Expanded Access Program for Treatment With Ecopipam for Tourette's Disorder

Available (expanded access)

Conditions studied: Tourettes Disorder, Tourette Disorder

In brief

The goal of this expanded access study is to learn about the long-term effects of Ecopipam in participants with Tourette's Disorder (TD). The main question it aims to answer is: * Is ecopipam safe and well-tolerated in participants equal and over the age of 6 to 50 with TD? * Assess quality of life measures and to provide access to ecopipam while NDA is being prepared and submitted for review for marketing approval Eligible participants with TD will take ecopipam with guidance from their treating physician. In addition to the standard of care from the treating physician, treatment with ecopipam will continue until ecopipam becomes commercially available in the United States or the program is terminated. While in the EAP program, participants will be asked regarding how safe and tolerable ecopipam is to the participant. Several questionnaires will be collected regarding participants experiences while taking ecopipam during monthly visits for the first year and every three months thereafter until the conclusion of the study.

Key facts

Study ID

NCT07093541

Run by

Emalex Biosciences Inc.

Last updated by the study team

2026-05-18

Who can join

Age: 6 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants must fulfill all of the following inclusion criteria to be eligible for participation in the Study:
• Age 6 to 50 years old inclusive at the time of informed consent/assent.
• Participant must sign/date the most current Informed Consent Form (ICF). For minor participants the LAR must/sign date the most current consent and the participant must sign/date the most current age-appropriate assent.
• In the opinion of the principal investigator (PI), the participant or in the case of a minor their LAR is capable of understanding and complying with protocol and program requirements, oral drug administration and care instructions.
• Weight ≥18 kg (39.6 lbs.).
• In the opinion of the PI, the participant meets diagnostic criteria for Tourette's Syndrome (TS) per Diagnostic and Statistical manual for Mental Disorders - 5th Edition (DSM-5-TR diagnostic criteria for TD).
• The participant must have been treated with an FDA-approved treatment for TD (aripiprazole, haloperidol, or pimozide) or any other D2r antagonist (D2rA) and have experienced treatment failure, tolerability or safety issues as per LAR, participant or prescribing clinician or not have access to any of these approved medications.
• Females of child-bearing potential must have a negative urine pregnancy test at Screening.
• Females of child-bearing potential who are sexually active or become sexually active must be using double barrier method (condom and IUD, condom and spermicide, etc.) in addition to any oral contraceptive they may be using and agree to continue use of double barrier contraception for the duration of their participation in the study and for 30 days after their last dose of study drug. Females of non-childbearing potential are exempt from this criterion.
• Non-Childbearing potential is defined by:
• Post menopausal defined as >= 2 years amenorrheic, or,
• Bilateral salpingo-oopherectomy, or
• Hysterectomy, or
• Pre-menarche
• Sexually active male participants must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
• Male participants must agree to not donate sperm and female participants not to donate eggs for at least 30 days after the last dose of study drug.
• Participants cannot be enrolled in the study if they meet any of the following criteria.
• Clinically meaningful abnormalities on laboratory tests at Screening as determined by the PI.
• Specific laboratory findings at enrollment
• T. Bili >= 2.5 x ULN for age (Participants with a history of Gilbert's Disorder must have prior documentation of the disorder.)
• ALT >= 2.5 x ULN for age
• AST >= 2.5 x ULN for age
• Positive urine drug screen for cocaine, amphetamine, benzodiazepines, barbiturates, phencyclidine (PCP), or opiates at Screening, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder (ADHD) or a positive for benzodiazepine prescribed (either routine or as needed) for any anxiety disorder or anxiety associated with a depressive disorder
• Severe renal impairment or end stage renal disease as defined by eGFR < 60 ml/min/1.73m2
• Any of the following cardiovascular:

Where it is running

• Cortica Glendale — Glendale, California, United States
• Cortica Marin — San Rafael, California, United States
• Yale Child Study Center — New Haven, Connecticut, United States
• Parkinson's Center of Boca Raton — Boca Raton, Florida, United States
• University of Miami — Miami, Florida, United States
• Belkis Delgado, MD, PA — Miami, Florida, United States
• University of South Florida — St. Petersburg, Florida, United States
• Emory University — Atlanta, Georgia, United States
• Northwestern University — Chicago, Illinois, United States
• University of Chicago (Pritzker) — Chicago, Illinois, United States
• Norton Children's Research Institute — Louisville, Kentucky, United States
• Johns Hopkins Hospital — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Massachusetts- Memorial Health — Worcester, Massachusetts, United States
• Neurobehavioral Medicine Group — Bloomfield Hills, Michigan, United States
• Washington University in St. Louis — St Louis, Missouri, United States
• NYU Langone — New York, New York, United States
• Mount Sinai — New York, New York, United States
• Stony Brook Medicine — Stony Brook, New York, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• North Star Medical Research, LLC — Middleburg Heights, Ohio, United States
• Providence — Portland, Oregon, United States
• Access Clinical Trials, INC — Nashville, Tennessee, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States

Full record on ClinicalTrials.gov

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