Ignite Creation Date:
2025-12-24 @ 7:24 PM
Ignite Modification Date:
2026-04-13 @ 9:35 PM
Study NCT ID:
NCT01767103
Status:
COMPLETED
Last Update Posted:
2014-09-10
First Post:
2013-01-09
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild and Moderate Hepatic Insufficiency and Healthy Volunteers
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008107', 'term': 'Liver Diseases'}], 'ancestors': [{'id': 'D004066', 'term': 'Digestive System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077543', 'term': 'Deferiprone'}, {'id': 'D007531', 'term': 'Isoflurophate'}, {'id': 'D020084', 'term': 'Long Interspersed Nucleotide Elements'}], 'ancestors': [{'id': 'D011728', 'term': 'Pyridones'}, {'id': 'D011725', 'term': 'Pyridines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D063066', 'term': 'Organofluorophosphonates'}, {'id': 'D063065', 'term': 'Organophosphonates'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D018626', 'term': 'Retroelements'}, {'id': 'D020071', 'term': 'Interspersed Repetitive Sequences'}, {'id': 'D012091', 'term': 'Repetitive Sequences, Nucleic Acid'}, {'id': 'D001483', 'term': 'Base Sequence'}, {'id': 'D015394', 'term': 'Molecular Structure'}, {'id': 'D001669', 'term': 'Biochemical Phenomena'}, {'id': 'D055598', 'term': 'Chemical Phenomena'}, {'id': 'D040342', 'term': 'Genetic Structures'}, {'id': 'D055614', 'term': 'Genetic Phenomena'}, {'id': 'D040481', 'term': 'Genome Components'}, {'id': 'D016678', 'term': 'Genome'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ftricta@apopharma.com', 'phone': '416-401-7332', 'title': 'Fernando Tricta, MD', 'organization': 'ApoPharma Inc.'}, 'certainAgreement': {'otherDetails': "Sponsor retained title to and the right to publish all documentation, records, raw data, specimens or other work product generated in connection with the trial. Such publications shall not be made without the prior written consent of Sponsor. Neither Party will use the other Party's name in connection with any publication or promotion without the other Party's prior written consent. However, Sponsor has the right to publish appropriate information in order to satisfy regulatory requirements.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '2 days', 'description': 'Safety data were collected from the time of dosing up to the follow-up visit 48 hours post-dose', 'eventGroups': [{'id': 'EG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function', 'otherNumAtRisk': 7, 'otherNumAffected': 0, 'seriousNumAtRisk': 7, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by the Child-Pugh Class C: 5-6 points', 'otherNumAtRisk': 7, 'otherNumAffected': 1, 'seriousNumAtRisk': 7, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points', 'otherNumAtRisk': 7, 'otherNumAffected': 3, 'seriousNumAtRisk': 7, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}, {'term': 'Gout', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}], 'seriousEvents': [{'term': 'Liver injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}, {'term': 'Renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.1)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points'}], 'classes': [{'title': 'Cmax for serum deferiprone', 'categories': [{'measurements': [{'value': '49.00', 'spread': '24.081', 'groupId': 'OG000'}, {'value': '36.26', 'spread': '8.4799', 'groupId': 'OG001'}, {'value': '35.94', 'spread': '7.8407', 'groupId': 'OG002'}]}]}, {'title': 'Cmax for serum deferiprone 3-O-glucuronide', 'categories': [{'measurements': [{'value': '69.03', 'spread': '9.9153', 'groupId': 'OG000'}, {'value': '56.91', 'spread': '11.416', 'groupId': 'OG001'}, {'value': '55.33', 'spread': '25.335', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24-hour interval', 'description': 'Cmax was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.', 'unitOfMeasure': 'ug/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK Analysis Set consisted of all subjects who had sufficient data to derive at least one PK parameter'}, {'type': 'SECONDARY', 'title': 'Safety and Tolerability of Ferriprox in Subjects With or Without Hepatic Impairment.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Time of dosing until 48 hours post-dose', 'description': 'The number of participants who experienced adverse events following a single dose of Ferriprox, between the time of dosing and the follow-up visit (including any changes of clinical significance in physical examinations, vital signs, 12-lead ECG, and clinical laboratory tests).', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Analysis Set consisted of all subjects who received study medication and had at least one safety assessment'}, {'type': 'PRIMARY', 'title': 'Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by Child-Pugh Class B: 7-9 points'}], 'classes': [{'title': 'Tmax of deferiprone', 'categories': [{'measurements': [{'value': '0.7500', 'groupId': 'OG000', 'lowerLimit': '0.500', 'upperLimit': '2.00'}, {'value': '0.7500', 'groupId': 'OG001', 'lowerLimit': '0.500', 'upperLimit': '1.00'}, {'value': '0.7500', 'groupId': 'OG002', 'lowerLimit': '0.500', 'upperLimit': '1.33'}]}]}, {'title': 'Tmax of deferiprone 3-O-glucuronide', 'categories': [{'measurements': [{'value': '2.000', 'groupId': 'OG000', 'lowerLimit': '1.00', 'upperLimit': '3.00'}, {'value': '3.000', 'groupId': 'OG001', 'lowerLimit': '1.67', 'upperLimit': '4.00'}, {'value': '3.000', 'groupId': 'OG002', 'lowerLimit': '3.00', 'upperLimit': '4.00'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': '24-hour interval', 'description': 'Tmax was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.', 'unitOfMeasure': 'hour', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK Analysis Set consisted of all subjects who had sufficient data to derive at least one PK parameter'}, {'type': 'PRIMARY', 'title': 'AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points'}], 'classes': [{'title': 'AUC0-∞ for serum deferiprone', 'categories': [{'measurements': [{'value': '92.64', 'spread': '19.221', 'groupId': 'OG000'}, {'value': '82.02', 'spread': '28.213', 'groupId': 'OG001'}, {'value': '105.5', 'spread': '41.684', 'groupId': 'OG002'}]}]}, {'title': 'AUC0-∞ for serum deferiprone 3-O-glucuronide', 'categories': [{'measurements': [{'value': '337.3', 'spread': '45.343', 'groupId': 'OG000'}, {'value': '305.1', 'spread': '53.651', 'groupId': 'OG001'}, {'value': '305.9', 'spread': '111.75', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24-hour interval', 'description': 'AUC (area under the curve) from zero to infinity was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.', 'unitOfMeasure': 'ug*hr/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK Analysis Set consisted of all subjects who had sufficient data to derive at least one PK parameter'}, {'type': 'PRIMARY', 'title': 'T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points'}], 'classes': [{'title': 'T1/2 for serum deferiprone', 'categories': [{'measurements': [{'value': '1.994', 'spread': '0.26971', 'groupId': 'OG000'}, {'value': '1.855', 'spread': '0.45370', 'groupId': 'OG001'}, {'value': '2.180', 'spread': '0.83153', 'groupId': 'OG002'}]}]}, {'title': 'T1/2 for deferiprone 3-O-glu', 'categories': [{'measurements': [{'value': '2.621', 'spread': '0.35653', 'groupId': 'OG000'}, {'value': '2.518', 'spread': '0.55679', 'groupId': 'OG001'}, {'value': '2.479', 'spread': '0.67212', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24-hour interval', 'description': 'T1/2 was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.', 'unitOfMeasure': 'hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK Analysis Set consisted of all subjects who had sufficient data to derive at least one PK parameter'}, {'type': 'PRIMARY', 'title': 'CumAe for Urine Deferiprone and Deferiprone 3-O-glucuronide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points'}], 'classes': [{'title': 'CumAe of urine deferiprone', 'categories': [{'measurements': [{'value': '69.0', 'spread': '25.9', 'groupId': 'OG000'}, {'value': '59.5', 'spread': '19.4', 'groupId': 'OG001'}, {'value': '62.9', 'spread': '31.1', 'groupId': 'OG002'}]}]}, {'title': 'CumAe of urine deferiprone 3-O-glucuronide', 'categories': [{'measurements': [{'value': '6670', 'spread': '2520', 'groupId': 'OG000'}, {'value': '6787', 'spread': '619', 'groupId': 'OG001'}, {'value': '5979', 'spread': '887', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24-hour interval', 'description': "Cumulative amount of deferiprone and deferiprone 3-O-glucuronide excreted in the urine. Urine samples were collected at the intervals of -2 to 0 hours pre-dose, and 0-2, 2-4, 4-8, 8-12 ,and 12- 24 hours post-dose.\n\nNote: For unknown reasons, the urine samples for one subject in the moderate hepatic failure group had low or zero volume and there were no measurable levels of deferiprone or its metabolite in any of the samples. Accordingly, the urine PK results were derived both with and without this subject's data, and are here presented without them (i.e., N=6 rather than 7).", 'unitOfMeasure': 'mg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK Analysis Set consisted of all subjects who had sufficient data to derive at least one PK parameter. The data of one subject in the moderate hepatic failure group were dropped due to low or zero volume of urine samples.'}, {'type': 'PRIMARY', 'title': 'Fe% for Urine Deferiprone and Deferiprone 3-O-glucuronide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'OG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'OG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points'}], 'classes': [{'title': 'Fe of urine deferiprone', 'categories': [{'measurements': [{'value': '2.56', 'spread': '0.773', 'groupId': 'OG000'}, {'value': '2.18', 'spread': '0.669', 'groupId': 'OG001'}, {'value': '2.36', 'spread': '1.20', 'groupId': 'OG002'}]}]}, {'title': 'Fe of urine 3-O-glucuronide', 'categories': [{'measurements': [{'value': '109', 'spread': '32.4', 'groupId': 'OG000'}, {'value': '110', 'spread': '14.0', 'groupId': 'OG001'}, {'value': '97.7', 'spread': '13.7', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24-hour interval', 'description': "Cumulative fraction of Ferriprox dose excreted in urine as deferiprone or deferiprone 3-O-glucuronide. Urine samples were collected at the intervals of -2 to 0 hours pre-dose, and 0-2, 2-4, 4-8, 8-12 ,and 12- 24 hours post-dose.\n\nNote: For unknown reasons, the urine samples for one subject in the moderate hepatic failure group had low or zero volume and there were no measurable levels of deferiprone or its metabolite in any of the samples. Accordingly, the urine PK results were derived both with and without this subject's data, and are here presented without them (i.e., N=6 rather than 7).", 'unitOfMeasure': 'percentage of dose excreted in urine', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK Analysis Set consisted of all subjects who had sufficient data to derive at least one PK parameter. The data of one subject in the moderate hepatic failure group were dropped due to low or zero volume of urine samples.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function. All subjects received a single 33 mg/kg oral dose of deferiprone.'}, {'id': 'FG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic impairment as defined by Child-Pugh Class C: 5-6 points. All subjects received a single 33 mg/kg oral dose of deferiprone.'}, {'id': 'FG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic impairment as defined by Child-Pugh Class : 7-9 points. All subjects received a single 33 mg/kg oral dose of deferiprone.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '7'}, {'groupId': 'FG002', 'numSubjects': '7'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '7'}, {'groupId': 'FG002', 'numSubjects': '7'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '21', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Normal Hepatic Function (Healthy Volunteers)', 'description': 'Healthy volunteers with normal hepatic function'}, {'id': 'BG001', 'title': 'Mild Hepatic Failure', 'description': 'Mild hepatic failure as defined by Child-Pugh Class C: 5-6 points'}, {'id': 'BG002', 'title': 'Moderate Hepatic Failure', 'description': 'Moderate hepatic failure as defined by Child-Pugh Class B: 7-9 points'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '20', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '16', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}, {'value': '19', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '21', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 21}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-04', 'completionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-09-03', 'studyFirstSubmitDate': '2013-01-09', 'resultsFirstSubmitDate': '2014-09-03', 'studyFirstSubmitQcDate': '2013-01-10', 'lastUpdatePostDateStruct': {'date': '2014-09-10', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-09-03', 'studyFirstPostDateStruct': {'date': '2013-01-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-09-10', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'timeFrame': '24-hour interval', 'description': 'Cmax was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.'}, {'measure': 'Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'timeFrame': '24-hour interval', 'description': 'Tmax was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.'}, {'measure': 'AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'timeFrame': '24-hour interval', 'description': 'AUC (area under the curve) from zero to infinity was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.'}, {'measure': 'T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide', 'timeFrame': '24-hour interval', 'description': 'T1/2 was assessed over a 24-hour interval for analyses of deferiprone and its 3-O-glucuronide metabolite in subjects with normal hepatic function, mild hepatic impairment, or moderate hepatic impairment. Blood samples were obtained prior to dosing and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 9, 12, 16, and 24 hours post-dose.'}, {'measure': 'CumAe for Urine Deferiprone and Deferiprone 3-O-glucuronide', 'timeFrame': '24-hour interval', 'description': "Cumulative amount of deferiprone and deferiprone 3-O-glucuronide excreted in the urine. Urine samples were collected at the intervals of -2 to 0 hours pre-dose, and 0-2, 2-4, 4-8, 8-12 ,and 12- 24 hours post-dose.\n\nNote: For unknown reasons, the urine samples for one subject in the moderate hepatic failure group had low or zero volume and there were no measurable levels of deferiprone or its metabolite in any of the samples. Accordingly, the urine PK results were derived both with and without this subject's data, and are here presented without them (i.e., N=6 rather than 7)."}, {'measure': 'Fe% for Urine Deferiprone and Deferiprone 3-O-glucuronide', 'timeFrame': '24-hour interval', 'description': "Cumulative fraction of Ferriprox dose excreted in urine as deferiprone or deferiprone 3-O-glucuronide. Urine samples were collected at the intervals of -2 to 0 hours pre-dose, and 0-2, 2-4, 4-8, 8-12 ,and 12- 24 hours post-dose.\n\nNote: For unknown reasons, the urine samples for one subject in the moderate hepatic failure group had low or zero volume and there were no measurable levels of deferiprone or its metabolite in any of the samples. Accordingly, the urine PK results were derived both with and without this subject's data, and are here presented without them (i.e., N=6 rather than 7)."}], 'secondaryOutcomes': [{'measure': 'Safety and Tolerability of Ferriprox in Subjects With or Without Hepatic Impairment.', 'timeFrame': 'Time of dosing until 48 hours post-dose', 'description': 'The number of participants who experienced adverse events following a single dose of Ferriprox, between the time of dosing and the follow-up visit (including any changes of clinical significance in physical examinations, vital signs, 12-lead ECG, and clinical laboratory tests).'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Hepatic Impairment', 'Liver Impairment', 'Liver Disease', 'Ferriprox®', 'LI', 'DFP', 'Deferiprone'], 'conditions': ['Hepatic Impairment']}, 'descriptionModule': {'briefSummary': 'Multi-center, non-randomized, open-label, single-dose, parallel group study to determine the effect of impaired hepatic function on the PK of deferiprone and its 3-O-glucuronide metabolite following a single oral dose of 33mg/kg Ferriprox®.', 'detailedDescription': 'Post-marketing study to evaluate the effect of impaired hepatic function on the pharmacokinetics (PK) of deferiprone and its 3-O-glucuronide metabolite and on the safety of Ferriprox® in subjects with mild and moderate hepatic impairment as compared to healthy volunteers.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Main Inclusion Criteria:\n\nAll subjects:\n\n1. Adult males or females, 18 - 75 years of age (inclusive);\n2. Body weight ≥ 50 kg;\n3. Body mass index (BMI) between 19 and 32 kg/mE2 (inclusive);\n4. Absolute neutrophil count (ANC) of \\>1.5x10E9/L ;\n\nHealthy volunteers:\n\n1. Medically healthy with clinically insignificant screening results (e.g., laboratory profiles, medical history, vital signs, physical examination);\n2. Matched to hepatically impaired subjects in the study by age (+/- 10 years), sex and weight (+/- 15% BMI).\n\nHepatically impaired subjects:\n\n1. Considered clinically stable in the opinion of the Investigator;\n2. Subjects with different degrees of impaired hepatic function as assessed by a Child-Pugh classification score: mild (Class A: 5-6 points) and moderate (Class B: 7-9 points) impaired hepatic function.\n\nMain Exclusion Criteria:\n\n1. For subjects with hepatic impairment, fluctuating or rapidly deteriorating hepatic function as indicated by clinical and/or laboratory signs of hepatic impairment (e.g. advanced ascites, infection of ascites, fever, or active gastrointestinal bleeding).\n2. Evidence of liver impairment in healthy volunteers: hepatitis B and C; or aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin, clotting factors, serum protein that is considered clinically significant by the Investigator;\n3. History or presence of significant clinically unstable respiratory, cardiovascular, pulmonary, hepatic (except for subjects assigned to one of the hepatically impaired groups), renal, hematologic, gastrointestinal, endocrine (except for subjects with hepatic impairment with clinically stable and treated diabetes, hypertension and thyroid disorders), immunologic, dermatologic, neurologic, or psychiatric disease;\n4. Disorders or surgery of the gastrointestinal tract which may interfere with drug absorption or may otherwise influence the PK of the investigational medicinal product (e.g. resections of the small or large intestine, febrile conditions, chronic diarrhea, chronic vomiting, clinically unstable endocrine disease, severe infections, acute inflammations, etc.);\n5. Received a pharmacological agent in another clinical trial within 28 days prior to the first dose of the study;'}, 'identificationModule': {'nctId': 'NCT01767103', 'briefTitle': 'An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild and Moderate Hepatic Insufficiency and Healthy Volunteers', 'organization': {'class': 'INDUSTRY', 'fullName': 'ApoPharma'}, 'officialTitle': 'An Open-Label Study to Compare the Pharmacokinetic Profiles of a Single Dose of Ferriprox® in Subjects With Impaired Hepatic Function and Healthy Volunteers', 'orgStudyIdInfo': {'id': 'LA40-0412'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Normal Hepatic Function (healthy volunteers)', 'description': 'Healthy volunteers with normal hepatic function received a single 33 mg/kg dose of Ferriprox®.', 'interventionNames': ['Drug: Ferriprox®']}, {'type': 'EXPERIMENTAL', 'label': 'Mild Hepatic Failure', 'description': 'Subjects with mild hepatic failure as defined by the Child-Pugh Class C: 5-6 points received a single 33 mg/kg dose of Ferriprox®.', 'interventionNames': ['Drug: Ferriprox®']}, {'type': 'EXPERIMENTAL', 'label': 'Moderate Hepatic Failure', 'description': 'Subjects with moderate hepatic failure as defined by the Child-Pugh Class B: 7-9 points received a single 33 mg/kg dose of Ferriprox®.', 'interventionNames': ['Drug: Ferriprox®']}], 'interventions': [{'name': 'Ferriprox®', 'type': 'DRUG', 'otherNames': ['DFP', 'Deferiprone', 'L1'], 'armGroupLabels': ['Mild Hepatic Failure', 'Moderate Hepatic Failure', 'Normal Hepatic Function (healthy volunteers)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '32809', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Orlando Clinical Research Center', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}], 'overallOfficials': [{'name': 'Fernando Tricta, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'ApoPharma'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'ApoPharma', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}