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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01127503
Recruitment Status : Terminated (Enrollment, study-design and execution challenges.)
First Posted : May 21, 2010
Last Update Posted : September 29, 2011
Information provided by (Responsible Party):
Valeant Pharmaceuticals International, Inc.

Brief Summary:
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 or disease Intervention/treatment Phase
Velo-cardio-facial Syndrome Psychosis Drug: Metyrosine Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : June 2010
Primary Completion Date : November 2010
Estimated Study Completion Date : September 2011

Arm Intervention/treatment
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.

Primary Outcome Measures :
  1. To evaluate the safety of metyrosine (Demser®) for the treatment of psychosis in patients with VCFS [ Time Frame: 13 weeks ]

Secondary Outcome Measures :
  1. To evaluate the efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with VCFS [ Time Frame: 13 weeks ]

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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. -

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01127503

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

Responsible Party: Valeant Pharmaceuticals International, Inc. Identifier: NCT01127503     History of Changes
Other Study ID Numbers: 09-MET-101
First Posted: May 21, 2010    Key Record Dates
Last Update Posted: September 29, 2011
Last Verified: September 2011

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

Additional relevant MeSH terms:
Craniofacial Abnormalities
Psychotic Disorders
Mental Disorders
DiGeorge Syndrome
Facial Paralysis
Pathologic Processes
Schizophrenia Spectrum and Other Psychotic Disorders
22q11 Deletion Syndrome
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
Parathyroid Diseases
Endocrine System Diseases
Mouth Diseases
Stomatognathic Diseases
Neurologic Manifestations
Nervous System Diseases