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

NCT06419322 | Active Not Recruiting

• 2 other identifiers: ACE SMAPID17165
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

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.

Conditions

Spinal Muscular Atrophy

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• 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)

• 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).

• 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.

Other Outcomes (7)

• 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.

  Baseline, Month 6, Month 12 (end of study).

• 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.

  Baseline, Month 6, Month 12 (end of study).

• 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.

  Baseline, Month 6, Month 12 (end of study).

Study Arms (1)

Treated cohort

OTHER

Other: Optimized rehabilitation program

Interventions

Optimized rehabilitation program OTHER

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

Treated cohort

Eligibility Criteria

• Age: 1 Year - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• University of Oxford: lead
• Oxford Brookes University: collaborator
• Oxford University Hospitals NHS Trust: collaborator
• ACE SMA Charity: collaborator
• Roche Products Limited: collaborator
• Scholar Rock, Inc.: collaborator
• Biohaven Therapeutics Ltd.: collaborator
• Rally Round Rupert Charity: collaborator

Study Sites (1)

Oxford Brookes University

Oxford, Oxfordshire, OX3 0BP, United Kingdom

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Charlotte Lilien

  University of Oxford, Department of Paediatrics

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: All participants will receive the optimized rehabilitation program (intervention). An external control group will be used to determine the potential efficacy.

Study Record Dates

• First Submitted: May 2, 2024
• First Posted: May 17, 2024
• Study Start: June 1, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: February 3, 2025

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)