Effects of Ecopipam or Placebo in Adults With Stuttering (Speak Freely)

• ClinicalTrials.gov Identifier: NCT04492956
• Recruitment Status: Completed
• First Posted: July 30, 2020
• Last Update Posted: April 28, 2022
• Sponsor: Emalex Biosciences Inc.
• Collaborators: University of California, Riverside; Premier Research Group plc
• Information provided by (Responsible Party): Emalex Biosciences Inc.

Tracking Information

• First Submitted Date: July 27, 2020
• First Posted Date: July 30, 2020
• Last Update Posted Date: April 28, 2022
• Actual Study Start Date: October 2, 2020
• Actual Primary Completion Date: March 7, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 27, 2020)

Change in Stuttering Severity Instrument, 4th edition (SSI-4) baseline to Week 12 [ Time Frame: 12 weeks ]

Measures stuttering severity in children and adults

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 27, 2020)

Clinical Global Impression - Severity (CGI-S) baseline to Week 12 [ Time Frame: 12 weeks ]

Rates the severity of the subject's illness at the time of assessment, relative to the the clinician's past experience with patients who have the same diagnosis.

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

Descriptive Information

• Brief Title: Effects of Ecopipam or Placebo in Adults With Stuttering (Speak Freely)
• Official Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 2 Exploratory Study to Evaluate the Efficacy and Safety of Ecopipam Tablets in Adults With Childhood Onset Fluency Disorder (Stuttering)
• Brief Summary: This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 exploratory study in adult subjects with childhood onset fluency disorder.

Detailed Description

At the Baseline visit, eligible subjects will be randomized 1:1 to receive either a target steady-state dose of ecopipam HCl ~2 mg/kg/day or matching placebo for a 12-week Treatment Period consisting of a 4-week Titration Phase followed by an 8-week Maintenance Phase.

Subjects will return to the clinic at 4, 8, and 12 weeks after Randomization and Follow-up visits 7 and 14 days after completing the Treatment Period or Early Discontinuation. Efficacy assessments will be conducted at Weeks 4, 8, and 12 and safety assessments will be conducted at all visits. Subjects will have adverse events and other safety parameters assessed by phone or video conference at Weeks 2, 6 and 10 and 30 days after the last study drug administration. Signs or symptoms of withdrawal, abuse, and dependence will be monitored throughout the study.

At the end of the Treatment Period or Early Discontinuation, subjects will taper study drug by 25 mg/day until off study drug, for up to 1 week.

• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Childhood-Onset Fluency Disorder (Stuttering)

Intervention

• Drug: Ecopipam
  Oral tablet
• Drug: Placebo
  Oral tablet

Study Arms

• Experimental: Ecopipam HCl ~2mg/kg/day
  Ecopipam HCl tablets of 12.5, 50, and 75 mg for daily, oral administration for 12 weeks
  Intervention: Drug: Ecopipam
• Placebo Comparator: Matching Placebo
  Matching placebo tablets for daily, oral administration for 12 weeks
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 27, 2020): 68
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: April 4, 2022
• Actual Primary Completion Date: March 7, 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Able to read and write in English and provide informed consent
• 18 years or older of age at screening
• Weighs >= 45 kg (~99 lbs)
• Satisfies DSM-5 criteria for childhood onset fluency disorder
• History of stuttering for >=2 years with onset consistent to developmental in nature
• Must meet an allowed severity of stuttering at screening and baseline, scored by a central rater
• Completed an adequate course of speech therapy
• Has a qualifying IOS or Android smartphone
• Must discontinue all medications used to treat stuttering for at least 14 days prior to screening
• Sexually active females of child bearing potential must be using effective contraception during and 30 days after participation
• Sexually active males must use a double barrier method of contraception during and 30 days after participation

Exclusion Criteria:

• Stuttering is related to a known neurological cause
• Initiation of new behavioral therapies for stuttering within 10 weeks prior to baseline
• Unstable medical illness or clinically significant abnormalities on screening tests/exams
• At a significant risk of committing suicide
• Are pregnant or lactating
• Positive urine drug screen
• Lifetime history of major depressive episode
• History of seizures
• Have been previously treated with ecopipam
• Unstable use of medications prior to screening
• Use of prohibited medications or need for medications which would have unfavorable interactions with ecopipam
• Not suitable for study in the opinion of the Principle Investigator

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04492956
• Other Study ID Numbers: EBS-101-COFD-201
• Has Data Monitoring Committee: No

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

• Plan to Share IPD: No

• Responsible Party: Emalex Biosciences Inc.
• Study Sponsor: Emalex Biosciences Inc.

Collaborators

• University of California, Riverside
• Premier Research Group plc

Investigators

• Study Director: Atul R Mahableshwarkar, MD, DFAPA; Emalex Biosciences Inc.

• PRS Account: Emalex Biosciences Inc.
• Verification Date: April 2022