Psychosis, Velo-cardio-facial Syndrome
Conditions
Keywords
Patients with velocardiofacial syndrome and psychosis
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).
Interventions
DRUGMetyrosine
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).
DRUGPlacebo
Placebo capsules were identically matched to Metyrosine.
Sponsors
Bausch Health Americas, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
16 Years to 65 Years
Healthy volunteers
No
Inclusion 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. -
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS | 13 weeks |
Secondary
| Measure | Time frame |
|---|---|
| To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS | 13 weeks |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Metyrosine 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). | 1 |
| Placebo Placebo: Placebo capsules were identically matched to Metyrosine. | 1 |
| Total | 2 |
Baseline characteristics
| Characteristic | Metyrosine | Placebo | Total |
|---|---|---|---|
| Age, Customized 22 | 1 participants | 0 participants | 1 participants |
| Age, Customized 32 | 0 participants | 1 participants | 1 participants |
| Region of Enrollment United States | 1 participants | 1 participants | 2 participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | 1 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 1 | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 | 0 / 1 |
Outcome results
Primary
To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time frame: 13 weeks
Population: The study was terminated early and no data was collected
Secondary
To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time frame: 13 weeks
Population: The study was terminated early and no data was collected