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

• ClinicalTrials.gov Identifier: NCT01127503
• Recruitment Status: Terminated
• First Posted: May 21, 2010
• Results First Posted: May 8, 2018
• Last Update Posted: November 22, 2019
• Sponsor: Bausch Health Americas, Inc.
• Information provided by (Responsible Party): Bausch Health Americas, Inc.

Study Description

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 Design

• 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
• Actual Primary Completion Date: November 2010
• Actual Study Completion Date: September 2011

Arms and Interventions

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.

Outcome Measures

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 ]

Eligibility Criteria

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

Criteria

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. -

Contacts and Locations

Locations

United States, New York

VCFS International Center

Syracuse, New York, United States, 13210

Sponsors and Collaborators

Bausch Health Americas, Inc.

Investigators

• Principal Investigator: Robert J Shprintzen, PhD; Upstate Medical University

More Information

• Responsible Party: Bausch Health Americas, Inc.
• ClinicalTrials.gov Identifier: NCT01127503
• Other Study ID Numbers: 09-MET-101
• First Posted: May 21, 2010
• Results First Posted: May 8, 2018
• Last Update Posted: November 22, 2019
• Last Verified: November 2019

Keywords provided by Bausch Health Americas, Inc.:

Patients with velocardiofacial syndrome and psychosis

Additional relevant MeSH terms:

DiGeorge Syndrome; Syndrome; Psychotic Disorders; Mental Disorders; Disease; Pathologic Processes; Schizophrenia Spectrum and Other Psychotic Disorders; 22q11 Deletion Syndrome; Craniofacial Abnormalities; 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; Hypoparathyroidism; Parathyroid Diseases; Endocrine System Diseases; alpha-Methyltyrosine; Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action