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

Bausch Health

Status and phase: Terminated; Phase 2

Conditions: Psychosis; Velo-cardio-facial Syndrome

Treatments: Drug: Placebo; Drug: Metyrosine

Study type: Interventional

Funder types: Industry

Identifiers

NCT01127503

09-MET-101

Details and patient eligibility

About

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).

Enrollment: 2 patients

Sex: All

Ages: 16 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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. -

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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2 participants in 2 patient groups, including a placebo group

Metyrosine

Experimental group

Treatment:

Drug: Metyrosine

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo