Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension

NCT04114539 | Completed Phase 2 Results FDA Drug

• 1 other identifier: EBS-101-OL-001
• Study Type: interventional
• Target: 124
• Locations: 5 countries
• Sites: 65

Brief Summary

This study was an international, multicenter, open-label, long term extension study evaluating the safety of ecopipam tablets for the treatment of children and adolescent subjects with Tourette Syndrome.

Conditions

Tourette Syndrome in Children; Tourette Syndrome in Adolescence

Keywords

Tourette Syndrome

Outcome Measures

Primary Outcomes (22)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related)

  An AE was any untoward medical condition that occurs in a participant while participating in this clinical study. It can be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study drug, whether or not related to the study. An SAE was any untoward medical occurrence that at any dose met one, more of the following criteria: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event. The relationship of the study drug in causing or contributing to the AE whether unrelated, possibly related, or probably related was decided by investigator medical judgment.

  From start of study drug administration until 30 days after last dose (Up to Month 13)

• Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells

  Basophils/Leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in basophils to leukocytes ratio is reported in terms of percentage of cells.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells

  Eosinophils/Leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in eosinophils to leukocytes ratio is reported in terms of percentage of cells.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Erythrocytes

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameter erythrocytes was reported.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Hematocrit

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameter hematocrit was reported.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Hemoglobin

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameter hemoglobin was reported.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Leukocytes and Platelets

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameters (Leukocytes and Platelets) expressed in 10\^9 cells per liter were reported.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells

  Lymphocytes/leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in lymphocytes to leukocytes ratio is reported in terms of percentage of cells.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells

  Monocytes/leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in monocytes to leukocytes ratio is reported in terms of percentage of cells.

  Baseline up to Month 12

• Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells

  Neutrophils/leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in neutrophils to leukocytes ratio is reported in terms of percentage of cells.

  Baseline up to Month 12

• Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase, and lactate dehydrogenase) were reported.

  Baseline up to Month 12

• Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (albumin, globulin and protein) were reported.

  Baseline up to Month 12

• Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (bicarbonate, calcium, chloride, cholesterol, glucose, phosphate, potassium, sodium, triglyceride, urea nitrogen) expressed in millimoles per liter (mmol/L) were reported.

  Baseline up to Month 12

• Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (bilirubin, creatinine and direct bilirubin) expressed in micromole per liter (mcmol/L) were reported.

  Baseline up to Month 12

• Change From Baseline in Hemoglobin A1c (HbA1c)

  HbA1c is the glycosylated fraction of hemoglobin A. It is measured to identify average blood glucose concentration over prolonged periods of time. Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in HbA1c was reported.

  Baseline up to Month 12

• Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs parameters diastolic blood pressure and systolic blood pressure and according to the assessment position (supine and standing) expressed in millimeter(s) of mercury (mmHg) was reported.

  Baseline up to Month 12

• Change From Baseline in Vital Signs Parameter: Pulse Rate

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital sign parameter pulse rate and according to assessment position (supine and standing) was reported.

  Baseline up to Month 12

• Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI]

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs parameter BMI was reported.

  Baseline up to Month 12

• Change From Baseline in Vital Signs Parameter: Height

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs height was reported.

  Baseline up to Month 12

• Change From Baseline in Vital Signs Parameter: Weight

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs parameter weight was reported.

  Baseline up to Month 12

• Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval

  Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change From Baseline in ECG parameters aggregate PR interval, aggregate QRS duration, aggregate QT interval, and aggregate QTc interval expressed in millisecond (msec) was reported.

  Baseline to Month 12

• Number of Participants With Clinically Significant Abnormal Physical Examination Findings

  Physical examination included examination of the following body areas and systems: Head, Eyes, Ears, Nose, Mouth, Throat, Neck (including Thyroid), Thorax, Abdomen, Urogenital, Extremities, Neurological, Skin and Mucosae and Others. Any clinically significant abnormalities in physical examination were judged by the investigator. Only non-zero values are reported.

  Baseline up to Month 12

Secondary Outcomes (6)

• Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12

  Baseline up to Months 1, 3, 6, 9 and 12

• Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12

  Baseline up to Months 1, 3, 6, 9 and 12

• Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12

  Baseline up to Months 1, 3, 6, 9 and 12

• Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12

  Baseline up to Months 1, 3, 6, 9, 12

• Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12

  Months 1, 3, 6, 9 and 12

Study Arms (1)

Ecopipam

EXPERIMENTAL

Drug: Ecopipam

Interventions

Ecopipam DRUG

Ecopipam HCl 12.5-, 37.5-. 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings.

Ecopipam

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects must have completed the EBS-101-CL-001 study through the Day 14 Follow Up Visit within the last 30 days (or longer with permission of the medical monitor) without a major reportable protocol deviation and must be someone the Investigator feels would benefit from continued participation.

You may not qualify if:

• Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder).
• Unstable medical illness or clinically significant lab abnormalities.
• Risk of suicide.
• Pregnant or lactating women.
• Moderate to severe renal insufficiency.
• Positive urine drug screen.
• Certain medications that would lead to drug interactions.

Sponsors & Collaborators

• Emalex Biosciences Inc.: lead

Study Sites (65)

Harmonex Neuroscience Research

Dothan, Alabama, 36303, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Advanced Research Center Inc.

Anaheim, California, 92805, United States

Location

UCLA

Los Angeles, California, 90095, United States

Location

PCSD-Feighner Research

San Diego, California, 92108, United States

Location

Syrentis Clinical Research

Santa Ana, California, 92705, United States

Location

Yale School of Medicine

New Haven, Connecticut, 06519, United States

Location

Sarkis Clinical Trials

Gainesville, Florida, 32607, United States

Location

Northwest Florida Clinical Research Group, LLC

Gulf Breeze, Florida, 32561, United States

Location

Research in Miami Inc.

Hialeah, Florida, 33013, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

MedBio Trials

North Miami, Florida, 33180, United States

Location

APG Research LLC

Orlando, Florida, 32803, United States

Location

University of South Florida

St. Petersburg, Florida, 33701-4825, United States

Location

Pediatric Epilepsy and Neurology Specialists

Tampa, Florida, 33609-4181, United States

Location

Pediatric Neurology, PA

Winter Park, Florida, 32789, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

Location

Meridian Clinical Research

Savannah, Georgia, 31406, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612-3841, United States

Location

The University of Chicago Hospitals

Chicago, Illinois, 60637-1447, United States

Location

AMR - Baber Research Inc.

Naperville, Illinois, 60563-6510, United States

Location

Psychiatric Associates

Overland Park, Kansas, 66211, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Michigan Clinical Research Institute PC

Ann Arbor, Michigan, 48105, United States

Location

Neurobehavioral Medicine Group

Bloomfield Hills, Michigan, 48302-1952, United States

Location

Helen DeVos Children's Hospital / Spectrum Health Medical Group

Wyoming, Michigan, 49418, United States

Location

St. Charles Psychiatric Associates dba Midwest Research Group

Saint Charles, Missouri, 63304, United States

Location

Movement Disorders Center

St Louis, Missouri, 63110-1093, United States

Location

Alivation Research, LLC

Lincoln, Nebraska, 68526, United States

Location

Center for Psychiatry and Behavioral Medicine Inc.

Las Vegas, Nevada, 89128, United States

Location

The NeuroCognitive Institute

Mount Arlington, New Jersey, 07856, United States

Location

Clinical Research Center of NJ

Voorhees Township, New Jersey, 08043-1910, United States

Location

New York Neurology Associates P.C

New York, New York, 10003, United States

Location

Hapworth Research Inc.

New York, New York, 10019, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029-6504, United States

Location

Mood Disorders Consulting Medicine PLLC

New York, New York, 10036, United States

Location

Finger Lakes Clinical Research

Rochester, New York, 14618, United States

Location

Quest Therapeutics of Avon Lake

Avon Lake, Ohio, 44012-1004, United States

Location

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229-3026, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

North Star Medical Research LLC

Middleburg Heights, Ohio, 44130, United States

Location

Suburban Research Associates

Media, Pennsylvania, 19063, United States

Location

Coastal Pediatric Research

Charleston, South Carolina, 29414, United States

Location

Access Clinical Trials, Inc.

Nashville, Tennessee, 37203-6502, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-0028, United States

Location

Houston Clinical Trials LLC

Bellaire, Texas, 77401, United States

Location

Relaro Medical Trials

Dallas, Texas, 75243, United States

Location

North Texas Clinical Trials

Fort Worth, Texas, 76104, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Road Runner Research Ltd.

San Antonio, Texas, 78249-3539, United States

Location

Noetic Psychiatry

Springville, Utah, 84663, United States

Location

University of Virginia

Charlottesville, Virginia, 22908-0829, United States

Location

Eastside Therapeutic Resource Inc dba Core Clinical Research

Everett, Washington, 98201-4077, United States

Location

The Kids Clinic Inc

Ajax, Ontario, L1Z 0M1, Canada

Location

Center for Pediatric Excellence

Ottawa, Ontario, K2G 1W2, Canada

Location

CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

CHU Poitiers

Poitiers, 86021, France

Location

Pharmakologisches Studienzentrum Chemnitz GmbH

Mittweida, 09648, Germany

Location

Gdanskie Centrum Zdrowia Sp z o.o.

Gdansk, 80-542, Poland

Location

Centrum Bada Klinicznych PI-House Sp. z o.o.

Gdansk, 80-546, Poland

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis

Katowice, 40-123, Poland

Location

Centrum Medyczne Plejady

Krakow, 30-363, Poland

Location

Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie

Krakow, 31-503, Poland

Location

Med-Polonia Sp. z o. o.

Poznan, 60-693, Poland

Location

MeSH Terms

Conditions

Tourette Syndrome

Interventions

ecopipam

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Sr. Director of Clinical Operations
• Organization: Emalex Biosciences, Inc.

dkim@emalexbiosciences.com

1847

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open Label

Study Record Dates

• First Submitted: September 30, 2019
• First Posted: October 3, 2019
• Study Start: October 4, 2019
• Primary Completion: November 11, 2022
• Study Completion: November 11, 2022
• Last Updated: February 1, 2024
• Results First Posted: December 1, 2023

Record last verified: 2023-11

Locations

DE (1); CA (3); FR (1); US (53); PL (7)