Abstract Details

Title RECLAIM-DCP: A Randomized, Double-Blind, Placebo-Controlled Study of Deutetrabenazine for the Treatment of Dyskinesia in Cerebral Palsy in Children and Adolescents

Topic Movement Disorders

Presentation(s) P4 - Poster Session 4 (5:30 PM-6:30 PM)

Poster/Presentation
Number 10-036

Objective The primary objective of this study is to evaluate the efficacy of deutetrabenazine in the treatment of dyskinetic involuntary movements associated with cerebral palsy.

Background Dyskinesia in cerebral palsy (DCP) represents a hyperkinetic movement disorder associated with static encephalopathy due to a disturbance in the developing fetal or infant brain. There is a marked unmet medical need for the treatment of DCP, but no approved treatment is currently available. Deutetrabenazine is a well-tolerated vesicular monoamine transporter type 2 inhibitor, approved by the FDA for the treatment of chorea associated with Huntington’s disease and tardive dyskinesia in adults. Deutetrabenazine is currently under development for the treatment of tics in pediatric/adolescent patients with Tourette syndrome.

Design/Methods 185 pediatric/adolescent patients with predominantly choreiform dyskinesia are planned to be randomized to deutetrabenazine vs placebo (2:1 ratio). The primary endpoint is change from baseline in Movement Disorder Childhood Rating Scale Index II (MD-CRS II, centrally read, DCP severity). Key secondary endpoints are change from baseline in MD-CRS Index I (MD-CRS I, centrally read, functional activity), Caregiver Global Impression of Improvement (CaGI-I), and the Clinical Global Impression of Improvement (CGI-I) that together with exploratory endpoints will inform on functional and health-economics outcomes and quality of life. Another objective is to evaluate the safety and tolerability of deutetrabenazine in patients with DCP. An interim analysis (IA) will be performed based on centrally read MD-CRS II scores to adapt the study based on observed conditional power.

Results The design of this innovative, double-blind, placebo-controlled trial and its challenges and limitations will be discussed.

Conclusions RECLAIM-DCP (Reduction of Childhood and Adolescent Abnormal Involuntary Movements) is a large placebo-controlled study of deutetrabenazine in pediatric/adolescent patients with DCP that introduces the novel MD-CRS instrument for efficacy assessment and includes an IA for study adaptation.

Authors/Disclosures
Daniel O. Claassen, MD, FAAN (Vanderbilt University Medical Center) PRESENTER	Dr. Claassen has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Alterity. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lundbeck. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Teva. Dr. Claassen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for AskBio. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for University of Michigan. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cognition Therapeutics . Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx. The institution of Dr. Claassen has received research support from NIH. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from HDSA. The institution of Dr. Claassen has received research support from Department of Defense. The institution of Dr. Claassen has received research support from CHDI.
Jonathan W. Mink, MD, PhD, FAAN	The institution of Dr. Mink has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. The institution of Dr. Mink has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurogene. Dr. Mink has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TEVA. Dr. Mink has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. Mink has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. Dr. Mink has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN. The institution of Dr. Mink has received research support from Neurogene. The institution of Dr. Mink has received research support from NIH. Dr. Mink has received publishing royalties from a publication relating to health care. Dr. Mink has received personal compensation in the range of $500-$4,999 for serving as a Member, Study Section with NINDS.
Mered Parnes, MD	Dr. Parnes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Teva Pharmaceuticals. Dr. Parnes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Teva Pharmaceuticals. The institution of Dr. Parnes has received research support from NIH. The institution of Dr. Parnes has received research support from Alexion.
Mark F. Gordon, MD, FAAN (Teva Pharmaceuticals)	Dr. Gordon has received personal compensation for serving as an employee of Teva. Dr. Gordon has stock in Teva.
	No disclosure on file
Maria Wieman, MPH	No disclosure on file
Juha M. Savola, MD, PhD (Spark Therapeutics, Inc)	Dr. Savola has received personal compensation for serving as an employee of Teva Pharmaceuticals. Dr. Savola has received intellectual property interests from a discovery or technology relating to health care.
	No disclosure on file
Joseph Jankovic, MD, FAAN (Baylor College of Medicine)	Dr. Jankovic has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Revance. Dr. Jankovic has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Revance, Allergan. The institution of Dr. Jankovic has received research support from Baylor College of Medicine. Dr. Jankovic has received research support from Abbvie. The institution of Dr. Jankovic has received research support from Abbvie.
Leon S. Dure, MD (Univ of Alabama-Birmingham)	Dr. Dure has nothing to disclose.

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