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Enrollment, study-design and execution challenges.
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This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (DemserĀ®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Metyrosine | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Metyrosine | Drug | Metyrosine (250 mg capsules) were to be used at all dose levels (administered as multiples of that dosing unit). The starting dose was 250 mg/day of metyrosine. Dose escalation was to be carried out weekly for 8 weeks (up to a maximum of 8 capsules/day [2000 mg/day if metyrosine]) with dosage increments of 1 capsule/day per week. Weekly dose escalation was to stop based upon the investigator's assessment of safety, but not efficacy (i.e., dose escalation was to be forced to the maximum of 8 capsules/day assuming acceptable safety and tolerability). |
| Measure | Description | Time Frame |
|---|---|---|
| To Evaluate the Safety of Metyrosine (DemserĀ®) for the Treatment of Psychosis in Patients With VCFS | 13 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| To Evaluate the Efficacy of Metyrosine (DemserĀ®) for the Treatment of Psychosis in Patients With VCFS | 13 weeks |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Robert J Shprintzen, PhD | Upstate Medical University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| VCFS International Center | Syracuse | New York | 13210 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Metyrosine | Metyrosine: Metyrosine (250 mg capsules) were to be used at all dose levels (administered as multiples of that dosing unit). The starting dose was 250 mg/day of metyrosine. Dose escalation was to be carried out weekly for 8 weeks (up to a maximum of 8 capsules/day [2000 mg/day if metyrosine]) with dosage increments of 1 capsule/day per week. Weekly dose escalation was to stop based upon the investigator's assessment of safety, but not efficacy (i.e., dose escalation was to be forced to the maximum of 8 capsules/day assuming acceptable safety and tolerability). |
| FG001 | Placebo | Placebo: Placebo capsules were identically matched to Metyrosine. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Metyrosine | Metyrosine: Metyrosine (250 mg capsules) were to be used at all dose levels (administered as multiples of that dosing unit). The starting dose was 250 mg/day of metyrosine. Dose escalation was to be carried out weekly for 8 weeks (up to a maximum of 8 capsules/day [2000 mg/day if metyrosine]) with dosage increments of 1 capsule/day per week. Weekly dose escalation was to stop based upon the investigator's assessment of safety, but not efficacy (i.e., dose escalation was to be forced to the maximum of 8 capsules/day assuming acceptable safety and tolerability). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number |
| 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 | To Evaluate the Safety of Metyrosine (DemserĀ®) for the Treatment of Psychosis in Patients With VCFS | The study was terminated early and no data was collected | Posted | 13 weeks |
|
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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 | Metyrosine | Metyrosine: Metyrosine (250 mg capsules) were to be used at all dose levels (administered as multiples of that dosing unit). The starting dose was 250 mg/day of metyrosine. Dose escalation was to be carried out weekly for 8 weeks (up to a maximum of 8 capsules/day [2000 mg/day if metyrosine]) with dosage increments of 1 capsule/day per week. Weekly dose escalation was to stop based upon the investigator's assessment of safety, but not efficacy (i.e., dose escalation was to be forced to the maximum of 8 capsules/day assuming acceptable safety and tolerability). |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Johnson Varughese | Valeant Pharmaceuticals | 9089271400 |
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| ID | Term |
|---|---|
| D004062 | DiGeorge Syndrome |
| D011618 | Psychotic Disorders |
| ID | Term |
|---|---|
| D058165 | 22q11 Deletion Syndrome |
| D019465 | Craniofacial Abnormalities |
| D009139 | Musculoskeletal Abnormalities |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D019805 | alpha-Methyltyrosine |
| ID | Term |
|---|---|
| D008781 | Methyltyrosines |
| D014443 | Tyrosine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
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|
| Placebo | Drug | Placebo capsules were identically matched to Metyrosine. |
|
| BG001 | Placebo | Placebo: Placebo capsules were identically matched to Metyrosine. |
| BG002 | Total | Total of all reporting groups |
| participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Placebo: Placebo capsules were identically matched to Metyrosine. |
|
| Secondary | To Evaluate the Efficacy of Metyrosine (DemserĀ®) for the Treatment of Psychosis in Patients With VCFS | The study was terminated early and no data was collected | Posted | 13 weeks |
|
|
| 0 |
| 1 |
| 0 |
| 1 |
| EG001 | Placebo | Placebo: Placebo capsules were identically matched to Metyrosine. | 0 | 1 | 0 | 1 |
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| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D044148 | Lymphatic Abnormalities |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D007011 | Hypoparathyroidism |
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |
| D019967 | Schizophrenia Spectrum and Other Psychotic Disorders |
| D001523 | Mental Disorders |
| D000596 |
| Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |