Ignite Creation Date:
2025-12-24 @ 11:14 PM
Ignite Modification Date:
2026-01-05 @ 10:52 PM
Study NCT ID:
NCT02971969
Status:
COMPLETED
Last Update Posted:
2022-01-06
First Post:
2016-11-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002836', 'term': 'Hemophilia B'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Blood'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-01', 'completionDateStruct': {'date': '2021-11-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-01-04', 'studyFirstSubmitDate': '2016-11-21', 'studyFirstSubmitQcDate': '2016-11-21', 'lastUpdatePostDateStruct': {'date': '2022-01-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-11-23', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2021-11-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of adverse events and serious adverse events by dosing group', 'timeFrame': '208 weeks'}, {'measure': 'Change from baseline in FIX activity level', 'timeFrame': '208 weeks'}], 'secondaryOutcomes': [{'measure': 'Number of bleeding episodes requiring recombinant FIX infusion', 'timeFrame': '208 weeks'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['gene therapy', 'Hemophilia B'], 'conditions': ['Hemophilia B']}, 'referencesModule': {'references': [{'pmid': '40197980', 'type': 'DERIVED', 'citation': 'Pipe S, Poma A, Rajasekhar A, Everington T, Sankoh S, Allen J, Cataldo J, Crombez E. Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies. Blood Adv. 2025 Jun 24;9(12):2980-2987. doi: 10.1182/bloodadvances.2024015184.'}]}, 'descriptionModule': {'briefSummary': 'A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.', 'detailedDescription': 'Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation. Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to chronic arthropathy (joint damage), intracranial hemorrhage, and even death. In patients with moderate/severe to severe hemophilia B, the majority of bleeding episodes occur in the joints and, if not treated, lead to debilitating damage and a decreased quality of life.\n\nStudy 101HEMB02 is a long-term follow-up study to evaluate the safety, tolerability, and efficacy of AAVrh10-mediated gene therapy of human FIX in subjects with moderate/severe to severe hemophilia B. The primary objective of the study is to determine the long-term safety and efficacy of DTX101 following a single IV infusion (administered during Study 101HEMB01) in adults with moderate/severe to severe hemophilia B.\n\nThis study was previously posted by Dimension Therapeutics, which has been acquired by Ultragenyx.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01 clinical study', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Willing and able to provide written informed consent.\n2. Completed the Week 52 visit in Study 101HEMB01.\n3. Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable.\n4. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.\n\nExclusion Criteria:\n\n1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX101 during the duration of this study.\n2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study'}, 'identificationModule': {'nctId': 'NCT02971969', 'briefTitle': 'Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ultragenyx Pharmaceutical Inc'}, 'officialTitle': 'A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B', 'orgStudyIdInfo': {'id': '101HEMB02'}, 'secondaryIdInfos': [{'id': '2016-003430-25', 'type': 'EUDRACT_NUMBER'}]}, 'contactsLocationsModule': {'locations': [{'zip': '72202', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': "Arkansas Children's Hospital", 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'UF CRC - Clinical Research Center', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '48109-5872', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'University of Michigan Hospital and Health Systems', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': 'RG24 9NA', 'city': 'Basingstoke', 'state': 'Hampshire', 'country': 'United Kingdom', 'facility': 'Haemophilia, Haemostasis & Thrombosis Centre', 'geoPoint': {'lat': 51.26249, 'lon': -1.08708}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Manchester Haemophilia Comprehensive Care Center', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ultragenyx Pharmaceutical Inc'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ultragenyx Pharmaceutical Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}