Ignite Creation Date:
2025-12-24 @ 11:02 PM
Ignite Modification Date:
2026-02-21 @ 9:44 AM
Study NCT ID:
NCT02571569
Status:
COMPLETED
Last Update Posted:
2018-10-18
First Post:
2015-10-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}, {'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': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'BASIC_SCIENCE', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 32}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-10-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-10', 'completionDateStruct': {'date': '2018-10-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-10-17', 'studyFirstSubmitDate': '2015-10-07', 'studyFirstSubmitQcDate': '2015-10-07', 'lastUpdatePostDateStruct': {'date': '2018-10-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-10-08', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-07-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of participants (single dose cohors) with adverse events as measure of safety and tolerability', 'timeFrame': 'Up to 56 days', 'description': 'Adverse events including abnormal laboratory findings and local injection site reactions'}, {'measure': 'Plasma levels of anti-BAY1093884 antibodies', 'timeFrame': 'Pre-dose, Day 14, 21,28, 43 and 56'}, {'measure': 'Plasma concentration of BAY1093884 characterized by AUC(0-tlast)', 'timeFrame': 'Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion', 'description': 'AUC from time 0 to the last data point \\> LLOQ (lower limit of quantitation)'}, {'measure': 'Plasma concentration of BAY1093884 characterized by AUC(0-tlast)/D', 'timeFrame': 'Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion', 'description': 'AUC(0-last) divided by dose'}, {'measure': 'Plasma concentration of BAY1093884 characterized by Cmax', 'timeFrame': 'Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion', 'description': 'Maximum observed drug concentration in measured matrix after single dose administration'}, {'measure': 'Plasma concentration of BAY1093884 characterized by Cmax/D', 'timeFrame': 'Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion', 'description': 'Cmax divided by dose'}], 'secondaryOutcomes': [{'measure': 'Tissue factor pathway inhibitor (TFPI) activity', 'timeFrame': 'Up to 77 days'}, {'measure': 'Number of participants (multiple dose cohort) with adverse events as a measure of safety and tolerability', 'timeFrame': 'Up to 77 days', 'description': 'Adverse events including abnormal laboratory findings and local injection site reactions'}, {'measure': 'Plasma levels of anti-BAY1093884 antibodies (multiple dose cohort)', 'timeFrame': 'Pre-dose, Day 14, 28, 49 and 77'}, {'measure': 'Plasma concentration of BAY1093884 characterized by AUC(0-7d and AUC(0-tau) (multiple dose cohort)', 'timeFrame': 'Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42', 'description': 'AUC from time 0 to 7d after first and last dose (AUC(0-tau)'}, {'measure': 'Plasma concentration of BAY1093884 characterized by AUC(0-7d/D and AUC(0-tau)/D after multiple dose', 'timeFrame': 'Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42', 'description': 'AUC(0-7d) after first dose and AUC(0-tau) after last dose divided by dose'}, {'measure': 'Plasma concentration of BAY1093884 characterized by Cmax after first dose and last dose (Cmax,md)', 'timeFrame': 'Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42', 'description': 'maximum observed drug concentration in measured matrix after first and last dose'}, {'measure': 'Plasma concentration of BAY1093884 characterized by Cmax/D after first dose and last dose (Cmax,md/D)', 'timeFrame': 'Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42', 'description': 'Cmax after first and last dose divided by dose'}, {'measure': 'Accumulation of BAY 1093884 in plasma as defined by ratio for Cmax and AUC (after first and last dose)', 'timeFrame': 'Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42', 'description': 'Cmax after last dose divided by Cmax after first dose, AUC after last dose divided by AUC after first dose'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hemophilia A', 'Hemophilia B']}, 'descriptionModule': {'briefSummary': 'Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males with severe congenital Hemophilia A or B defined as \\<1% FVIII or Factor IX (FIX) concentration by measurement at the time of screening or from reliable prior documentation\n* For subjects in Cohorts I-IV, I-SC1 and I-SC2; If history of inhibitors is evident, inhibitor titer of ≥5 Bethesda Units (BU) at screening or prior to screening at any time from medical records.\n* Age: 18 to 65 years of age at screening\n* Body mass index (BMI): 18 to 29.9 kg/m²\n\nExclusion Criteria:\n\n* Subjects with known bleeding disorders (such as von Willebrand factor \\[vWF\\] deficiency, FXI deficiency, platelet disorders, or known acquired or inherited thrombophilia etc.) other than congenital Hemophilia A or B with or without inhibitors\n* History of angina pectoris or treatment for angina pectoris\n* History of coronary and/or peripheral atherosclerotic disease, congestive heart failure, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) ≥160 mmHg or diastolic blood pressure (DBP) ≥100 mmHg even if controlled\n* History of thrombophlebitis, venous / arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction, cerebrovascular accident, ischemic heart disease, transient ischemic attack)\n* Known or suspected hypersensitivity of the immune system, history of anaphylactic reaction, known (clinically relevant) allergies, non-allergic drug reactions, or multiple drug allergies'}, 'identificationModule': {'nctId': 'NCT02571569', 'briefTitle': 'A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors', 'organization': {'class': 'INDUSTRY', 'fullName': 'Bayer'}, 'officialTitle': 'A Phase 1, First in Man, Multicenter, Open Label, Single Escalating Dose Study of BAY1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors', 'orgStudyIdInfo': {'id': '16144'}, 'secondaryIdInfos': [{'id': '2014-003283-20', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Without inhibitors', 'description': 'Dose escalation steps for participants without inhibitors - intravenous infusion and subcutaneous injection', 'interventionNames': ['Drug: BAY1093884']}, {'type': 'EXPERIMENTAL', 'label': 'With inhibitors', 'description': 'Dose escalation steps for participants with inhibitors - intravenous infusion and subcutaneous injection', 'interventionNames': ['Drug: BAY1093884']}, {'type': 'EXPERIMENTAL', 'label': 'Without inhibitors_multiple dose', 'description': 'Multiple dose cohort for participants without inhibitors - a single subcutaneous injection once a week for 6 weeks', 'interventionNames': ['Drug: BAY1093884']}], 'interventions': [{'name': 'BAY1093884', 'type': 'DRUG', 'description': 'Single escalating dose with a starting dose of 0.3 mg/kg for the first cohort. Drug will be administered via IV infusion over 1 hour and SC injection. Based on safety, PK and PD results the doses for the other cohorts will be determined.', 'armGroupLabels': ['With inhibitors', 'Without inhibitors']}, {'name': 'BAY1093884', 'type': 'DRUG', 'description': 'Multiple dose cohort with a single 150-mg SC injection once a week for 6 weeks.', 'armGroupLabels': ['Without inhibitors_multiple dose']}]}, 'contactsLocationsModule': {'locations': [{'zip': '4002', 'city': 'Plovdiv', 'country': 'Bulgaria', 'geoPoint': {'lat': 42.15387, 'lon': 24.75001}}, {'zip': '1756', 'city': 'Sofia', 'country': 'Bulgaria', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'zip': '9010', 'city': 'Varna', 'country': 'Bulgaria', 'geoPoint': {'lat': 43.21912, 'lon': 27.91024}}, {'zip': '35392', 'city': 'Giessen', 'state': 'Hesse', 'country': 'Germany', 'geoPoint': {'lat': 50.58727, 'lon': 8.67554}}, {'zip': '10249', 'city': 'Berlin', 'country': 'Germany', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '167-0035', 'city': 'Suginami', 'state': 'Tokyo', 'country': 'Japan'}, {'city': 'Kiev', 'country': 'Ukraine', 'geoPoint': {'lat': 50.45466, 'lon': 30.5238}}, {'zip': '79044', 'city': 'Lviv', 'country': 'Ukraine', 'geoPoint': {'lat': 49.83826, 'lon': 24.02324}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'country': 'United Kingdom', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'Bayer Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Bayer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bayer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}