Ignite Creation Date:
2025-12-25 @ 12:00 AM
Ignite Modification Date:
2025-12-29 @ 10:02 PM
Study NCT ID:
NCT01065558
Status:
COMPLETED
Last Update Posted:
2013-04-18
First Post:
2010-02-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007926', 'term': 'Lesch-Nyhan Syndrome'}, {'id': 'D016728', 'term': 'Self-Injurious Behavior'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D011686', 'term': 'Purine-Pyrimidine Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D001526', 'term': 'Behavioral Symptoms'}, {'id': 'D001519', 'term': 'Behavior'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C058081', 'term': 'ecopipam'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'rchipkin@psyadonrx.com', 'phone': '301-919-2020', 'title': 'Dr. Richard Chipkin', 'organization': 'Psyadon Pharmaceuticals'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': "This was an open label trial of oral ecopipam in pediatric, adolescent and adult patients with Lesch-Nyhan Disease.In this population, ecopipam's safety and PK was similar to non-LND subjects."}}, 'adverseEventsModule': {'timeFrame': '12 days of active treatment and 28 days follow-up', 'eventGroups': [{'id': 'EG000', 'title': 'Ecopipam Treated Patients', 'otherNumAtRisk': 5, 'otherNumAffected': 5, 'seriousNumAtRisk': 5, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Sedation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'Dystonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}], 'seriousEvents': [{'term': 'Flank Pain', 'notes': 'Patient reported flank pain which was caused by a kidney stone. Investigator defined as not drug related, and resolved without incident. Patient received all doses and was not removed from study.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'Respiratory Infection', 'notes': 'Patient developed respiratory infection (pneumonia)following the trial. Investigator reported this as not related to ecopipam administration. Patient was briefly hosptialized and infection resolved.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Clinically Significant Changes in Standard Laboratory Tests', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ecopipam Treated Patients', 'description': 'These were patients who had diagnoses Lesch-Nyhan Disease based either on their genetic changes or on changes of specific enzymes in their blood.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Two weeks', 'description': "This study's primary outcome is the safety of ecopipam in Lesch-Nyhan patients as measured by standard clinical laboratory tests. The patients will also be observed and questioned about other side effects, such as whether they feel more or less tired.Standard clinical laboratory tests for liver, kidney and blood function were conducted. The normal ranges for each of these tests were different and are too numerous to be individually listed here. However, if any individual value were to be either three-times greater or lesser than the upper or the lower limit of the test, then that value was considered to have been changed.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All patients were analyzed'}, {'type': 'SECONDARY', 'title': 'Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ecopipam Treated Patients', 'description': 'These were patients who had diagnoses Lesch-Nyhan Disease based either on their genetic changes or on changes of specific enzymes in their blood.'}], 'classes': [{'categories': [{'measurements': [{'value': '22.5', 'spread': '13.4', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Screening visit and end of study (two weeks)', 'description': "Change in the self-injurious subscale of the Behavior Problems Inventory (BPI). the BPI is a well-validate test to evaluate the frequency and severity of a patient's self-injurious behavior. Values range from 0 to 50, and a low score means few/less severe behaviors", 'unitOfMeasure': 'Change in BPI score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All patients who received any dose of ecopipam were in the analysis population'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ecopipam (12.5- 200 mg/Day)', 'description': 'Patients were given ecopipapm over an 11 day period as follows:\n\nday 1 12.5 mg/day day 2-3 25 mg/day day 4-5 50 mg/day day 6-9 100 mg/day day 9-11 200 mg/day\n\nNote: Doses were reduced as necessary to a previously tolerated dose if paitents reached doses that were not safely tolerable.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Patients were recruited by the investigators according to the inclusion and exclusion criteria', 'preAssignmentDetails': 'No preassignment'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ecopipam'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age Continuous', 'classes': [{'categories': [{'measurements': [{'value': '20.4', 'spread': '17.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Lesch-Nyhan patients with moderate to severe self-injurious behaviors at the start of the study'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-04', 'completionDateStruct': {'date': '2011-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-04-17', 'studyFirstSubmitDate': '2010-02-06', 'resultsFirstSubmitDate': '2012-12-18', 'studyFirstSubmitQcDate': '2010-02-08', 'lastUpdatePostDateStruct': {'date': '2013-04-18', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2013-04-17', 'studyFirstPostDateStruct': {'date': '2010-02-09', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2013-04-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Clinically Significant Changes in Standard Laboratory Tests', 'timeFrame': 'Two weeks', 'description': "This study's primary outcome is the safety of ecopipam in Lesch-Nyhan patients as measured by standard clinical laboratory tests. The patients will also be observed and questioned about other side effects, such as whether they feel more or less tired.Standard clinical laboratory tests for liver, kidney and blood function were conducted. The normal ranges for each of these tests were different and are too numerous to be individually listed here. However, if any individual value were to be either three-times greater or lesser than the upper or the lower limit of the test, then that value was considered to have been changed."}], 'secondaryOutcomes': [{'measure': 'Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening', 'timeFrame': 'Screening visit and end of study (two weeks)', 'description': "Change in the self-injurious subscale of the Behavior Problems Inventory (BPI). the BPI is a well-validate test to evaluate the frequency and severity of a patient's self-injurious behavior. Values range from 0 to 50, and a low score means few/less severe behaviors"}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Ecopipam', 'Lesch-Nyhan Disease', 'Safety', 'Self-Injurious Behavior'], 'conditions': ['Lesch-Nyhan Disease']}, 'referencesModule': {'references': [{'pmid': '2905002', 'type': 'BACKGROUND', 'citation': 'Chipkin RE, Iorio LC, Coffin VL, McQuade RD, Berger JG, Barnett A. Pharmacological profile of SCH39166: a dopamine D1 selective benzonaphthazepine with potential antipsychotic activity. J Pharmacol Exp Ther. 1988 Dec;247(3):1093-102.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam does this by stopping the chemical (dopamine) from binding to one of its family of receptors (i.e, the D1 family). LND is a very rare genetic disease. The sponsor is doing this study to find out what side effects ecopipam causes in LND patients, and whether ecopipam may be able to relieve the self-injurious behaviors seen in these patients.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria\n\n* Diagnosis of Lesch-Nyhan disease with moderate to severe Self- Injurious Behavior and are routinely restrained\n* Male - 6 years of age and above\n* Body weight greater than or equal to 44 pounds\n\nExclusion Criteria:\n\n* Taking Neuroleptic or Dopamine Depleting medications\n* Currently treated with medications for depression or seizures\n* Impaired renal function\n* Medical conditions that may interfere with completing the study'}, 'identificationModule': {'nctId': 'NCT01065558', 'briefTitle': 'Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Psyadon Pharma'}, 'officialTitle': 'A Safety and Pilot Activity Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease', 'orgStudyIdInfo': {'id': 'Ecopipam and Lesch-Nyhan'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ecopipam 12.5 - 200 mg/day', 'description': 'Patients were administered ecopipam on an escalated dosing schedule over 11 days starting at 12.5 mg/day and increasing to the maximal tolerated dose or to 200 mg/day.', 'interventionNames': ['Drug: Ecopipam']}], 'interventions': [{'name': 'Ecopipam', 'type': 'DRUG', 'otherNames': ['PSYRX101, SCH 39166'], 'description': 'Tablets, once daily, for two weeks up to 200 mg/day', 'armGroupLabels': ['Ecopipam 12.5 - 200 mg/day']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92103', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 'University of California at San Diego', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '30329', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory Univesity', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}], 'overallOfficials': [{'name': 'Hyder A Jinnah, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Emory University'}, {'name': 'William L Nyhan, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of California, San Diego'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Psyadon Pharma', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}