Rock, Inc.

INDUSTRY

Sponsor Role: collaborator

Biohaven Therapeutics Ltd.

INDUSTRY

Sponsor Role: collaborator

Rally Round Rupert Charity

UNKNOWN

Sponsor Role: collaborator

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Charlotte Lilien

Role: PRINCIPAL_INVESTIGATOR

University of Oxford, Department of Paediatrics

Locations

Oxford Brookes University

Oxford, Oxfordshire, United Kingdom

Countries

United Kingdom

Other Identifiers

PID17165

Identifier Type: OTHER

Identifier Source: secondary_id

ACE SMA

Identifier Type: -

Identifier Source: org_study_id