Spinal Muscular Atrophy
Conditions
Keywords
physiotherapy, rehabilitation, SMA, acceptability, feasibility, United Kingdom, physical therapy, prospective, Spinal Muscular Atrophy
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.
Interventions
OTHEROptimized rehabilitation program
Consisting of: goal-oriented rehabilitation, hands on physiotherapy sessions and home use of SMA approved rehabilitation device.
Sponsors
University of Oxford
Oxford Brookes University
Oxford University Hospitals NHS Trust
ACE SMA Charity
Roche Products Limited
Scholar Rock, Inc.
Biohaven Therapeutics Ltd.
Rally Round Rupert Charity
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Intervention model description
All participants will receive the optimized rehabilitation program (intervention). An external control group will be used to determine the potential efficacy.
Eligibility
Sex/Gender
ALL
Age
1 Years to 10 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. | Baseline visit | Number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate. |
| 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. | Month 12 (end of study) | Number of patients who complete the study compared to the number of included patients. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety of an optimized rehabilitation program. Measured as the number of serious adverse events (SAE) compared to the year before the baseline. | From baseline to month 12 (end of study) | Number of Serious Adverse Events (SAEs). |
| 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. | Month 6 and Month 12 (end of study). | Potential effects of the optimized rehabilitation program on patients experience. |
| 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. | Baseline visit: CGI-S. | Potential effects of the optimized rehabilitation program on patients experience. |
Countries
United Kingdom
Contacts
PRINCIPAL_INVESTIGATORCharlotte Lilien
University of Oxford, Department of Paediatrics
Outcome results
None listed