Spinal Muscular AtrophyACE SMANCT06419322

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

A study testing Optimized rehabilitation program in people with Spinal Muscular Atrophy, run by University of Oxford at 1 site in United Kingdom.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 1 year to 10 years
Conditions the study is for: Spinal Muscular Atrophy
What is being tested?: Optimized rehabilitation program
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 14 participants
Where: 1 siteUnited Kingdom
Study dates: 1 Jun 2024to 31 Aug 2026
Phase: Interventional

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 1 year to 10 years
All sexes
No healthy volunteers
Child

Who may be able to join

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.

Who cannot join

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:
No exclusion criteria.

Trial locations and contacts

1 site in 1 country.

United Kingdom1 site

Oxford Brookes University
Oxford, Oxfordshire, OX3 0BP

Other trials in United Kingdom

About this study

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.

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.

Read the 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
Being tested: Optimized rehabilitation program
Keywords: physiotherapy, rehabilitation, SMA, acceptability, feasibility, United Kingdom, physical therapy, prospective, Spinal Muscular Atrophy
Medical subject headings: Muscular Atrophy, Spinal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT06419322
Study group	Role	What they receive
Treated cohort	Other	Not described Receives: Other: Optimized rehabilitation program

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: Month 12 (end of study)

Secondary outcomes (3)

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).
Measured: 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.
Measured: 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.
Measured: Baseline visit: CGI-S.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Jun 202431 Aug 2026

First posted17 May 2024
Study start1 Jun 2024
Primary completion30 Jun 2026
Study completion31 Aug 2026

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
More trials run by University of OxfordThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Other
Allocation: Not applicable
Intervention model: Single group
Model description: All participants will receive the optimized rehabilitation program (intervention). An external control group will be used to determine the potential efficacy.
Masking: None (open label)
Enrolment: 14 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06419322
Sponsor's protocol ID: ACE SMA
Other IDs: PID17165
Acronym: ACE SMA
Status last verified: Sep 2026
First submitted: 2 May 2024
Last updated: 15 Sep 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
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RecruitingPhase 3
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Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
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HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
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