Back to resultsMetyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
Primary Purpose
Velo-cardio-facial Syndrome, Psychosis
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Metyrosine
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Velo-cardio-facial Syndrome focused on measuring Patients with velocardiofacial syndrome and psychosis
Eligibility Criteria
Key Inclusion Criteria:
- 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. -
Key Exclusion Criteria:
- 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. -
Sites / Locations
- VCFS International Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Metyrosine
Placebo
Arm Description
Outcomes
Primary Outcome Measures
To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Secondary Outcome Measures
To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Full Information
NCT ID
NCT01127503
First Posted
May 19, 2010
Last Updated
November 12, 2019
Sponsor
Bausch Health Americas, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01127503
Brief Title
Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
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 Type
Interventional
2. Study Status
Record Verification Date
November 2019
Overall Recruitment Status
Terminated
Why Stopped
Enrollment, study-design and execution challenges.
Study Start Date
June 2010 (undefined)
Primary Completion Date
November 2010 (Actual)
Study Completion Date
September 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bausch Health Americas, Inc.
4. Oversight
Data Monitoring Committee
No
5. 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Velo-cardio-facial Syndrome, Psychosis
Keywords
Patients with velocardiofacial syndrome and psychosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Metyrosine
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Metyrosine
Intervention Description
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
Intervention Name(s)
Placebo
Intervention Description
Placebo capsules were identically matched to Metyrosine.
Primary Outcome Measure Information:
Title
To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time Frame
13 weeks
Secondary Outcome Measure Information:
Title
To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time Frame
13 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
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. -
Key Exclusion Criteria:
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. -
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert J Shprintzen, PhD
Organizational Affiliation
Upstate Medical University
Official's Role
Principal Investigator
Facility Information:
Facility Name
VCFS International Center
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
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