Ignite Creation Date:
2025-12-25 @ 1:23 AM
Ignite Modification Date:
2026-01-19 @ 2:04 PM
Study NCT ID:
NCT06431893
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-04-11
First Post:
2024-05-08
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006712', 'term': 'Homocystinuria'}], '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': 'D020138', 'term': 'Hyperhomocysteinemia'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2024-04-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-04-08', 'studyFirstSubmitDate': '2024-05-08', 'studyFirstSubmitQcDate': '2024-05-21', 'lastUpdatePostDateStruct': {'date': '2025-04-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-05-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Treatment-emergent AEs', 'timeFrame': 'Week 1 - Week 108', 'description': 'Incidence of treatment-emergent AEs'}, {'measure': 'Hypermethioninemia', 'timeFrame': 'Week 1 - Week 108', 'description': 'Incidence of hypermethioninemia'}, {'measure': 'Hypomethioninemia', 'timeFrame': 'Week 1 - Week 108', 'description': 'Incidence of hypomethioninemia'}, {'measure': 'Dietary Protein Rescue', 'timeFrame': 'Week 1 - Week 108', 'description': 'Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia'}], 'secondaryOutcomes': [{'measure': 'Total Homocysteine (tHcy) Levels', 'timeFrame': 'Week 1 - Week 108', 'description': 'Relative and absolute changes from baseline in tHcy levels'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Classical homocystinuria', 'HCU', 'Cystathionine Beta Synthase Deficiency'], 'conditions': ['Homocystinuria']}, 'descriptionModule': {'briefSummary': 'The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1/2 COMPOSE study or those who complete the 24 weeks of treatment in the Phase 3 HARMONY are eligible to participate.\n\nParticipants will be in this clinical study for up to about 13 months including:\n\n* a treatment period of up to 104 weeks\n* a 4-week safety follow-up period', 'detailedDescription': 'Overall Design:\n\nThis is a global, multicenter, single-arm, open-label study. Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety, efficacy, and clinical assessments of pegtibatinase treatment.\n\nAll participants will follow a protocol of self-administration of pegtibatinase unless exempted. During the ENSEMBLE study, an optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants active in the COMPOSE study or who completed the 24-week blinded period on the full target dose of study intervention in the Phase 3 HARMONY study\n\nExclusion Criteria:\n\n* Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance'}, 'identificationModule': {'nctId': 'NCT06431893', 'acronym': 'ENSEMBLE', 'briefTitle': 'A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Travere Therapeutics, Inc.'}, 'officialTitle': 'An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)', 'orgStudyIdInfo': {'id': 'TVTX-TVT058-302'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'pegtibatinase', 'interventionNames': ['Drug: Pegtibatinase']}], 'interventions': [{'name': 'Pegtibatinase', 'type': 'DRUG', 'otherNames': ['OT-58', 'TVT-058'], 'description': 'Full Target Dose of pegtibatinase BIW', 'armGroupLabels': ['pegtibatinase']}]}, 'contactsLocationsModule': {'locations': [{'zip': '04012', 'city': 'Portland', 'state': 'Maine', 'country': 'United States', 'facility': 'Travere Investigational Site', 'geoPoint': {'lat': 43.65737, 'lon': -70.2589}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Travere Investigational Site', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27560', 'city': 'Morrisville', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Travere Investigational Site - Virtual Site', 'geoPoint': {'lat': 35.82348, 'lon': -78.82556}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Travere Investigational Site', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}], 'overallOfficials': [{'name': 'Michael Imperiale, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Travere Therapeutics, Inc.'}]}, 'ipdSharingStatementModule': {'timeFrame': 'Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication.', 'ipdSharing': 'YES', 'description': 'Requests for clinical trial data, including language stating its intended use, should be directed to datarequest@travere.com. If approved, the requested information will be provided to the requestor after signing a data access agreement. Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication. Travere reserves the right to decline or recommend modifications to a request if it does not comply with the data sharing policy or if it is determined that the request is made by a biased source.', 'accessCriteria': 'Requires submission and approval of intended use and a data sharing agreement.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Travere Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}