Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults (D1AMOND)

• ClinicalTrials.gov Identifier: NCT05615220
• Recruitment Status: Recruiting
• First Posted: November 14, 2022
• Last Update Posted: October 17, 2023
• Sponsor: Emalex Biosciences Inc.
• Information provided by (Responsible Party): Emalex Biosciences Inc.

Tracking Information

• First Submitted Date: November 7, 2022
• First Posted Date: November 14, 2022
• Last Update Posted Date: October 17, 2023
• Actual Study Start Date: January 31, 2023
• Estimated Primary Completion Date: December 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 7, 2022)

Yale Global Tic Severity Scale (YGTSS) [ Time Frame: Screening up to Week 24 ]

Clinician-completed rating scale with score ranging from 0 to 50 (0=none to 50=severe)

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults
• Official Title: A Multicenter, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Maintenance of Efficacy of Ecopipam in Children, Adolescents and Adults With Tourette's Disorder
• Brief 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 double-blind, placebo-controlled, randomized withdrawal period.
• Detailed Description: Following a 28-day Screening period and Baseline visit, eligible subjects will be enrolled into the open-label Stabilization period comprised of a 4-week Titration phase to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) followed by an 8-week open-label Maintenance phase. Responders to ecopipam will be randomized to ecopipam 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) or placebo in a 1:1 fashion and enter the double-blind Randomized-Withdrawal (R/WD) period at Week 12. During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.
• Study Type: Interventional
• Study Phase: Phase 3

Study Design

Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

This is a multicenter study which includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Masking: Triple (Participant, Care Provider, Investigator)
Masking Description:

This includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Primary Purpose: Treatment

• Condition: Tourette Disorder

Intervention

Drug: Ecopipam Hydrochloride

Selective dopamine D1 and D5 receptor antagonist

Study Arms

• Experimental: 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl)
  Ecopipam 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 mg tablets (containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively); 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) target dose; oral administration daily in evenings.
  Intervention: Drug: Ecopipam Hydrochloride
• Placebo Comparator: Placebo during R/WD Phase
  Matching Placebo tablets during R/WD period taken orally in the evening.
  Intervention: Drug: Ecopipam Hydrochloride

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: November 7, 2022): 196
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2024
• Estimated Primary Completion Date: December 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• ≥ 6 years of age
• ≥ 18 kg (~ 40 lbs.)
• TD diagnosis and both motor and vocal tics that cause impairment with normal routines
• Minimum score of 20 on the YGTSS-R Total Tic Score
• May not be taking any medications used to treat motor or vocal tics for at least 14 days prior to Baseline.
• Effective contraception during the study and 30 days after last study dose for sexually active subjects

Exclusion Criteria:

• Previous exposure to ecopipam
• Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder)
• Unstable unstable medical illness or clinically significant lab abnormalities
• Risk of suicide
• Pregnant or lactating women
• Moderate to severe renal insufficiency
• Hepatic insufficiency
• Positive urine drug screen
• Unstable doses for drugs to treat anxiety, depression, Attention Deficit Hyperactivity Disorder
• Certain medications that would lead to drug interactions
• Recent behavioral therapy

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Meredith M Miller; 773 343 0671; mmiller@emalexbiosciences.com

Contact: David JB Kim; 312 847 1342; dkim@emalexbiosciences.com

• Listed Location Countries: Bulgaria, Canada, Poland, Spain, United States

Administrative Information

• NCT Number: NCT05615220
• Other Study ID Numbers: EBS-101-TD-301
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Emalex Biosciences Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Emalex Biosciences Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Emalex Biosciences Inc.
• Verification Date: July 2023