Ecopipam Treatment of Tourette Syndrome

• Sponsor: Psyadon Pharma (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01244633
• Collaborator: (none)
• Enrollment: 18
• Locations: 4
• Arm: 1
• Duration (Months): 33
• Patients Per Site: 4.5
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Tourette's Syndrome	Drug: Ecopipam	Phase 1/Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 18 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Ecopipam Treatment of Tourette Syndrome
• Study Start Date: Oct 1, 2010
• Actual Primary Completion Date: Jul 1, 2012
• Actual Study Completion Date: Jul 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Ecopipam Active treatment	Drug: Ecopipam; 50 or 100 mg tablets given once per day for eight weeks; Other Names: SCH 39166 PSYRX 101

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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.

2. Hamilton Depression Scale [Every 7 days]

   This is a measure of feelings of depression that the patient might have.

3. 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.

4. 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.

5. 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.

6. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Johns Hopkins University	Baltimore	Maryland	United States	21287
2	Atlantic Neuroscience Institute Overlook Hospital	Summit	New Jersey	United States	07092
3	North Shore Hospital	Manhasset	New York	United States	11030
4	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229

Sponsors and Collaborators

• Psyadon Pharma

Investigators

• Principal Investigator: Donald Gilbert, MD, Children's Hospital Medical Center, Cincinnati

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Link to Sponsor's Website

Publications

• Chipkin RE, Iorio LC, Coffin VL, McQuade RD, Berger JG, Barnett A. Pharmacological profile of SCH39166: a dopamine D1 selective benzonaphthazepine with potential antipsychotic activity. J Pharmacol Exp Ther. 1988 Dec;247(3):1093-102.

• Responsible Party: Psyadon Pharma
• ClinicalTrials.gov Identifier: NCT01244633
• Other Study ID Numbers: PSY301
• First Posted: Nov 19, 2010
• Last Update Posted: Sep 29, 2015
• Last Verified: Jun 1, 2015

• Keywords provided by Psyadon Pharma: Tourette's; Ecopipam; Dopamine receptors; Efficacy; Safety; Adults
• Additional relevant MeSH terms: Tourette Syndrome; Syndrome; Ecopipam

Study Results

Participant Flow

Arm/Group Title	Ecopipam
Arm/Group Description	Active treatment Ecopipam: 50 or 100 mg tablets given once per day for eight weeks
Period Title: Overall Study
STARTED	18
COMPLETED	15
NOT COMPLETED	3

Baseline Characteristics

Arm/Group Title	Ecopipam
Arm/Group Description	Active treatment Ecopipam: 50 or 100 mg tablets given once per day for eight weeks
Overall Participants	18
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	18 100%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	36 (15)
Sex: Female, Male (Count of Participants)
Female	3 16.7%
Male	15 83.3%
Region of Enrollment (participants) [Number]
United States	18 100%

Outcome Measures

1. Primary Outcome

Title	Yale Global Tic Severity Score
Description	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

Outcome Measure Data

Analysis Population Description

Subjects completing the study

Arm/Group Title	Ecopipam
Arm/Group Description	Active treatment Ecopipam: 50 or 100 mg tablets given once per day for eight weeks
Measure Participants	15
Mean (Standard Deviation) [units on a scale]	25.3 (9.2)

2. Secondary Outcome

Title	Adult Attention Deficit/Hyperactivity Disorder (ADHD) Self-report Symptom Checklist (ASRS)
Description	This is a standard measure of ADHD severity that is typically used in these types of clinical trials.
Time Frame	Every 7 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

3. Secondary Outcome

Title	Hamilton Depression Scale
Description	This is a measure of feelings of depression that the patient might have.
Time Frame	Every 7 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

4. Secondary Outcome

Title	Premonitory Urge for Tics Scale (PUTS-1)
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

5. Secondary Outcome

Title	Clinician Global Impression - Improvement and Severity Scales (CGI)
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

6. Secondary Outcome

Title	Safety Assessments
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

7. Secondary Outcome

Title	Columbia Scale for Suicide Risk
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

Adverse Events

Time Frame	8 weeks
Arm/Group Title	Ecopipam
Arm/Group Description	Active treatment Ecopipam: 50 or 100 mg tablets given once per day for eight weeks
All Cause Mortality
	Ecopipam
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Ecopipam
	Affected / at Risk (%)	# Events
Total	0/18 (0%)
Other (Not Including Serious) Adverse Events
	Ecopipam
	Affected / at Risk (%)	# Events
Total	6/18 (33.3%)
Nervous system disorders
Insomnia	6/18 (33.3%)

Limitations/Caveats

This was an open label study with no placebo control

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.