Ignite Creation Date:
2025-12-24 @ 10:12 PM
Ignite Modification Date:
2026-01-01 @ 11:39 PM
Study NCT ID:
NCT04938635
Status:
WITHDRAWN
Last Update Posted:
2022-11-10
First Post:
2021-06-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000708305', 'term': 'VIT-2763'}, {'id': 'C494814', 'term': 'BID protein, human'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'Strategic reasons', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2021-09-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-03', 'completionDateStruct': {'date': '2023-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2022-11-07', 'studyFirstSubmitDate': '2021-06-16', 'studyFirstSubmitQcDate': '2021-06-16', 'lastUpdatePostDateStruct': {'date': '2022-11-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-06-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of patients achieving ≥33% reduction of RBC transfusions from baseline and a reduction of ≥2 units assessed consecutively from Week 13 to Week 24 compared to the baseline transfusion', 'timeFrame': 'Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)'}], 'secondaryOutcomes': [{'measure': 'Change from baseline in RBC transfusions over Weeks 13 to 24 compared to the baseline RBC transfusion burden derived using the last 12 weeks prior to randomization.', 'timeFrame': 'Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)'}, {'measure': 'Proportion of patients achieving ≥50% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.', 'timeFrame': 'Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)'}, {'measure': 'Proportion of patients achieving ≥33% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.', 'timeFrame': 'Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)'}, {'measure': 'Mean change from baseline in Quality of Life (QoL) total score', 'timeFrame': 'Week 15 and Week 24 comparing to Baseline (Day 1)', 'description': 'Transfusion-dependent QoL Questionnaire (TranQuol): a disease-specific, validated, QoL measure developed for thalassemia patients. The adult version includes 36 questions grouped into 5 domains: physical health, emotional health, sexual health, family functioning, and school/career functioning. The total score ranges from 0 (worst) to 100 (best).'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Beta-Thalassemia', 'Transfusion-dependent Thalassemia'], 'conditions': ['Beta-Thalassemia']}, 'descriptionModule': {'briefSummary': 'The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe dose.', 'detailedDescription': 'All patients giving written informed consent will undergo a 12-week screening period to determine eligibility for study entry. At Day 1, patients who meet the eligibility requirements will be randomized in a double-blind manner to 1 of 4 treatment groups: VIT-2763 60 mg (once daily), 60 mg (twice daily), or 120 mg (twice daily) or placebo.\n\nThe randomisation will be stratified (balanced allocation across treatment groups) according to β0/β0 genotype (yes/no).\n\nThe duration of the treatment with VIT-2763 or placebo is 24 weeks, after which patients will undergo a 12-week post-treatment follow-up period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Body weight ≥40.0 kg and ≤100 kg at screening\n* Documented diagnosis of beta-thalassemia or Hb E/beta-thalassemia\n* Red blood cell (RBC) transfusion dependence, defined as at least 6 RBC units in the 24 weeks prior to randomization and no transfusion-free period for ≥35 days during that period\n* Ability to understand the requirements of the study and provide written informed consent\n\nExclusion Criteria:\n\n* Documented diagnosis of Hb S/beta-thalassemia, alpha-thalassemia, or delta beta (δβ)-thalassemia, or hereditary persistence of foetal Hb.\n* History of partial or total splenectomy within 4 months prior to screening.\n* History of myocardial iron overload\n* Chronic liver disease or history of liver cirrhosis\n* Clinically relevant renal disease\n* History or clinically important finding of cardiac disorders\n* History of clinically significant lung disease\n* Uncontrolled hypertension (\\> Grade 1 according to NCI CTCAE current version)\n* Unable to take and absorb oral medications.\n* Pregnancy or breastfeeding\n* History of drug or alcohol abuse within 2 years prior to screening\n* History or concomitant solid tumors and/or hematological malignancies unless resolved in the ≥5 past years.'}, 'identificationModule': {'nctId': 'NCT04938635', 'briefTitle': 'Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vifor Pharma'}, 'officialTitle': 'A Phase 2b, Double-blind, Randomised, Placebo-controlled, Multicentre Study to Assess the Efficacy and Safety of VIT-2763 Multiple Doses in Adults With Transfusion-dependent Beta-thalassaemia', 'orgStudyIdInfo': {'id': 'VIT-2763-THAL-203'}, 'secondaryIdInfos': [{'id': '2021-001639-23', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'VIT-2763 60 mg QD', 'description': 'VIT-2763 60 mg administered once daily', 'interventionNames': ['Drug: VIT-2763 60 mg QD']}, {'type': 'EXPERIMENTAL', 'label': 'VIT-2763 60 mg BID', 'description': 'VIT-2763 60 mg administered twice daily', 'interventionNames': ['Drug: VIT-2763 60 mg BID']}, {'type': 'EXPERIMENTAL', 'label': 'VIT-2763 120 mg BID', 'description': 'VIT-2763 120 mg administered twice daily', 'interventionNames': ['Drug: VIT-2763 120 mg BID']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo capsule administered twice daily', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'VIT-2763 60 mg QD', 'type': 'DRUG', 'otherNames': ['Vamifeport 60 mg QD'], 'description': 'Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally in the morning, and 2 Placebo capsules orally in the evening, for 24 weeks.', 'armGroupLabels': ['VIT-2763 60 mg QD']}, {'name': 'VIT-2763 60 mg BID', 'type': 'DRUG', 'otherNames': ['Vamifeport 60 mg BID'], 'description': 'Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.', 'armGroupLabels': ['VIT-2763 60 mg BID']}, {'name': 'VIT-2763 120 mg BID', 'type': 'DRUG', 'otherNames': ['Vamifeport 120 mg BID'], 'description': 'Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.', 'armGroupLabels': ['VIT-2763 120 mg BID']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90603-2137', 'city': 'Whittier', 'state': 'California', 'country': 'United States', 'facility': 'Investigator site #710', 'geoPoint': {'lat': 33.97918, 'lon': -118.03284}}, {'city': 'Plovdiv', 'country': 'Bulgaria', 'facility': 'Investigational site #802', 'geoPoint': {'lat': 42.15387, 'lon': 24.75001}}, {'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'Investigational site #801', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'city': 'Stara Zagora', 'country': 'Bulgaria', 'facility': 'Investigational site #804', 'geoPoint': {'lat': 42.43205, 'lon': 25.64262}}, {'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Investigator Site 404', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'city': 'Petah Tikva', 'country': 'Israel', 'facility': 'Investigator Site 406', 'geoPoint': {'lat': 32.08707, 'lon': 34.88747}}, {'city': 'Safed', 'country': 'Israel', 'facility': 'Investigator Site 405', 'geoPoint': {'lat': 32.96465, 'lon': 35.496}}], 'overallOfficials': [{'name': 'Peter Szecsödy', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Vifor (International) Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vifor (International) Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Labcorp Corporation of America Holdings, Inc', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}