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

Overview

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).

Full Title of Study: “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 Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: November 2010

Interventions

  • 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).
  • Drug: Placebo
    • Placebo capsules were identically matched to Metyrosine.

Arms, Groups and Cohorts

  • Experimental: Metyrosine
  • Placebo Comparator: Placebo

Clinical Trial Outcome Measures

Primary Measures

  • To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
    • Time Frame: 13 weeks

Secondary Measures

  • To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
    • Time Frame: 13 weeks

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 16 Years

Maximum Age: 65 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Bausch Health Americas, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Robert J Shprintzen, PhD, Principal Investigator, Upstate Medical University

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