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

Phase 2

Terminated

• Conditions: Velo-cardio-facial Syndrome; Psychosis
• Interventions: Drug: Placebo; Drug: Metyrosine

• Registration Number: NCT01127503
• Lead Sponsor: Bausch Health Americas, 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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-05-19
• Study First Submitted QC: 2010-05-20
• Study First Posted: 2010-05-21
• Study Start: 2010-06
• Primary Completion: 2010-11
• Study Completion: 2011-09
• Results First Submitted: 2017-12-19
• Results First Submitted QC: 2018-04-05
• Results First Posted: 2018-05-08
• Status Verified: 2019-11
• Last Update Submitted: 2019-11-12
• Last Update Posted: 2019-11-22

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2

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. -

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
Metyrosine	Metyrosine	-

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

VCFS International Center; 🇺🇸 Syracuse, New York, United States