Ignite Creation Date:
2025-12-25 @ 12:53 AM
Ignite Modification Date:
2025-12-26 @ 1:39 PM
Study NCT ID:
NCT00999167
Status:
COMPLETED
Last Update Posted:
2024-07-10
First Post:
2009-10-08
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy
Sponsor:
Organization:
Raw JSON
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'HPN-100 BID', 'description': '6 mL or 9 mL BID HPN-100 (Part A)'}], 'classes': [{'title': 'Any AE', 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': 'Gastrointestinal disorders', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Metabolism and nutrition disorders', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Infection and infestations', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Nervous system disorders', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Blood and lymphatic system disorders', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Injury, poisoning and procedural complications', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Musculoskeletal and connective tissue disorders', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Psychiatric disorders', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Any SAE', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Death', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Part A: 28 days', 'description': 'Part A: The rate of AEs and tolerability of 6 mL and 9 mL doses of HPN-100 were considered the primary safety endpoints for Part A. Safety assessments included adverse events, laboratory tests (including ammonia, hematology, coagulation, liver function and serum chemistry parameters), vital signs, physical and neurological examinations, and electrocardiograms.', 'unitOfMeasure': 'Subjects', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}, {'type': 'SECONDARY', 'title': 'Total Number of HE Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'OG000'}, {'value': '88', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'HPN-100', 'description': '6 mL BID'}, {'id': 'OG001', 'title': 'Placebo', 'description': '6 mL BID'}], 'classes': [{'categories': [{'measurements': [{'value': '35', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0354', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'statisticalMethod': 'Poisson regression adjusting for country', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'NUMBER', 'timeFrame': '112 Days', 'description': 'Secondary efficacy endpoint. The total number of HE events during the treatment phase for subjects in the placebo and active arms.', 'unitOfMeasure': 'HE event', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat (ITT)'}, {'type': 'PRIMARY', 'title': 'Part B: Proportion of Subjects Who Exhibit an HE Episode, Defined as Either of the Following During the Treatment Phase: WH ≥2; WH Grade and Asterixis Grade Increase of 1 Each, if Baseline WH = 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'OG000'}, {'value': '88', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'HPN-100', 'description': 'HPN-100 6 mL BID (Part B)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo 6 mL BID (Part B)'}], 'classes': [{'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}, {'value': '32', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0214', 'groupIds': ['OG000', 'OG001'], 'ciPctValue': '95', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'NUMBER', 'timeFrame': 'Part B: 112 Days', 'description': 'An HE event was defined as occurrences of either a West Haven (WH) Grade ≥2 or a WH Grade 1 and asterixis grade increase of 1 (if baseline WH = 0).\n\nThe WH criteria are widely used for rating the severity of HE and are summarized below:\n\nGrade 1: trivial lack of awareness, euphoria or anxiety, shortened attention span, impaired performance of addition Grade 2: lethargy or apathy, minimal disorientation for time or place, subtle personality change, inappropriate behavior, impaired performance of subtraction Grade 3: somnolence to semi-stupor but responsive to verbal stimuli, confusion, gross disorientation Grade 4: coma (unresponsive to verbal or noxious stimuli)\n\nAsterixis was assessed after arm and forearm extension along with wrist dorsiflexion for 30 seconds and assigned a grade according to the following criteria:\n\nGrade 1: rare flaps Grade 2: occasional irregular flaps Grade 3: frequent flaps Grade 4: continuous flaps', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to Treat (ITT)'}, {'type': 'SECONDARY', 'title': 'Time to Meeting the Primary Endpoint', 'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'OG000'}, {'value': '88', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'HPN-100', 'description': 'HPN-100 6 mL BID (Part B)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo 6 mL BID (Part B)'}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'Data are based on Kaplan-Meier point estimates. Because of low event rate (21.1%) it is not possible to estimate the median.', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'Data are based on Kaplan-Meier point estimates. Because of low event rate (36.4%) it is not possible to estimate the median', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'analyses': [{'pValue': '0.047', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Hazard Ratio (HR)', 'ciPctValue': '95', 'paramValue': '0.56', 'statisticalMethod': 'Regression, Cox', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'MEDIAN', 'timeFrame': '112 Days', 'description': 'Secondary efficacy endpoint. The time to the first HE episode during the treatment period was calculated using the Kaplan-Meier method. Subjects who did not experience an HE episode were censored at the time of their last asterixis assessment. Subjects who had no post-randomization data for the primary endpoint were considered to have an HE episode at Day 1.', 'unitOfMeasure': 'Days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat (ITT)'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'OG000'}, {'value': '88', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'HPN-100', 'description': 'HPN-100 6 mL BID (Part B)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo 6 mL BID (Part B)'}], 'classes': [{'title': 'Change from Baseline to D56 (Total Score)', 'categories': [{'measurements': [{'value': '-0.5', 'spread': '1.54', 'groupId': 'OG000'}, {'value': '3.2', 'spread': '1.37', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline to Final Visit (Total Score)', 'categories': [{'measurements': [{'value': '-10.7', 'spread': '2.85', 'groupId': 'OG000'}, {'value': '-9.7', 'spread': '3.33', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0713', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'groupDescription': 'Changes in the Total Index Score from Baseline and Day 56.', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}, {'pValue': '0.2520', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'groupDescription': 'Changes in the Total Index Score from Baseline and the Final Visit.', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Day 56, Final Visit (D112)', 'description': 'Changes from Baseline to Day 56 and the Final Visit were compared between treatment groups using an ANCOVA model for the total index RBANS score ). The index score is a sum of the scores for each of the 5 individual domains (immediate memory, visuospatial/constructional, language, attention). The minimum and maximum total index scores are 40 and 160, respectively; a higher score is better.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat (ITT)'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'HPN-100 6 mL and 9 mL', 'description': 'Subjects will undergo a one step dose escalation over 4 weeks. Subjects will initially receive 6 mL HPN-100 BID for 1 week. On Day 7 and following a satisfactory safety assessment of the subject, the dose will be escalated to 9 mL BID for an additional 3 weeks.'}, {'id': 'FG001', 'title': 'HPN-100 6 mL', 'description': 'Subjects will receive 6 mL BID HPN-100 for 16 weeks (Part B)'}, {'id': 'FG002', 'title': 'Placebo', 'description': 'Subjects will receive 6 mL BID placebo for 16 weeks (Part B)'}], 'periods': [{'title': 'Part A: Open Label Safety run-in', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Received 9 mL BID', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Met protocol defined stopping rule', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}, {'title': 'Part B: Randomized, Placebo Controlled', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'comment': '4 subjects who participated in Part A enrolled in Part B and remainder were newly enrolled', 'groupId': 'FG001', 'numSubjects': '90'}, {'groupId': 'FG002', 'numSubjects': '88'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '55'}, {'groupId': 'FG002', 'numSubjects': '67'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '35'}, {'groupId': 'FG002', 'numSubjects': '21'}]}], 'dropWithdraws': [{'type': 'Met protocol defined stopping rule', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '19'}, {'groupId': 'FG002', 'numSubjects': '16'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '7'}, {'groupId': 'FG002', 'numSubjects': '4'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '6'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Noncompliance', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Physician stopped drug', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Part A enrollment: 01 December 2009 to 24 February 2010 Part B enrollment: 01 June 2010 to 31 October 2011', 'preAssignmentDetails': 'The study consisted of Part A, an open-label, dose-escalation lead-in to assess HPN-100 safety and PK, followed by Part B, a randomized, placebo controlled study to assess safety and efficacy of HPN-100.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'BG000'}, {'value': '88', 'groupId': 'BG001'}, {'value': '178', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'HPN-100', 'description': '6 mL BID'}, {'id': 'BG001', 'title': 'Placebo', 'description': '6 mL BID'}, {'id': 'BG002', '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'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '85', 'groupId': 'BG000'}, {'value': '78', 'groupId': 'BG001'}, {'value': '163', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '53.8', 'spread': '8.94', 'groupId': 'BG000'}, {'value': '55.4', 'spread': '8.85', 'groupId': 'BG001'}, {'value': '54.6', 'spread': '8.85', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '45', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '74', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '45', 'groupId': 'BG000'}, {'value': '59', 'groupId': 'BG001'}, {'value': '104', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '44', 'groupId': 'BG000'}, {'value': '44', 'groupId': 'BG001'}, {'value': '88', 'groupId': 'BG002'}]}]}, {'title': 'Russian Federation', 'categories': [{'measurements': [{'value': '26', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '50', 'groupId': 'BG002'}]}]}, {'title': 'Ukraine', 'categories': [{'measurements': [{'value': '20', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '40', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Only the baseline characteristics of the subjects enrolled in Part B are presented.'}}, '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': 189}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-06', 'completionDateStruct': {'date': '2012-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-06-18', 'studyFirstSubmitDate': '2009-10-08', 'resultsFirstSubmitDate': '2015-07-01', 'studyFirstSubmitQcDate': '2009-10-20', 'lastUpdatePostDateStruct': {'date': '2024-07-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2015-08-27', 'studyFirstPostDateStruct': {'date': '2009-10-21', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-09-30', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A: The Rate of AEs and Tolerability of HPN-100', 'timeFrame': 'Part A: 28 days', 'description': 'Part A: The rate of AEs and tolerability of 6 mL and 9 mL doses of HPN-100 were considered the primary safety endpoints for Part A. Safety assessments included adverse events, laboratory tests (including ammonia, hematology, coagulation, liver function and serum chemistry parameters), vital signs, physical and neurological examinations, and electrocardiograms.'}, {'measure': 'Part B: Proportion of Subjects Who Exhibit an HE Episode, Defined as Either of the Following During the Treatment Phase: WH ≥2; WH Grade and Asterixis Grade Increase of 1 Each, if Baseline WH = 0', 'timeFrame': 'Part B: 112 Days', 'description': 'An HE event was defined as occurrences of either a West Haven (WH) Grade ≥2 or a WH Grade 1 and asterixis grade increase of 1 (if baseline WH = 0).\n\nThe WH criteria are widely used for rating the severity of HE and are summarized below:\n\nGrade 1: trivial lack of awareness, euphoria or anxiety, shortened attention span, impaired performance of addition Grade 2: lethargy or apathy, minimal disorientation for time or place, subtle personality change, inappropriate behavior, impaired performance of subtraction Grade 3: somnolence to semi-stupor but responsive to verbal stimuli, confusion, gross disorientation Grade 4: coma (unresponsive to verbal or noxious stimuli)\n\nAsterixis was assessed after arm and forearm extension along with wrist dorsiflexion for 30 seconds and assigned a grade according to the following criteria:\n\nGrade 1: rare flaps Grade 2: occasional irregular flaps Grade 3: frequent flaps Grade 4: continuous flaps'}], 'secondaryOutcomes': [{'measure': 'Total Number of HE Events', 'timeFrame': '112 Days', 'description': 'Secondary efficacy endpoint. The total number of HE events during the treatment phase for subjects in the placebo and active arms.'}, {'measure': 'Time to Meeting the Primary Endpoint', 'timeFrame': '112 Days', 'description': 'Secondary efficacy endpoint. The time to the first HE episode during the treatment period was calculated using the Kaplan-Meier method. Subjects who did not experience an HE episode were censored at the time of their last asterixis assessment. Subjects who had no post-randomization data for the primary endpoint were considered to have an HE episode at Day 1.'}, {'measure': 'Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score', 'timeFrame': 'Day 56, Final Visit (D112)', 'description': 'Changes from Baseline to Day 56 and the Final Visit were compared between treatment groups using an ANCOVA model for the total index RBANS score ). The index score is a sum of the scores for each of the 5 individual domains (immediate memory, visuospatial/constructional, language, attention). The minimum and maximum total index scores are 40 and 160, respectively; a higher score is better.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Cirrhosis', 'Episodic Hepatic Encephalopathy', 'HE', 'HPN-100', 'GT4P', 'glycerol phenylbutyrate'], 'conditions': ['Cirrhosis', 'Hepatic Encephalopathy']}, 'referencesModule': {'references': [{'pmid': '23847109', 'type': 'RESULT', 'citation': "Rockey DC, Vierling JM, Mantry P, Ghabril M, Brown RS Jr, Alexeeva O, Zupanets IA, Grinevich V, Baranovsky A, Dudar L, Fadieienko G, Kharchenko N, Klaryts'ka I, Morozov V, Grewal P, McCashland T, Reddy KG, Reddy KR, Syplyviy V, Bass NM, Dickinson K, Norris C, Coakley D, Mokhtarani M, Scharschmidt BF; HALT-HE Study Group. Randomized, double-blind, controlled study of glycerol phenylbutyrate in hepatic encephalopathy. Hepatology. 2014 Mar;59(3):1073-83. doi: 10.1002/hep.26611."}, {'pmid': '24144944', 'type': 'DERIVED', 'citation': 'Mokhtarani M, Diaz GA, Rhead W, Berry SA, Lichter-Konecki U, Feigenbaum A, Schulze A, Longo N, Bartley J, Berquist W, Gallagher R, Smith W, McCandless SE, Harding C, Rockey DC, Vierling JM, Mantry P, Ghabril M, Brown RS Jr, Dickinson K, Moors T, Norris C, Coakley D, Milikien DA, Nagamani SC, Lemons C, Lee B, Scharschmidt BF. Elevated phenylacetic acid levels do not correlate with adverse events in patients with urea cycle disorders or hepatic encephalopathy and can be predicted based on the plasma PAA to PAGN ratio. Mol Genet Metab. 2013 Dec;110(4):446-53. doi: 10.1016/j.ymgme.2013.09.017. Epub 2013 Oct 8.'}]}, 'descriptionModule': {'briefSummary': 'This is a phase 2 study of HPN-100 in subjects with hepatic encephalopathy (HE) consisting of an open label safety lead-in (Part A), followed by randomized, double-blind, placebo-controlled treatment (Part B).', 'detailedDescription': 'Part A: Open-label, dose-escalation lead-in to assess HPN-100 safety and PK Approximately 10 subjects with HE and cirrhosis classified as Child Pugh B or C will undergo a one-step dose escalation over 4 weeks. Subjects will initially receive 6 mL HPN-100 BID for 1 week. On Day 7 and following satisfactory safety assessment of the subject, the dose will be escalated to 9 mL BID for an additional 3 weeks.\n\nIn addition to a safety assessment, subjects will undergo 12-hour PK assessments on Days 7 and 28, with sampling at the following time points (relative to the first dose): 0 (pre-first daily dose of HPN-100), 2, 4, 8 (approximately 2 hours before the second daily dose of HPN 100), and 12 hours post-first dose (approximately 2 hours after the second daily dose of HPN-100). Additional PK samples will be collected on Days 8, 15, and 21 (at pre-first dose and 4 hours post-first dose).\n\nThe DSMB will review all safety information, including laboratory values, to determine if Part B may be initiated.\n\nSubjects enrolled in Part A may be eligible for Part B as long as they meet the eligibility criteria.\n\nPart B: Randomized, double-blind assessment of HPN-100 in HE subjects Subjects who meet all entry criteria and are judged to be compliant with their prescribed SOC will be eligible for randomization to receive either HPN-100 or matching placebo for 16 weeks. Efficacy will be assessed by the proportion of subjects experiencing episodes of HE, as well as by other outcome measures, including daily home assessments.\n\nStudy acquired from Horizon in 2024.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects aged 18 and over\n* Clinical diagnosis of cirrhosis of any cause\n* Potential to benefit from HE treatment\n* History of greater than or equal to 2 documented episodes of WH Grade 2 or more HE within the past 6 months, at least one of which occurred within the preceding 3 months\n* No change in other HE-specific medications within 1 week before randomization\n* Able to give informed consent and comply with study activities\n* Availability of at least one designated family member or caregiver who is capable of and willing to assume responsibility for facilitating subject compliance with study procedures\n* All females of childbearing age and all sexually active males must agree to use an acceptable method of contraception throughout the study.\n\nExclusion Criteria:\n\n* Use of any investigational drug within 30 days\n* Use of prohibited medications\n* Uncontrolled infection\n* Active GI bleeding or a history of GI bleeding requiring blood transfusion (\\> 2 units) within 3 months\n* Transjugular intrahepatic portosystemic shunt (TIPS) placement or revision within the past 90 days\n* Recreational drug use or alcohol consumption for subjects with a history of alcohol or drug abuse within 6 months\n* Lactating and/or pregnant females\n* Active malignancy\n* Clinically significant bowel disease, including obstruction, inflammatory bowel disease, or malabsorption\n* Expected to undergo transplantation within 6 months\n* Model for end-stage liver disease (MELD) score of \\> 25'}, 'identificationModule': {'nctId': 'NCT00999167', 'acronym': 'HALT-HE', 'briefTitle': 'A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amgen'}, 'officialTitle': 'A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of HPN-100 for Maintaining Remission in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy', 'orgStudyIdInfo': {'id': 'HPN-100-008'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HPN-100', 'interventionNames': ['Drug: HPN-100']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'HPN-100', 'type': 'DRUG', 'otherNames': ['GT4P'], 'description': 'Part B: 6 mL BID for 16 weeks.', 'armGroupLabels': ['HPN-100']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Part B: same as experimental arm', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University Medical Center, Division of Gastroenterology and Hepatology', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '20007', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': 'Georgetown University Hospital', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami / Center for Liver Diseases', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '33606', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'Tampa General Hospital', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '60637', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'The University of Chicago Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa Hospitals and Clinics', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '70112', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Tulane University Health Science Center', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '21201', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'University of Maryland', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '48202', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': 'Henry Ford Hospital / Department of 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States', 'facility': 'University of Virginia Health System', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}, {'zip': '53792', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'University of Wisconsin Hospital & Clinics', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'overallOfficials': [{'name': 'MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Amgen'}]}, 'ipdSharingStatementModule': {'url': 'https://www.amgen.com/datasharing', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'timeFrame': 'Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.', 'ipdSharing': 'YES', 'description': 'De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.', 'accessCriteria': 'Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amgen', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}