Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
This study has been terminated.
(Enrollment, study-design and execution challenges.)
Information provided by (Responsible Party):
Valeant Pharmaceuticals International, Inc.
First received: May 19, 2010
Last updated: September 28, 2011
Last verified: September 2011
This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
||A Double-Blind, Placebo-Controlled, Multi-Center, Randomized Trial of the Safety and Efficacy of Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velo-Cardio-Facial Syndrome
Primary Outcome Measures:
- To evaluate the safety of metyrosine (Demser®) for the treatment of psychosis in patients with VCFS [ Time Frame: 13 weeks ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- To evaluate the efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with VCFS [ Time Frame: 13 weeks ] [ Designated as safety issue: No ]
| Study Start Date:
| Estimated Study Completion Date:
| Primary Completion Date:
||November 2010 (Final data collection date for primary outcome measure)
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).
Placebo Comparator: Placebo
Placebo capsules were identically matched to Metyrosine.
|Ages Eligible for Study:
||16 Years to 65 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Key Inclusion Criteria:
- Females of childbearing potential cannot be at risk of pregnancy during the study.
- Genetically confirmed diagnosis of VCFS at the time of screening.
- Must have one of the following Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition Text Revision (DSM-IV TR) diagnoses (applicable based upon clinical assessments): schizophrenia, schizoaffective disorder, psychosis not otherwise specified (NOS), bipolar disorder, or mood disorder with psychotic features.
- A total PANSS composite score >65.
- Willing to discontinue psychotropic medications. -
Key Exclusion Criteria:
- Evidence of acute suicidality.
- Known or observed clinically significant cardiovascular, pulmonary, renal, hepatic, or gastrointestinal disorders; other clinically significant psychiatric/neurological and sleep disorders by DSM-IV-TR criteria; endocrine, or hematological or metabolic diseases.
- Full scale IQ of less than 50.
- Not using a reliable means of contraception.
- Systolic blood pressure of ≤110 mm/Hg or ≥160 mm/Hg, diastolic blood pressure ≤60 mm/Hg or ≥90 mm/Hg, or has clinically symptomatic orthostatic changes.
- QTcF > 450 msec, or PR > 250 msec, or QRS > 110 msec on ECG.
- History of seizure disorder. -
Please refer to this study by its ClinicalTrials.gov identifier: NCT01127503
|VCFS International Center
|Syracuse, New York, United States, 13210 |
Valeant Pharmaceuticals International, Inc.
||Robert J Shprintzen, PhD
||Upstate Medical University
No publications provided
||Valeant Pharmaceuticals International, Inc.
History of Changes
|Other Study ID Numbers:
|Study First Received:
||May 19, 2010
||September 28, 2011
||United States: Food and Drug Administration
Keywords provided by Valeant Pharmaceuticals International, Inc.:
Patients with velocardiofacial syndrome and psychosis
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on March 09, 2014
Schizophrenia and Disorders with Psychotic Features
22q11 Deletion Syndrome
Heart Defects, Congenital
Genetic Diseases, Inborn
Endocrine System Diseases
Nervous System Diseases
Signs and Symptoms