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

• Sponsor: Bausch Health Americas, Inc. (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT01127503
• Collaborator: (none)
• Enrollment: 2
• Location: 1
• Arms: 2
• Duration (Months): 15
• Patients Per Site Per Month: 0.1

Study Details

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
• 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: Jun 1, 2010
• Actual Primary Completion Date: Nov 1, 2010
• Actual Study Completion Date: Sep 1, 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 [13 weeks]

Secondary Outcome Measures

1. To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS [13 weeks]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 16 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

	Site	City	State	Country	Postal Code
1	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Bausch Health Americas, Inc.
• ClinicalTrials.gov Identifier: NCT01127503
• Other Study ID Numbers: 09-MET-101
• First Posted: May 21, 2010
• Last Update Posted: Nov 22, 2019
• Last Verified: Nov 1, 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; alpha-Methyltyrosine

Study Results

Participant Flow

Arm/Group Title	Metyrosine	Placebo
Arm/Group Description	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: Placebo capsules were identically matched to Metyrosine.
Period Title: Overall Study
STARTED	1	1
COMPLETED	1	1
NOT COMPLETED	0	0

Baseline Characteristics

Arm/Group Title	Metyrosine	Placebo	Total
Arm/Group Description	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: Placebo capsules were identically matched to Metyrosine.	Total of all reporting groups
Overall Participants	1	1	2
Age, Customized (participants) [Number]
22	1 100%	0 0%	1 50%
32	0 0%	1 100%	1 50%
Sex: Female, Male (Count of Participants)
Female	1 100%	0 0%	1 50%
Male	0 0%	1 100%	1 50%
Region of Enrollment (participants) [Number]
United States	1 100%	1 100%	2 100%

Outcome Measures

1. Primary Outcome

Title	To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time Frame	13 weeks

Outcome Measure Data

Analysis Population Description

The study was terminated early and no data was collected

Arm/Group Title	Metyrosine	Placebo
Arm/Group Description	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: Placebo capsules were identically matched to Metyrosine.
Measure Participants	0	0

2. Secondary Outcome

Title	To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time Frame	13 weeks

Outcome Measure Data

Analysis Population Description

The study was terminated early and no data was collected

Arm/Group Title	Metyrosine	Placebo
Arm/Group Description	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: Placebo capsules were identically matched to Metyrosine.
Measure Participants	0	0

Adverse Events

Arm/Group Title	Metyrosine		Placebo
Arm/Group Description	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: Placebo capsules were identically matched to Metyrosine.
All Cause Mortality
	Metyrosine		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Metyrosine		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/1 (0%)		0/1 (0%)
Other (Not Including Serious) Adverse Events
	Metyrosine		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/1 (0%)		0/1 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.