Ignite Creation Date:
2025-12-24 @ 7:48 PM
Ignite Modification Date:
2025-12-30 @ 6:16 PM
Study NCT ID:
NCT01127503
Status:
TERMINATED
Last Update Posted:
2019-11-22
First Post:
2010-05-19
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004062', 'term': 'DiGeorge Syndrome'}, {'id': 'D011618', 'term': 'Psychotic Disorders'}], 'ancestors': [{'id': 'D058165', 'term': '22q11 Deletion Syndrome'}, {'id': 'D019465', 'term': 'Craniofacial Abnormalities'}, {'id': 'D009139', 'term': 'Musculoskeletal Abnormalities'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D044148', 'term': 'Lymphatic Abnormalities'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D007011', 'term': 'Hypoparathyroidism'}, {'id': 'D010279', 'term': 'Parathyroid Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D019967', 'term': 'Schizophrenia Spectrum and Other Psychotic Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019805', 'term': 'alpha-Methyltyrosine'}], 'ancestors': [{'id': 'D008781', 'term': 'Methyltyrosines'}, {'id': 'D014443', 'term': 'Tyrosine'}, {'id': 'D024322', 'term': 'Amino Acids, Aromatic'}, {'id': 'D000598', 'term': 'Amino Acids, Cyclic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'phone': '9089271400', 'title': 'Johnson Varughese', 'organization': 'Valeant Pharmaceuticals'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Metyrosine', '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).", 'otherNumAtRisk': 1, 'otherNumAffected': 0, 'seriousNumAtRisk': 1, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Placebo: Placebo capsules were identically matched to Metyrosine.', 'otherNumAtRisk': 1, 'otherNumAffected': 0, 'seriousNumAtRisk': 1, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Metyrosine', '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)."}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo: Placebo capsules were identically matched to Metyrosine.'}], 'timeFrame': '13 weeks', 'reportingStatus': 'POSTED', 'populationDescription': 'The study was terminated early and no data was collected'}, {'type': 'SECONDARY', 'title': 'To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Metyrosine', '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)."}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo: Placebo capsules were identically matched to Metyrosine.'}], 'timeFrame': '13 weeks', 'reportingStatus': 'POSTED', 'populationDescription': 'The study was terminated early and no data was collected'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Metyrosine', '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)."}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Placebo: Placebo capsules were identically matched to Metyrosine.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Metyrosine', '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)."}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Placebo: Placebo capsules were identically matched to Metyrosine.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': '22', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': '32', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'whyStopped': 'Enrollment, study-design and execution challenges.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2010-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-11', 'completionDateStruct': {'date': '2011-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-11-12', 'studyFirstSubmitDate': '2010-05-19', 'resultsFirstSubmitDate': '2017-12-19', 'studyFirstSubmitQcDate': '2010-05-20', 'lastUpdatePostDateStruct': {'date': '2019-11-22', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-04-05', 'studyFirstPostDateStruct': {'date': '2010-05-21', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-05-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2010-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS', 'timeFrame': '13 weeks'}], 'secondaryOutcomes': [{'measure': 'To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS', 'timeFrame': '13 weeks'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Patients with velocardiofacial syndrome and psychosis'], 'conditions': ['Velo-cardio-facial Syndrome', 'Psychosis']}, 'descriptionModule': {'briefSummary': '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).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Females of childbearing potential cannot be at risk of pregnancy during the study.\n2. Genetically confirmed diagnosis of VCFS at the time of screening.\n3. 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.\n4. A total PANSS composite score \\>65.\n5. Willing to discontinue psychotropic medications. -\n\nKey Exclusion Criteria:\n\n1. Evidence of acute suicidality.\n2. 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.\n3. Full scale IQ of less than 50.\n4. Pregnancy.\n5. Not using a reliable means of contraception.\n6. 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.\n7. QTcF \\> 450 msec, or PR \\> 250 msec, or QRS \\> 110 msec on ECG.\n8. History of seizure disorder. -'}, 'identificationModule': {'nctId': 'NCT01127503', 'briefTitle': 'Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Bausch Health Americas, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': '09-MET-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Metyrosine', 'interventionNames': ['Drug: Metyrosine']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Metyrosine', 'type': 'DRUG', '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).", 'armGroupLabels': ['Metyrosine']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo capsules were identically matched to Metyrosine.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '13210', 'city': 'Syracuse', 'state': 'New York', 'country': 'United States', 'facility': 'VCFS International Center', 'geoPoint': {'lat': 43.04812, 'lon': -76.14742}}], 'overallOfficials': [{'name': 'Robert J Shprintzen, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Upstate Medical University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bausch Health Americas, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}