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

NCT ID: NCT01127503

Last Updated: 2019-11-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-06-30
• Study Completion Date: 2011-09-30

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).

Conditions

Velo-cardio-facial Syndrome; Psychosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Metyrosine

Group Type: EXPERIMENTAL

Metyrosine

Intervention Type: 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).

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo capsules were identically matched to Metyrosine.

Interventions

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).

Intervention Type: DRUG

Placebo

Placebo capsules were identically matched to Metyrosine.

Intervention Type: DRUG

Eligibility Criteria

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. -

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. -

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bausch Health Americas, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Robert J Shprintzen, PhD

Role: PRINCIPAL_INVESTIGATOR

Upstate Medical University

Locations

VCFS International Center

Syracuse, New York, United States

Countries

United States

Other Identifiers

09-MET-101

Identifier Type: -

Identifier Source: org_study_id