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Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

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ClinicalTrials.gov Identifier: NCT01751802
Recruitment Status : Terminated (Study terminated for commercial reasons)
First Posted : December 18, 2012
Results First Posted : October 8, 2015
Last Update Posted : October 8, 2015
Sponsor:
Information provided by (Responsible Party):
Psyadon Pharma

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Lesch-Nyhan Disease
Self-injurious Behavior
Interventions Drug: Ecopipam
Drug: Placebo
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ecopipam Then Placebo Then Ecopipam Placebo Then Ecopipam Then Placebo
Hide Arm/Group Description Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks
Period Title: Overall Study
Started 5 4
Completed Period 1 2 4
Completed Period 2 2 1
Completed Period 3 2 1
Completed 1 2
Not Completed 4 2
Reason Not Completed
Adverse Event             4             2
Arm/Group Title Ecopipam Then Placebo Then Ecopipam Placebo Then Ecopipam Then Placebo Total
Hide Arm/Group Description Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks Total of all reporting groups
Overall Number of Baseline Participants 5 4 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 5 participants 4 participants 9 participants
10.8
(6 to 22)
9.8
(6 to 12)
10.3
(6 to 22)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 4 participants 9 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
5
 100.0%
4
 100.0%
9
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 5 participants 4 participants 9 participants
United States 2 1 3
Spain 3 3 6
Weight  
Mean (Full Range)
Unit of measure:  Kg
Number Analyzed 5 participants 4 participants 9 participants
28.3
(16.5 to 55.4)
21.9
(16.0 to 26.5)
25.4
(16.0 to 55.4)
BMI  
Mean (Full Range)
Unit of measure:  Kg/m^2
Number Analyzed 5 participants 4 participants 9 participants
15.6
(13.9 to 18.1)
13.8
(13.1 to 15.5)
14.8
(13.1 to 18.1)
1.Primary Outcome
Title Behavior Problems Inventory - Self-Injurious Behavior Subscale
Hide Description The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.
Time Frame Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who completed the BPI-Self Injurious Behavior survey for 3 or more time points
Arm/Group Title Subject #1: Ecopipam Then Placebo Then Ecopipam Subject #2: Ecopipam Then Placebo Then Ecopipam Subject #3: Placebo Then Ecopipam Then Placebo Subject #4: Placebo Then Ecopipam Then Placebo
Hide Arm/Group Description:
Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks
Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks
Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks
Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks; then Ecopipam, the active substance being tested, 100mg tablets for body weight > 20kg OR 50 mg tablets for body weight up to 20kg, once daily at bedtime for 6 weeks; then Placebo, the inactive substance being tested to be taken once a day at bedtime for 6 weeks
Overall Number of Participants Analyzed 1 1 1 1
Mean (Standard Deviation)
Unit of Measure: scores on a scale
BPI Self-Injurious Behavior Subscale, Baseline 26 10 78 27
BPI Self-Injurious Behavior Subscale, 6 weeks 24.1 4.3 58.3 22.6
BPI Self-Injurious Behavior Subscale, 12 weeks 25.2 11.6 21.5 16.0
BPI Self-Injurious Behavior Subscale, 18 weeks 21 6.6 28.7 NA [1] 
[1]
participant did not complete subscale survey at 18 wks
2.Secondary Outcome
Title Effect of Ecopipam Withdrawal and Maintenance
Hide Description The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND. Measured by the number of participants whose score changes significantly from baseline on ecopipam or placebo
Time Frame Baseline, 6 weeks, 12 weeks, 18 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
0 participants analyzed because data are not reliable
Arm/Group Title Ecopipam Placebo
Hide Arm/Group Description:

Active substance being tested, orally once a day at bedtime

Ecopipam: Antagonist of the dopamine D1 receptor

Inactive substance being tested, orally once a day at bedtime

Placebo: Placebo for Ecopipam

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods
Hide Description An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks. Total number of serious and non-serious adverse events experienced by participants while receiving ecopipam or placebo. For additional detail, see Adverse Events
Time Frame Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose
Arm/Group Title Ecopipam Placebo
Hide Arm/Group Description:

Active substance being tested, orally once a day at bedtime for 6 weeks

Ecopipam: Antagonist of the dopamine D1 receptor

Inactive substance being tested, orally once a day at bedtime for 6 weeks

Placebo: Placebo for Ecopipam

Overall Number of Participants Analyzed 9 6
Measure Type: Number
Unit of Measure: adverse events
Non-Serious 42 7
Serious 2 1
Time Frame adverse events collected while participants were receiving ecopipam or placebo, either for 1 period (up to 6 weeks) or 2 periods (up to 12 weeks)
Adverse Event Reporting Description Adverse events were collected for 3 double-blind periods
 
Arm/Group Title Ecopipam Placebo
Hide Arm/Group Description

Active substance being tested, orally once a day at bedtime

Ecopipam: Antagonist of the dopamine D1 receptor

Inactive substance being tested, orally once a day at bedtime

Placebo: Placebo for Ecopipam

All-Cause Mortality
Ecopipam Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Ecopipam Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/9 (22.22%)      1/6 (16.67%)    
Nervous system disorders     
Dystonic crisis * [1]  1/9 (11.11%)  0/6 (0.00%) 
Psychiatric disorders     
Unusual somnolence * [2]  1/9 (11.11%)  0/6 (0.00%) 
Renal and urinary disorders     
Nephrolithiasis * [3]  0/9 (0.00%)  1/6 (16.67%)  1
*
Indicates events were collected by non-systematic assessment
[1]
Subject 05-001 was hospitalized a second time (July 2013) for a dystonic crisis, difficulty swallowing (dysphagia), and depressed mood.
[2]
Subject hospitalized for dysphagia, unusual somnolence and depressed mood, as reported by parents. Causality was rated Reasonable Possibility by investigator. Subject was discharged as somnolence resolved; and subject withdrew because of these AEs.
[3]
During hospitalization, subject also experienced opisthotonus, bronchospasm, hypereosinophilia, & elevated transaminase; all AEs rated as unrelated to ecopipam. Ecopipam continued during hospitalization. All symptoms resolved w/o sequelae.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Ecopipam Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/9 (88.89%)      2/6 (33.33%)    
Blood and lymphatic system disorders     
Eosinophilia *  1/9 (11.11%)  1 0/6 (0.00%)  0
Gastrointestinal disorders     
Abdominal pain *  2/9 (22.22%)  2 0/6 (0.00%)  0
Constipation *  2/9 (22.22%)  2 0/6 (0.00%)  0
Diarrhoea *  1/9 (11.11%)  1 0/6 (0.00%)  0
Dysphagia *  5/9 (55.56%)  5 0/6 (0.00%)  0
Vomiting *  1/9 (11.11%)  1 0/6 (0.00%)  0
General disorders     
Mucosal inflammation *  0/9 (0.00%)  0 1/6 (16.67%)  1
Pyrexia *  0/9 (0.00%)  0 1/6 (16.67%)  1
Infections and infestations     
Oral candidiasis *  1/9 (11.11%)  1 0/6 (0.00%)  0
Injury, poisoning and procedural complications     
Lip injury *  1/9 (11.11%)  1 0/6 (0.00%)  0
Investigations     
Body temperature increased *  1/9 (11.11%)  1 0/6 (0.00%)  0
Transaminases increased *  1/9 (11.11%)  1 0/6 (0.00%)  0
Nervous system disorders     
Dyskinesia *  0/9 (0.00%)  0 1/6 (16.67%)  1
Dystonia *  2/9 (22.22%)  2 0/6 (0.00%)  0
Opisthotonus *  2/9 (22.22%)  2 0/6 (0.00%)  0
Somnolence *  5/9 (55.56%)  5 0/6 (0.00%)  0
Tremor *  1/9 (11.11%)  1 0/6 (0.00%)  0
Psychiatric disorders     
Agitation *  2/9 (22.22%)  2 0/6 (0.00%)  0
Anxiety *  1/9 (11.11%)  1 1/6 (16.67%)  1
Compulsive lip biting *  1/9 (11.11%)  1 0/6 (0.00%)  0
Depressed mood *  1/9 (11.11%)  1 0/6 (0.00%)  0
Depression *  3/9 (33.33%)  3 0/6 (0.00%)  0
Insomnia *  2/9 (22.22%)  2 1/6 (16.67%)  1
Self injurious behaviour *  4/9 (44.44%)  4 1/6 (16.67%)  1
Renal and urinary disorders     
Nephrolithiasis *  1/9 (11.11%)  1 0/6 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Bronchospasm *  0/9 (0.00%)  0 1/6 (16.67%)  1
Choking *  1/9 (11.11%)  1 0/6 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
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.
Results Point of Contact
Name/Title: President/CEO
Organization: Psyadon Pharmaceuticals
Phone: 301-919-2020
Responsible Party: Psyadon Pharma
ClinicalTrials.gov Identifier: NCT01751802     History of Changes
Other Study ID Numbers: PSY102
First Submitted: December 14, 2012
First Posted: December 18, 2012
Results First Submitted: October 5, 2015
Results First Posted: October 8, 2015
Last Update Posted: October 8, 2015