Ignite Creation Date:
2025-12-25 @ 2:02 AM
Ignite Modification Date:
2025-12-27 @ 11:51 PM
Study NCT ID:
NCT00816660
Status:
COMPLETED
Last Update Posted:
2021-05-03
First Post:
2009-01-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D014842', 'term': 'von Willebrand Diseases'}], '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': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['PARTICIPANT']}, 'primaryPurpose': 'OTHER', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 32}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-12-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'completionDateStruct': {'date': '2010-08-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-29', 'studyFirstSubmitDate': '2009-01-02', 'studyFirstSubmitQcDate': '2009-01-02', 'lastUpdatePostDateStruct': {'date': '2021-05-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2009-01-05', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-08-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses', 'timeFrame': 'Up to 30 days after the last investigational product infusion'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Von Willebrand Disease']}, 'referencesModule': {'references': [{'pmid': '23777763', 'type': 'DERIVED', 'citation': 'Mannucci PM, Kempton C, Millar C, Romond E, Shapiro A, Birschmann I, Ragni MV, Gill JC, Yee TT, Klamroth R, Wong WY, Chapman M, Engl W, Turecek PL, Suiter TM, Ewenstein BM; rVWF Ad Hoc Study Group. Pharmacokinetics and safety of a novel recombinant human von Willebrand factor manufactured with a plasma-free method: a prospective clinical trial. Blood. 2013 Aug 1;122(5):648-57. doi: 10.1182/blood-2013-01-479527. Epub 2013 Jun 18.'}]}, 'descriptionModule': {'briefSummary': 'The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '60 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subject has voluntarily given written informed consent (before conduct of any study-related procedures)\n* The subject has hereditary type 3 VWD (\\<= 3 IU/dL VWF:Ag)or severe type 1 or type 2A VWD (VWF:RCo \\<= 10% and FVIII:C \\<20%)\n* The subject has a medical history of at least 25 exposure days to VWF/FVIII coagulation factor concentrates\n* The subject has a Karnofsky score \\>= 70%\n* The subject is between 18 to 60 years of age (on the day of signing the informed consent)\n* NOT APPLICABLE IN ITALY: Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice contraception using a method of proven reliability from the day of screening until the study completion visit\n* APPLICABLE ONLY IN ITALY: Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice non-hormonal-based contraception using a method of proven reliability (IUD acceptable) from the day of screening until 96 hours after the last investigational drug infusion\n* NOT APPLICABLE IN ITALY: The subject must agree not to be on any therapy (hormone-based contraception acceptable) interfering with coagulation factor pharmacokinetics until 96 hours after the last investigational drug infusion\n* APPLICABLE ONLY IN ITALY: The subject must agree not to be on any therapy interfering with coagulation factor pharmacokinetics until 96 hours after the last investigational drug infusion\n\nExclusion Criteria:\n\n* The subject has been diagnosed with a hereditary or acquired coagulation disorder other than VWD (including qualitative and quantitative platelet disorders and/or an international normalized ratio (INR) \\> 1.4)\n* The subject has been diagnosed with an ADAMTS13 deficiency with less than 10% ADAMTS13 activity\n* The subject has a history or presence of VWF inhibitor\n* The subject has a history or presence of FVIII inhibitor with a titer \\>= 0.4 BU (by Nijmegen assay) or \\>= 0.6 BU (by Bethesda assay)\n* The subject has a known hypersensitivity to mouse or hamster proteins\n* The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, food allergies or animal allergies\n* The subject has a medical history of a thromboembolic event\n* The subject is HIV positive with an absolute CD4 count \\< 200/mm3\n* The subject has been diagnosed with cardiovascular disease (New York Heart Association (NYHA) classes 1-4)\n* The subject has been diagnosed with insulin-dependent diabetes mellitus\n* The subject has an acute illness (e.g. influenza, flu-like syndrome, allergic rhinitis/conjunctivitis)\n* The subject has been diagnosed with liver disease, as evidenced by, but not limited to, any of the following: serum ALT three times the upper limit of normal, hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices)\n* The subject has been diagnosed with renal disease, with a serum creatinine level \\>= 2 mg/dL\n* In the judgment of the investigator, the subject has another clinically significant concomitant disease (e.g. uncontrolled hypertension, diabetes type II) that may pose additional risks for the subject\n* The subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g. ointments, nasal sprays) within 30 days before enrollment\n* The subject has been treated with drugs known to induce thrombotic thrombocytopenic purpura (TTP) (e.g. Adenosine diphosphate (ADP) receptor inhibitors (Clopidogrel, Ticlopidine)) within 60 days before enrollment\n* The subject is receiving or anticipates receiving another investigational and/or interventional drug within 30 days before enrollment\n* The subject is a lactating female\n* The subject has a history of drug or alcohol abuse within the last 5 years\n* The subject has a progressive fatal disease and/or life expectancy of less than 3 months\n* The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures\n* The subject suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude\n* Subject is in prison or compulsory detention by regulatory and/or juridical order'}, 'identificationModule': {'nctId': 'NCT00816660', 'briefTitle': 'Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex (rVWF:rFVIII): A Phase 1 Study Evaluating the Pharmacokinetics (PK), Safety, and Tolerability in Type 3 Von Willebrand Disease (VWD)', 'orgStudyIdInfo': {'id': '070701'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1', 'interventionNames': ['Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)']}, {'type': 'ACTIVE_COMPARATOR', 'label': '2', 'interventionNames': ['Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)', 'Biological: Marketed plasma-derived VWF/FVIII concentrate']}], 'interventions': [{'name': 'Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)', 'type': 'BIOLOGICAL', 'description': 'Single dose, dose escalation, various cohorts', 'armGroupLabels': ['1', '2']}, {'name': 'Marketed plasma-derived VWF/FVIII concentrate', 'type': 'BIOLOGICAL', 'description': 'Cross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate', 'armGroupLabels': ['2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '30092', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University School of Medicine, Dept. of Pediatrics', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46260', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana Hemophilia and Thrombosis Center', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '40536-0284', 'city': 'Lexington', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Kentucky Hemophilia Treatment Center', 'geoPoint': {'lat': 37.98869, 'lon': -84.47772}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'facility': 'Brown Cancer Center', 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Brigham & Women´s Hospital, Hematology Division', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '14621', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'Rochester General Hospital', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '15213-4306', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Hemophilia Center of Western PA', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '53225-3548', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Comprehensive Center for Bleeding Disorders', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'facility': 'General Hospital Vienna (Allgemeines Krankenhaus der Stadt Wien), University Department for Internal Medicine I', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': 'B3H 2YP', 'city': 'Halifax', 'state': 'Nova Scotia', 'country': 'Canada', 'facility': 'Q.E.II Health Sciences Centre', 'geoPoint': {'lat': 44.64269, 'lon': -63.57688}}, {'zip': '10249', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Vivantes Klinikum im Friedrichshain', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '30625', 'city': 'Hanover', 'country': 'Germany', 'facility': 'Hannover Medical School - Clinic for Haematology, Haemostaseology, Oncology and Stem Cell Transplantation', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'zip': '48143', 'city': 'Münster', 'country': 'Germany', 'facility': 'Institut für Thrombophilie und Hämostaseologie', 'geoPoint': {'lat': 51.96236, 'lon': 7.62571}}, {'zip': '50134', 'city': 'Florence', 'country': 'Italy', 'facility': 'Azienda Ospedaliero-universitaria "Careggi"', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'zip': '16147', 'city': 'Genova', 'country': 'Italy', 'facility': 'Giannia Gaslini Children´s Hospital', 'geoPoint': {'lat': 45.21604, 'lon': 11.87211}}, {'zip': '20122', 'city': 'Milan', 'country': 'Italy', 'facility': 'Ospedale Maggiore di Milano, Centro Emofilia e Trombosi "Angelo Bianchi Bonomi"', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': '80144', 'city': 'Naples', 'country': 'Italy', 'facility': 'Ospedale San Giovanni Bosco, Centro Emofilia Divisione di Ematologia', 'geoPoint': {'lat': 40.85216, 'lon': 14.26811}}, {'zip': '35128', 'city': 'Padua', 'country': 'Italy', 'facility': 'University of Padua Medical School', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'zip': '80144', 'city': 'Vicenza', 'country': 'Italy', 'facility': 'Ospedale di Vicenza - U.L.S.S.N.6', 'geoPoint': {'lat': 45.54672, 'lon': 11.5475}}, {'zip': 'B15 2TT', 'city': 'Birmingham', 'country': 'United Kingdom', 'facility': 'West Midlands Region Adult Haemophilia Centre, Queen Elizabeth Hospital', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'W12 0NN', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Imperial College School of Medicine, Hammersmith Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Central Manchester Healthcare NHS Trust, Manchester Haemophilia Comprehensive Care Centre', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'zip': 'TR1 3LJ', 'city': 'Truro', 'country': 'United Kingdom', 'facility': 'Royal Cornwall Hospital', 'geoPoint': {'lat': 50.26526, 'lon': -5.05436}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Baxalta now part of Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}