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

NCT01127503 | Terminated Phase 2 Results

• 1 other identifier: 09-MET-101
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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

Keywords

Patients with velocardiofacial syndrome and psychosis

Outcome Measures

Primary Outcomes (1)

• To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS

  13 weeks

Secondary Outcomes (1)

• To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS

  13 weeks

Study Arms (2)

Metyrosine

EXPERIMENTAL

Drug: Metyrosine

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Metyrosine 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).

Metyrosine

Placebo DRUG

Placebo capsules were identically matched to Metyrosine.

Placebo

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Females of childbearing potential cannot be at risk of pregnancy during the study.
• Genetically confirmed diagnosis of VCFS at the time of screening.
• 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.
• A total PANSS composite score \>65.
• Willing to discontinue psychotropic medications. -

You may not qualify if:

• Evidence of acute suicidality.
• 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.
• Full scale IQ of less than 50.
• Pregnancy.
• Not using a reliable means of contraception.
• 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.
• QTcF \> 450 msec, or PR \> 250 msec, or QRS \> 110 msec on ECG.
• History of seizure disorder. -

Sponsors & Collaborators

• Bausch Health Americas, Inc.: lead

Study Sites (1)

VCFS International Center

Syracuse, New York, 13210, United States

Location

MeSH Terms

Conditions

DiGeorge Syndrome; Psychotic Disorders

Interventions

alpha-Methyltyrosine

Condition Hierarchy (Ancestors)

22q11 Deletion Syndrome; Craniofacial Abnormalities; Musculoskeletal Abnormalities; Musculoskeletal Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Lymphatic Abnormalities; Lymphatic Diseases; Hemic and Lymphatic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Hypoparathyroidism; Parathyroid Diseases; Endocrine System Diseases; Schizophrenia Spectrum and Other Psychotic Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Methyltyrosines; Tyrosine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Johnson Varughese
• Organization: Valeant Pharmaceuticals

9089271400

Study Officials

• Robert J Shprintzen, PhD

  Upstate Medical University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2010
• First Posted: May 21, 2010
• Study Start: June 1, 2010
• Primary Completion: November 1, 2010
• Study Completion: September 1, 2011
• Last Updated: November 22, 2019
• Results First Posted: May 8, 2018

Record last verified: 2019-11

Locations

US (1)