Abstract Details

Title JASPER Early Intervention in TSC (JETS): Methods to enhance study participation and scalability in rare disorders

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-036

Objective To describe the design of a remote behavioral intervention clinical trial in infants with Tuberous Sclerosis Complex (TSC)

Background

Up to 80% of children with TSC suffer from cognitive impairment, and rates of autism approach 60% (Curatolo, 2010; Franz, 2010; Bolton, 2015; Jeste, 2014; Jeste, 2016). In a longitudinal study of development in TSC, we found that infants demonstrate delays in nonverbal cognition and social communication skills as early as 9 months of age, and these delays predict an autism diagnosis (Jeste, 2014; McDonald, 2017). These early delays necessitate the implementation of early intervention that targets social communication skills in TSC. However, implementing studies of behavioral intervention in a rare disorder, with families dispersed geographically, limits the feasibility of clinical trials.

Design/Methods In 2017, we began the first randomized, double blind controlled trial of JASPER, a well validated and rigorously studied social communication intervention for autism, in infants with TSC ages 12-36 months (JETS: NICHD RO1 HD090138-01A1). By the end of year one, despite investing considerable resources into recruitment, we enrolled only three participants. Parent interviews revealed that the greatest obstacle to participation was the weekly travel to the sites (UCLA and Boston Children’s Hospital) for the intervention. In response, we substantially revised the study design, with intervention delivery, assessments and parent communication all delivered remotely.

Results Since this modification was made 7 months ago, enrollment has increased from 3 to 27 participants. The average distance from the testing sites of participants enrolled is 320 miles, with states represented including Massachusetts, California, Maine, New York, Maryland, Arkansas, North Carolina.

Conclusions

The tremendous success in enrolling participants through these modifications reinforces the need for innovative research delivery methods in rare populations. In this presentation, we will describe not only the methods, but also lessons learned that can help guide future clinical trials across neurodevelopmental disorders.

Authors/Disclosures
Shafali Jeste, MD, FAAN (Children's Hospital of Los Angeles) PRESENTER	Dr. Jeste has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche Pharmaceuticals. Dr. Jeste has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche Pharmaceuticals. Dr. Jeste has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis Pharmaceuticals. Dr. Jeste has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN Continuum. The institution of Dr. Jeste has received research support from NIH. The institution of Dr. Jeste has received research support from DoD. The institution of Dr. Jeste has received research support from Dup15q Alliance.
	No disclosure on file
	No disclosure on file
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