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.