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.

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Childhood-Onset Fluency Disorder (Stuttering)	Drug: Ecopipam; Drug: Placebo	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 68 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: October 2, 2020
• Actual Primary Completion Date: March 7, 2022
• Actual Study Completion Date: April 4, 2022

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Change in Stuttering Severity Instrument, 4th edition (SSI-4) baseline to Week 12 [ Time Frame: 12 weeks ]
   Measures stuttering severity in children and adults

Secondary Outcome Measures :

1. 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.

Eligibility Criteria

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

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

Contacts and Locations

Locations

United States, California

UC Riverside

Riverside, California, United States, 92501

CI Trials

Santa Ana, California, United States, 92705

United States, Florida

Clinical Neuroscience Solutions Inc

Jacksonville, Florida, United States, 32256

Clinical Neuroscience Solutions Inc

Orlando, Florida, United States, 32801

United States, Georgia

Institute For Advanced Medical Research

Atlanta, Georgia, United States, 30341

United States, Kansas

Social Psychiatry Research Institute

Prairie Village, Kansas, United States, 66208

United States, Michigan

Michigan State University

East Lansing, Michigan, United States, 48824

United States, New York

Social Psychiatry Research Institute

Brooklyn, New York, United States, 11235

United States, Tennessee

Clinical Neuroscience Solutions Inc

Memphis, Tennessee, United States, 38119

Sponsors and Collaborators

Emalex Biosciences Inc.

University of California, Riverside

Premier Research Group plc

Investigators

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

More Information

• Responsible Party: Emalex Biosciences Inc.
• ClinicalTrials.gov Identifier: NCT04492956
• Other Study ID Numbers: EBS-101-COFD-201
• First Posted: July 30, 2020
• Last Update Posted: April 28, 2022
• Last Verified: April 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

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

Keywords provided by Emalex Biosciences Inc.:

Stuttering; Communication Disorders

Additional relevant MeSH terms:

Stuttering; Childhood-Onset Fluency Disorder; Speech Disorders; Language Disorders; Communication Disorders; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Neurodevelopmental Disorders; Mental Disorders; Ecopipam; Antipsychotic Agents; Tranquilizing Agents; Central Nervous System Depressants; Physiological Effects of Drugs; Psychotropic Drugs; Dopamine Antagonists; Dopamine Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action