Tourette's Syndrome
Conditions
Keywords
Tourette's, Ecopipam, Dopamine receptors, Efficacy, Safety, Adults
Brief summary
Tourette's Syndrome is a neurological disease characterized by verbal and motor tics. The currently available drug treatments are considered to be inadequate. This clinical trial is designed to test if ecopipam is effective for the treatment of Tourette's Syndrome in adults.
Detailed description
Tourette's Syndrome is a neurological disease characterized by verbal and motor tics. Although its causes are unknown, many researchers believe that changes in brain chemicals (called neurotransmitters) are critically involved. One of these neurotransmitters is called dopamine, and it exerts its actions through its receptors (called D1-type or D2-type). It has been suggested that the symptoms of Tourette's Syndrome are due to an overactivity at the D1-type receptor. Ecopipam is a selective antagonist of the D1-type receptors. The present clinical trial is designed to test if ecopipam is able to relieve the symptoms of the disease in adults patients with Tourette's Syndrome. Eligible patients will be treated for eight weeks.
Interventions
DRUGEcopipam
50 or 100 mg tablets given once per day for eight weeks
Sponsors
Psyadon Pharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects must have TS (Tourette's Syndrome) based on the clinician-administered DCI (Diagnostic Confidence Index) for TS. * Subjects must exhibit both motor and vocal tics. * Subjects must have exhibited tics for \>5 years. * Subjects must have a minimum score of 20 at both Screening and Baseline (just prior to the first treatment) on the YGTSS (Yale Global Tic Severity Score). * Subjects must be age ≥ 18 years. * Women must be postmenopausal (\> 12 months without menses) or surgically sterile (i.e., by hysterectomy and/or bilateral oophorectomy) or must be using effective contraception (i.e., oral contraceptives, intrauterine device, double barrier method of condom and spermicide) and agree to continue use of contraception for the duration of their participation in the study. Women of childbearing potential must agree to use contraception for 30 days after their last dose of study drug. * Sexually active male subjects 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. * Subject must execute a written informed consent.
Exclusion criteria
* Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening. * Subjects with a major depressive episode in the past 2 years * Subjects with a history of attempted suicide * Subjects with clinically significant suicidality (score of ≥ 2 on Item 3 for the Hamilton Depression Rating Scale \[HAM-D\]) * Subjects with a first-degree relative with a major depressive episode that resulted in any psychiatric hospitalization, attempted or completed suicide * Subjects with a history of seizures. * Subjects with a myocardial infarction within 6 months. * Women of childbearing potential who are currently pregnant or lactating. * Subjects who have a need for medication (other than ecopipam) with possible effects on TS symptoms (i.e., lithium, naltrexone, methylphenidate, or psychostimulants), unfavorable interactions with ecopipam (ie, dopamine agonists \[including bupropion\]), or monoamine oxidase inhibitors. * Subjects with a lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any psychotic disorder determined by the Structured Clinical Interview for Diagnostic Statistical Manual IV Text Revision (DSM-IV-TR) Axis-I Disorders (SCID). * Subjects with current or recent (past 3 months) DSM-IV substance abuse or dependence (with the exception of nicotine). * Subjects with positive urine drug screen (cocaine, amphetamine, methamphetamine, tetrahydrocannabinol (THC), benzodiazepines, barbiturates, phencyclidine (PCP), opiates) at Screening. Subjects with urine positive only for benzodiazepines and/or marijuana (i.e., a user but not an abuser as based on DSM-IV criteria) may be eligible. * Subjects who have had previous treatment with ecopipam. * Subjects who have had treatment with: * investigational medication or depot neuroleptics within 3 months * fluoxetine within 6 weeks * other psychotropics with possible effects on TS symptoms (ie, lithium, or naltrexone) within 2 weeks prior to Screening. * oral neuroleptics within 2 weeks * selective serotonin reuptake inhibitors unless the dosage has been stable for a minimum of 4 weeks prior to study start
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Yale Global Tic Severity Score | 8 weeks | The Yale Global Tic Severity Score is a composite of subject reported severity of motor (range 0-25) and vocal (range 0-25) tics , as well as an impairment score (range 0-50). The outcome we are using is the Total Tic Severity score which is the sum of the motor and vocal tic severity scores (range 0-50). The higher the score on this scale, the more severe the symptoms. A positive drug effect is associated with a decrease from baseline. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hamilton Depression Scale | Every 7 days | This is a measure of feelings of depression that the patient might have. |
| Premonitory Urge for Tics Scale (PUTS-1) | Every 7 days | This is a measure of the tic behavior that is seen in Tourette's patients, and it is typically used in these types of trials. |
| Adult Attention Deficit/Hyperactivity Disorder (ADHD) Self-report Symptom Checklist (ASRS) | Every 7 days | This is a standard measure of ADHD severity that is typically used in these types of clinical trials. |
| Safety Assessments | Every 7 days | Patients will be evaluated for any adverse events, and they will have a variety of blood tests to examine if any changes occur. |
| Columbia Scale for Suicide Risk | Every 7 days | This test monitors whether the patient has any feelings of committing self-harm. It is mandated by the FDA to include this scale in all clinical trials of new central nervous system drugs. |
| Clinician Global Impression - Improvement and Severity Scales (CGI) | End of trial | This is a measure of how the treating physician perceives the effectiveness of a drug treatment, and it is typically used in these types of clinical trials. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Ecopipam Active treatment
Ecopipam: 50 or 100 mg tablets given once per day for eight weeks | 18 |
| Total | 18 |
Baseline characteristics
| Characteristic | Ecopipam |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 18 Participants |
| Age, Continuous | 36 years STANDARD_DEVIATION 15 |
| Region of Enrollment United States | 18 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 6 / 18 |
| serious Total, serious adverse events | 0 / 18 |
Outcome results
Primary
Yale Global Tic Severity Score
The Yale Global Tic Severity Score is a composite of subject reported severity of motor (range 0-25) and vocal (range 0-25) tics , as well as an impairment score (range 0-50). The outcome we are using is the Total Tic Severity score which is the sum of the motor and vocal tic severity scores (range 0-50). The higher the score on this scale, the more severe the symptoms. A positive drug effect is associated with a decrease from baseline.
Time frame: 8 weeks
Population: Subjects completing the study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam | Yale Global Tic Severity Score | 25.3 units on a scale | Standard Deviation 9.2 |
Secondary
Adult Attention Deficit/Hyperactivity Disorder (ADHD) Self-report Symptom Checklist (ASRS)
This is a standard measure of ADHD severity that is typically used in these types of clinical trials.
Time frame: Every 7 days
Secondary
Clinician Global Impression - Improvement and Severity Scales (CGI)
This is a measure of how the treating physician perceives the effectiveness of a drug treatment, and it is typically used in these types of clinical trials.
Time frame: End of trial
Secondary
Columbia Scale for Suicide Risk
This test monitors whether the patient has any feelings of committing self-harm. It is mandated by the FDA to include this scale in all clinical trials of new central nervous system drugs.
Time frame: Every 7 days
Secondary
Hamilton Depression Scale
This is a measure of feelings of depression that the patient might have.
Time frame: Every 7 days
Secondary
Premonitory Urge for Tics Scale (PUTS-1)
This is a measure of the tic behavior that is seen in Tourette's patients, and it is typically used in these types of trials.
Time frame: Every 7 days
Secondary
Safety Assessments
Patients will be evaluated for any adverse events, and they will have a variety of blood tests to examine if any changes occur.
Time frame: Every 7 days