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NCT Number: NCT06419322

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

1 year–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Oxford Brookes University

Oxford, Oxfordshire, OX3 0BP, United Kingdom

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Optimized rehabilitation program

Other

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

Primary outcomes

  1. 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.

    Time frame: 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.

  2. 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.

    Time frame: Month 12 (end of study)

    Number of patients who complete the study compared to the number of included patients.

Secondary outcomes

  1. Safety of an optimized rehabilitation program. Measured as the number of serious adverse events (SAE) compared to the year before the baseline.

    Time frame: From baseline to month 12 (end of study)

    Number of Serious Adverse Events (SAEs).

  2. 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.

    Time frame: Month 6 and Month 12 (end of study).

    Potential effects of the optimized rehabilitation program on patients experience.

  3. 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.

    Time frame: Baseline visit: CGI-S.

    Potential effects of the optimized rehabilitation program on patients experience.

Other outcomes

  1. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

  2. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

  3. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

  4. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

  5. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

  6. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

  7. 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.

    Time frame: Baseline, Month 6, Month 12 (end of study).

    Evaluation potential therapeutic benefits through assessment of motor functional scales.

Sponsors and collaborators

Lead sponsor

University of Oxford

Other

Collaborators

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

Registry information

Official study 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.

Acronym: ACE SMA

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 17, 2024
Registry last updated
Feb 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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