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

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)

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 exploratory study in adult subjects with childhood onset fluency disorder.

Study Overview

• Status: Completed
• Conditions: Childhood-Onset Fluency Disorder (Stuttering)
• Intervention / Treatment: Drug: Placebo; Drug: Ecopipam

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
• Enrollment (Actual): 68
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Riverside, California, United States, 92501
      • UC Riverside
    • Santa Ana, California, United States, 92705
      • CI Trials
  • Florida
    • Jacksonville, Florida, United States, 32256
      • Clinical Neuroscience Solutions Inc
    • Orlando, Florida, United States, 32801
      • Clinical Neuroscience Solutions Inc
  • Georgia
    • Atlanta, Georgia, United States, 30341
      • Institute for Advanced Medical Research
  • Kansas
    • Prairie Village, Kansas, United States, 66208
      • Social Psychiatry Research Institute
  • Michigan
    • East Lansing, Michigan, United States, 48824
      • Michigan State University
  • New York
    • Brooklyn, New York, United States, 11235
      • Social Psychiatry Research Institute
  • Tennessee
    • Memphis, Tennessee, United States, 38119
      • Clinical Neuroscience Solutions Inc

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ecopipam HCl ~2mg/kg/day; Ecopipam HCl tablets of 12.5, 50, and 75 mg for daily, oral administration for 12 weeks	Drug: Ecopipam; Oral tablet
Placebo Comparator: Matching Placebo; Matching placebo tablets for daily, oral administration for 12 weeks	Drug: Placebo; Oral tablet

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Stuttering Severity Instrument, 4th edition (SSI-4) baseline to Week 12	Measures stuttering severity in children and adults	12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Global Impression - Severity (CGI-S) baseline to Week 12	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.	12 weeks

Collaborators and Investigators

• Sponsor: Emalex Biosciences Inc.
• Collaborators: Premier Research Group plc; University of California, Riverside
• Investigators: Study Director: Atul R Mahableshwarkar, MD, DFAPA, Emalex Biosciences Inc.

Study record dates

Study Major Dates

• Study Start (Actual): October 2, 2020
• Primary Completion (Actual): March 7, 2022
• Study Completion (Actual): April 4, 2022

Study Registration Dates

• First Submitted: July 27, 2020
• First Submitted That Met QC Criteria: July 27, 2020
• First Posted (Actual): July 30, 2020

Study Record Updates

• Last Update Posted (Actual): April 28, 2022
• Last Update Submitted That Met QC Criteria: April 26, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Keywords: Stuttering; Communication Disorders
• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Neurodevelopmental Disorders; Language Disorders; Communication Disorders; Speech Disorders; Stuttering; Childhood-Onset Fluency Disorder; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Antipsychotic Agents; Tranquilizing Agents; Psychotropic Drugs; Dopamine Agents; Dopamine Antagonists; Ecopipam
• Other Study ID Numbers: EBS-101-COFD-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No