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Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome

This study has been terminated.
(Enrollment, study-design and execution challenges.)
Sponsor:
Information provided by (Responsible Party):
Valeant Pharmaceuticals International, Inc.
ClinicalTrials.gov Identifier:
NCT01127503
First received: May 19, 2010
Last updated: September 28, 2011
Last verified: September 2011
History of Changes
  Purpose

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).


Condition Intervention Phase
Velo-cardio-facial Syndrome
Psychosis
 Drug: Metyrosine
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:

Drug Information available for: Metyrosine
U.S. FDA Resources

Further study details as provided by Valeant Pharmaceuticals International, Inc.:

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 ]

Enrollment: 2
Study Start Date: June 2010
Estimated Study Completion Date: September 2011
Primary Completion Date: November 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Metyrosine Drug: 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).
Placebo Comparator: Placebo Drug: Placebo
Placebo capsules were identically matched to Metyrosine.

  Eligibility

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Females of childbearing potential cannot be at risk of pregnancy during the study.
  2. Genetically confirmed diagnosis of VCFS at the time of screening.
  3. 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.
  4. A total PANSS composite score >65.
  5. Willing to discontinue psychotropic medications. -

Key Exclusion Criteria:

  1. Evidence of acute suicidality.
  2. 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.
  3. Full scale IQ of less than 50.
  4. Pregnancy.
  5. Not using a reliable means of contraception.
  6. 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.
  7. QTcF > 450 msec, or PR > 250 msec, or QRS > 110 msec on ECG.
  8. History of seizure disorder. -
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01127503

Locations
United States, New York
VCFS International Center
Syracuse, New York, United States, 13210
Sponsors and Collaborators
Valeant Pharmaceuticals International, Inc.
Investigators
Principal Investigator: Robert J Shprintzen, PhD Upstate Medical University
  More Information

No publications provided

Responsible Party: Valeant Pharmaceuticals International, Inc.
ClinicalTrials.gov Identifier: NCT01127503     History of Changes
Other Study ID Numbers: 09-MET-101
Study First Received: May 19, 2010
Last Updated: September 28, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Valeant Pharmaceuticals International, Inc.:
Patients with velocardiofacial syndrome and psychosis

Additional relevant MeSH terms:
Mental Disorders
Psychotic Disorders
DiGeorge Syndrome
Facial Paralysis
Schizophrenia and Disorders with Psychotic Features
22q11 Deletion Syndrome
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Musculoskeletal Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Lymphatic Abnormalities
Lymphatic Diseases
 Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Hypoparathyroidism
Parathyroid Diseases
Endocrine System Diseases
Mouth Diseases
Stomatognathic Diseases
Paralysis
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Alpha-Methyltyrosine
Enzyme Inhibitors

ClinicalTrials.gov processed this record on May 16, 2013