Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

Phase 3 Completed 216 enrolled Emalex Biosciences Inc.

RandomizedSequentialTriple-blindTreatment

Summary

This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period.

Timeline

Start: 2023-01-31
Primary completion: 2025-01-13
Completion: 2025-02-04

Outcome

Met primary endpoint

paper Ecopipam significantly reduced the relapse risk vs placebo in pediatric participants (hazard ratio [HR], 0.47; 95% CI, 0.26-0.84; P = .008; n = 90). PMID 42189524 ↗

Publications

Gilbert DL, Atkinson SD, Kim DJB, Miller MM, Rice PM, Flatt JA, Karkanias GB, Munschauer FE, Bittman RM, Wanaski SP, Cunniff TM, Tomczak KK. Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial. JAMA Neurol. 2026 Jul 1;83(7):645-653. doi: 10.1001/jamaneurol.2026.1431.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ecopipam	Small molecule	1.8 mg/kg	Oral