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

Spinal Muscular Atrophy Clinical Trial

— ACE SMA  

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06419322
• Other study ID #: ACE SMA
• Secondary ID: PID17165
• Status: Recruiting
• Phase: N/A
• Start date: June 2024
• Est. completion date: December 2026

Study information

• Verified date: May 2024
• Source: University of Oxford
• Contact: Serena Hill
• Phone: 01865618799
• Email: ace.sma[@]paediatrics.ox.ac.uk
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 14
• Est. completion date: December 2026
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 10 Years

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

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

Locations

Country	Name	City	State
United Kingdom	Oxford Brookes University	Oxford	Oxfordshire

Sponsors (7)

Lead Sponsor	Collaborator
University of Oxford	ACE SMA Charity, Biohaven Therapeutics Ltd., Oxford Brookes University, Oxford University Hospitals NHS Trust, Roche Products Limited, Scholar Rock, Inc.

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): maximum score is 64 points. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Other	The Hammersmith Infant Neurological Examination section 2 (HINE-2): maximum score is 26 points. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Other	Revised Hammersmith Scale (RHS). The maximum score for RHS is 69 points. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Other	Hammersmith Functional Motor Scale Expanded (HFMSE) combined assessment. The maximum score is 66 points. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Other	World Health organization (WHO) developmental milestones: the final result is the highest achievable motor milestone. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Other	Revised upper limb module (RULM): from 30 months of age: maximum score is 37 points. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Other	6 Minute Walk test (6MWT): from 4 years of age. Measuring a change in functional total score between the treated study cohort and a control dataset.	Evaluation potential therapeutic benefits through assessment of motor functional scales.	Baseline, Month 6, Month 12 (end of study).
Primary	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
Primary	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	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)
Secondary	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).
Secondary	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.