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Name: 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.
Creator: University of Oxford
Published: 2024-05-17
License: https://www.clinicaltrials.gov/about-site/terms-conditions#availability

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	Clinical Trial NCT06419322 (ACE SMA) for Spinal Muscular Atrophy is active, not recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here.

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Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA). (ACE SMA) 14 At-Home

Active, not recruiting

Clinical Trial NCT06419322 (ACE SMA) is an interventional study for Spinal Muscular Atrophy that is active, not recruiting. It started on 1 June 2024 with plans to enroll 14 participants. Led by University of Oxford, it is expected to complete by 1 December 2026. The latest data from ClinicalTrials.gov was last updated on 3 February 2025.

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...Show More

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 individ...

Official Title

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.

Conditions

Spinal Muscular Atrophy

Other Study IDs

ACE SMA
PID17165 (Other Identifier) (University Oxford Hospitals NHS Foundation Trust)

NCT ID Number

NCT06419322

Start Date (Actual)

2024-06-01

Last Update Posted

2025-02-03

Completion Date (Estimated)

2026-12

Enrollment (Estimated)

Study Type

Interventional

PHASE

N/A

Status

Active, not recruiting

Keywords

physiotherapy
rehabilitation
SMA
acceptability
feasibility
United Kingdom
physical therapy
prospective
Spinal Muscular Atrophy

Primary Purpose

Other

Design Allocation

N/A

Interventional Model

Single Group

Masking

None (Open Label)

Arms / Interventions

Participant Group/Arm	Intervention/Treatment
OtherTreated cohort	Optimized rehabilitation program Consisting of: goal-oriented rehabilitation, hands on physiotherapy sessions and home use of SMA approved rehabilitation device.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	Month 12 (end of study)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	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.	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.	Baseline visit: CGI-S.

Participation Assistant

Eligibility Criteria

Eligible Ages

Child

Minimum Age

1 Year

Eligible Sexes

All

Patient participant Eligibility 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.

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.

University of Oxford

🎓Oxfo...

No contact data.

1 Study Locations in 1 Countries

United Kingdom

Oxfordshire

Oxford Brookes University, Oxford, Oxfordshire, OX3 0BP, United Kingdom