Abstract Details

Title Interim Analysis of an Open-Label, Multi-Center Study of Ecopipam, a Dopamine D1 Receptor Antagonist, for Tic Reduction in Adults with Tourette Syndrome

Topic Movement Disorders

Presentation(s) P02 - (-)

Poster/Presentation
Number 058

Objective

Background BACKGROUND: In Tourette Syndrome, severe tics are treated with antipsychotic drugs which block dopamine D2-receptors. Suboptimal efficacy and side effects are common, prompting research into other treatment approaches.

Design/Methods DESIGN/METHODS: This multicenter, nonrandomized study in adults with Tourette Syndrome assessed open-label treatment with 50 mg ecopipam daily (weeks 1-2) and then 100 mg (weeks 3-8). The primary efficacy endpoint was the change in Yale Global Tic Severity Scale (YGTSS) scores. Analysis was intention to treat. Co-occurring attention deficit/hyperactivity disorder, obsessive/compulsive, and depression symptoms and premonitory urges were also rated. Weight, serum metabolic studies, and side effects were monitored. This abstract reports a pre-specified interim analysis.

Results RESULTS: 15 adults (12 male; 12 Caucasian, 2 African American, 1 Asian), mean age 34 years (range 19-60) enrolled and 12 completed the study. Mean YGTSS Total Tic score was 30.9 (SD 8.2) at baseline and 25.4 (9.5) at 8 weeks (two-tailed paired t14 = 3.9; p = .002). Mean Total YGTSS (with impairment score) was 58.7 (17.2) at baseline and 48.1 (22.5) at final visit (t14 = 2.4; p = .03). Three patients each reported nausea, fatigue, and sedation and two each reported restlessness and sleeplessness. There was no weight gain over the course of treatment (mean change -0.55 kg; p = .054). At the trend level, ADHD scores appeared to improve (p = .093). There was no significant change in premonitory urges (p = .44), obsessive compulsive (p = .46), or depressive (p = .31) symptoms.

Conclusions CONCLUSIONS: In this open-label study, oral ecopipam appeared to reduce tic severity without causing weight gain. To confirm treatment safety and efficacy, further double blind, placebo-controlled randomized trials in children and in adults with TS are warranted.

Authors/Disclosures
Donald Gilbert, MD, FAAN (Cincinnati Children's Hospital Med. Ctr.) PRESENTER	Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Illumina. Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Emalex Biosciences. The institution of Dr. Gilbert has received research support from NIMH. The institution of Dr. Gilbert has received research support from Emalex Biosciences. The institution of Dr. Gilbert has received research support from PTC Therapeutics. The institution of Dr. Gilbert has received research support from Department of Defense. The institution of Dr. Gilbert has received research support from Quince Therapeutics. Dr. Gilbert has received publishing royalties from a publication relating to health care. Dr. Gilbert has received publishing royalties from a publication relating to health care. Dr. Gilbert has received personal compensation in the range of $500-$4,999 for serving as a Medical Second Opinion Expert with Teldoc/Advanced Medical. Dr. Gilbert has received personal compensation in the range of $10,000-$49,999 for serving as a Medical Expert with Department of Health and Human Services/Vaccine Injury Compensation Program.
	No disclosure on file
Robert A. Duarte, MD (Northwell)	Dr. Duarte has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for LILLY . Dr. Duarte has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Allergan . Dr. Duarte has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for LILLY . Dr. Duarte has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for LILLY . Dr. Duarte has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Allergan. Dr. Duarte has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biohaven. Dr. Duarte has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for HPMB.
Harvey S. Singer, MD, FAAN (Johns Hopkins Hospital and Kennedy Krieger Institute)	No disclosure on file
Roger M. Kurlan, MD, FAAN	No disclosure on file
Rudolf M. Kwan, MD (Schering-Plough Research Institute)	No disclosure on file
Richard Chipkin (Psyadon Pharmaceuticals)	No disclosure on file
David H. Margolin, MD, PhD (Genzyme Corporation)	Dr. Margolin has received personal compensation for serving as an employee of uniQure, Inc.. Dr. Margolin has stock in Cerevance, Inc. Dr. Margolin has stock in Datacubed Health. Dr. Margolin has stock in uniQure, Inc.. Dr. Margolin has received intellectual property interests from a discovery or technology relating to health care.

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