Related Experiment Video
Updated: May 29, 2025
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Feasibility, Acceptability, and Reliability of Remote Motor Assessment in Children With Canavan Disease
Michael Kiefer1, Anzalee Khan2, Beth Leiro3
1PhD in Rehabilitation Sciences Program, School of Health and Rehabilitation Sciences, MGH Institute of Health Professions, Boston, Massachusetts; Department of Physical Therapy, Virginia Commonwealth University, Richmond, Virginia; BridgeBio Gene Therapy LLC, Palo Alto, California.
Insights
Remote motor assessments are feasible and reliable for children with Canavan disease. This study validates a phased approach for remote pediatric rare disease clinical trials, enhancing accessibility and data quality.
Area of Science:
- Neurology
- Pediatrics
- Clinical Trial Methodology
Background:
- Decentralized clinical trials offer benefits like reduced travel and improved access.
- Knowledge gaps exist in remote motor assessment protocols for pediatric rare diseases.
- This study details a method for transitioning Canavan disease motor assessments from in-person to remote.
Purpose of the Study:
- To develop and evaluate a rigorous method for remote motor assessment in children with Canavan disease.
- To assess the feasibility, acceptability, and reliability of remote motor assessments.
- To inform the implementation of remote protocols in pediatric rare disease research.
Main Methods:
- A two-phase study involving seven children with Canavan disease and their caregivers.
- Caregivers were trained to administer motor assessments remotely.
- Evaluated Gross Motor Function Measure-88, Hammersmith Infant Neurologic Exam Motor Milestones Part-2, Alberta Infant Motor Scale, and Infant Motor Profile.
Main Results:
- Phase 1: 95% of motor assessments were completed, demonstrating feasibility.
- Caregiver acceptability was high (86% support for remote protocols).
- Phase 2: Good to excellent inter-rater reliability (15/20) and intra-rater reliability (18/20) were achieved.
Conclusions:
- A rigorous, multiphased approach is crucial for developing remote motor assessment protocols in pediatric rare disease studies.
- Remote motor assessment is feasible, acceptable, and reliable for children with Canavan disease.
- Findings support the broader application of remote assessments in rare pediatric conditions.
Background:
Decentralized clinical trials have many advantages including reducing travel burden, improving access, and reducing costs. However, there is limited knowledge to inform the development and implementation of remote motor assessment protocols in rare pediatric diseases. This study describes a rigorous method for transitioning from in-person to remote motor assessment of children with Canavan disease.
Methods:
Seven children with Canavan disease (mean age 2.5 years; S.D. = 1.0) and their caregivers completed four remote visits during a two-phase study. Clinical raters were trained to coach caregivers using remote administration protocols for key motor assessment tools. The feasibility, acceptability, and reliability of remote administration of the Gross Motor Function Measure-88, Hammersmith Infant Neurologic Exam Motor Milestones Part-2, Alberta Infant Motor Scale, and Infant Motor Profile were evaluated.
Results:
In phase 1, remote assessment was feasible, with 95% (61 of 64) of motor assessments completed. Caregiver acceptability was high, with 86% of caregiver responses (n = 95 of 111) supporting the remote administration protocol. In phase 2, inter-rater reliability (intraclass correlation coefficient or Fleiss kappa >0.75) was good to excellent in 15 of 20 final visit assessments. Intra-rater reliability was good to excellent in 18 of 20 pairs of assessments by visits 3 and 4.
Conclusions:
This study highlights the value and importance of a rigorous multiphased approach to the development and implementation of remote motor assessment protocols for pediatric rare disease studies. Findings from this study support the feasibility, acceptability, and reliability of remote motor assessment in children with Canavan disease.
