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

Psychosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01127503
• Other study ID #: 09-MET-101
• Status: Terminated
• Phase: Phase 2
• Start date: June 2010
• Est. completion date: September 2011

Study information

• Verified date: November 2019
• Source: Bausch Health Americas, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: September 2011
• Est. primary completion date: November 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 65 Years

Eligibility

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. -

Related Conditions & MeSH terms

• DiGeorge Syndrome
• Mental Disorders
• Psychosis
• Psychotic Disorders
• Syndrome
• Velo-cardio-facial Syndrome

Intervention

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
Placebo capsules were identically matched to Metyrosine.

Locations

Country	Name	City	State
United States	VCFS International Center	Syracuse	New York

Sponsors (1)

Lead Sponsor	Collaborator
Bausch Health Americas, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS		13 weeks
Secondary	To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS		13 weeks