Ignite Creation Date:
2025-12-25 @ 4:12 AM
Ignite Modification Date:
2026-01-09 @ 6:44 AM
Study NCT ID:
NCT03432520
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-11-29
First Post:
2018-01-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], '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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2018-08-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-11', 'completionDateStruct': {'date': '2032-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-11-26', 'studyFirstSubmitDate': '2018-01-24', 'studyFirstSubmitQcDate': '2018-02-07', 'lastUpdatePostDateStruct': {'date': '2024-11-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-02-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2032-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Up to 9 years'}, {'measure': 'Number of Participants with Adverse Events of Special Interest (AESIs)', 'timeFrame': 'Up to 9 years'}, {'measure': 'Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development', 'timeFrame': 'Up to 9 years'}, {'measure': 'Number of Participants with Clinical Laboratory Abnormalities', 'timeFrame': 'Up to 9 years'}, {'measure': 'Number of Participants with Vital Sign Abnormalities', 'timeFrame': 'Up to 9 years'}, {'measure': 'Number of Participants with Physical Exam Abnormalities', 'timeFrame': 'Up to 9 years'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hemophilia A']}, 'referencesModule': {'references': [{'pmid': '34788507', 'type': 'DERIVED', 'citation': 'George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.'}]}, 'descriptionModule': {'briefSummary': 'This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.', 'detailedDescription': 'This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'genderBased': True, 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Males with hemophilia A, who received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored study.', 'genderDescription': 'Genetic males', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria\n\nParticipants are eligible to be included in the study only if all of the following criteria apply:\n\n1. Have received a single intravenous administration of SPK-8011 or SPK-8016 in either Study SPK-8011-101 or SPK-8016-101, respectively.\n2. Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures.\n\nExclusion Criteria\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.'}, 'identificationModule': {'nctId': 'NCT03432520', 'briefTitle': 'Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'Spark Therapeutics, Inc.'}, 'officialTitle': 'A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A', 'orgStudyIdInfo': {'id': 'SPK-8011/8016-LTFU'}}, 'armsInterventionsModule': {'interventions': [{'name': 'SPK-8011', 'type': 'GENETIC', 'description': 'Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study'}, {'name': 'SPK-8016', 'type': 'GENETIC', 'description': 'Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study'}]}, 'contactsLocationsModule': {'locations': [{'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '39110', 'city': 'Madison', 'state': 'Mississippi', 'country': 'United States', 'facility': 'Mississippi Center for Advanced Medicine', 'geoPoint': {'lat': 32.46181, 'lon': -90.11536}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': 'Truman Medical Centers', 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health & Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '10733', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Pennsylvania State University Milton S. Hershey Medical Center', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "The Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Thomas Jefferson University', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Hemophilia Center of Western Pennsylvania', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '23298', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': '2050', 'city': 'Sydney', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Royal Prince Alfred Hospital Department of Cell & Molecular Therapies', 'geoPoint': {'lat': -33.86785, 'lon': 151.20732}}, {'city': 'Melbourne', 'state': 'Victoria', 'country': 'Australia', 'facility': 'The Alfred Hospital & Monash Medical Centre', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'zip': 'M5B 1W8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': "St. Michael's Hospital", 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}], 'overallOfficials': [{'name': 'Clinical Trial Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Spark Therapeutics, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Spark Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}