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Study terminated for commercial reasons
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| Name | Class |
|---|---|
| Psyadon Pharma | INDUSTRY |
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The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness and safety of placebo (inactive substance) in subjects with Lesch-Nyhan Disease.
This study will be done in approximately 6 centers in approximately 4 countries, and approximately 24 subjects will be included. This study is divided into two parts. The first is a double-blinded portion lasting up to 18 weeks in total. The second portion is an optional open-label extension and lasts up to 54 weeks total. The total duration of the study, if you choose to participate in both portions, is anticipated to be up to approximately 78 weeks.
The first portion of this study is double-blind and assignment to a treatment group is done randomly. In this study, there are two treatment groups. One group will receive Ecopipam for one 6-week period and placebo for two 6-week periods, and the other group will receive Ecopipam for two 6-week periods and placebo for one 6-week period.
Subjects who did not experience any clinically significant side effects during the blinded portion of the study may be eligible to participate in an open-label extension that may last up to 54 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ecopipam | Experimental | Active substance being tested, orally once a day at bedtime |
|
| Placebo | Placebo Comparator | Inactive substance being tested, orally once a day at bedtime |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ecopipam | Drug | Antagonist of the dopamine D1 receptor |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Behavior Problems Inventory - Self-Injurious Behavior Subscale | 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. | Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks), |
| Measure | Description | Time Frame |
|---|---|---|
| Effect of Ecopipam Withdrawal and Maintenance | 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 | Baseline, 6 weeks, 12 weeks, 18 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| H J Jinnah, MD | Emory University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| H.J. Jinnah | Atlanta | Georgia | 30322 | United States | ||
| Hospital Universitario La Paz |
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| ID | Title | Description |
|---|---|---|
| FG000 | Ecopipam Then Placebo Then Ecopipam | 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 |
| FG001 | Placebo Then Ecopipam Then Placebo | 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 |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Ecopipam Then Placebo Then Ecopipam | 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 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Behavior Problems Inventory - Self-Injurious Behavior Subscale | 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. | All participants who completed the BPI-Self Injurious Behavior survey for 3 or more time points | Posted | Mean | Standard Deviation | scores on a scale | Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks), |
|
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 events were collected for 3 double-blind periods
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ecopipam | Active substance being tested, orally once a day at bedtime Ecopipam: Antagonist of the dopamine D1 receptor |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nephrolithiasis | Renal and urinary disorders | Non-systematic Assessment | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| President/CEO | Psyadon Pharmaceuticals | 301-919-2020 | info@psyadonrx.com |
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| ID | Term |
|---|---|
| D007926 | Lesch-Nyhan Syndrome |
| D016728 | Self-Injurious Behavior |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C058081 | ecopipam |
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| Placebo | Drug | Placebo for Ecopipam |
|
| 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 |
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 |
| Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks |
| Madrid |
| Spain |
| BG001 | Placebo Then Ecopipam Then Placebo | 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 |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Weight | Mean | Full Range | kg |
|
| BMI | Mean | Full Range | kg/m^2 |
|
| OG001 | Subject #2: Ecopipam Then Placebo Then Ecopipam | 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 |
| OG002 | Subject #3: Placebo Then Ecopipam Then Placebo | 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 |
| OG003 | Subject #4: Placebo Then Ecopipam Then Placebo | 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 |
|
|
| Secondary | Effect of Ecopipam Withdrawal and Maintenance | 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 | 0 participants analyzed because data are not reliable | Posted | Baseline, 6 weeks, 12 weeks, 18 weeks |
|
|
| Secondary | 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 | 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 | All participants who received at least one dose | Posted | Number | adverse events | Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks |
|
|
|
| 2 |
| 9 |
| 8 |
| 9 |
| EG001 | Placebo | Inactive substance being tested, orally once a day at bedtime Placebo: Placebo for Ecopipam | 1 | 6 | 2 | 6 |
|
| Dystonic crisis | Nervous system disorders | Non-systematic Assessment | Subject 05-001 was hospitalized a second time (July 2013) for a dystonic crisis, difficulty swallowing (dysphagia), and depressed mood. |
|
| Unusual somnolence | Psychiatric disorders | Non-systematic Assessment | 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. |
|
| Agitation | Psychiatric disorders | Non-systematic Assessment |
|
| Anxiety | Psychiatric disorders | Non-systematic Assessment |
|
| Body temperature increased | Investigations | Non-systematic Assessment |
|
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Choking | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Compulsive lip biting | Psychiatric disorders | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Non-systematic Assessment |
|
| Depressed mood | Psychiatric disorders | Non-systematic Assessment |
|
| Depression | Psychiatric disorders | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | Non-systematic Assessment |
|
| Dyskinesia | Nervous system disorders | Non-systematic Assessment |
|
| Dystonia | Nervous system disorders | Non-systematic Assessment |
|
| Eosinophilia | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Insomnia | Psychiatric disorders | Non-systematic Assessment |
|
| Lip injury | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Mucosal inflammation | General disorders | Non-systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | Non-systematic Assessment |
|
| Opisthotonus | Nervous system disorders | Non-systematic Assessment |
|
| Oral candidiasis | Infections and infestations | Non-systematic Assessment |
|
| Pyrexia | General disorders | Non-systematic Assessment |
|
| Self injurious behaviour | Psychiatric disorders | Non-systematic Assessment |
|
| Somnolence | Nervous system disorders | Non-systematic Assessment |
|
| Transaminases increased | Investigations | Non-systematic Assessment |
|
| Tremor | Nervous system disorders | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Non-systematic Assessment |
|
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| D009422 | Nervous System Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D008661 | Metabolism, Inborn Errors |
| D011686 | Purine-Pyrimidine Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D001526 | Behavioral Symptoms |
| D001519 | Behavior |