Phase
Condition
Von Willebrand Disease
Dysfunctional Uterine Bleeding
Thrombosis
Treatment
N/AClinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
- Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline Von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) less than (<) 20 International Units/Deciliter [IU/dL]) with a history of requiring substitutiontherapy with von Willebrand factor concentrate to control bleeding
- Type 1 (VWF:RCo <20 IU/dL) or,
- Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype),Type 2M or,
- Type 3 (Von Willebrand factor antigen (VWF:Ag) less than or equal to [< or =] 3IU/dL).
- Diagnosis is confirmed by genetic testing and multimer analysis, documented in patienthistory or at screening.
- For on-demand patient group, participant currently receiving on-demand treatment forwhom prophylactic treatment is recommended by the investigator.
- For Plasma derived von Willebrand factor (pdVWF) product switch patient group,participant has been receiving prophylactic treatment of pdVWF products for no lessthan 12 months prior to screening.
- For on-demand patient group, participant has greater than or equal to (>or=) 3documented spontaneous bleeds (not including menorrhagia) requiring von Willebrandfactor (VWF) treatment during the past 12 months.
- Availability of records to reliably evaluate type, frequency and treatment of bleedingepisodes during at least 12 months preceding enrollment. Up to 24 months retrospectivedata should be collected if available. Availability of dosing and factor consumptionduring 12 months (up to 24 months) of treatment prior to enrollment is required forpdVWF switch participants and is desired (but not a requirement) for on-demandparticipants.
- Participant is > or = 18 years old at the time of screening and has a body mass index > or = 15 but <40 kilogram per meter square (kg/m^2).
- If female of childbearing potential, participant presents with a negative blood/urinepregnancy test at screening and agrees to employ adequate birth control measures forthe duration of the study.
- Participant is willing and able to comply with the requirements of the protocol.
Exclusion
Exclusion Criteria:
- The participant has been diagnosed with Type 2N Von Willebrand disease (VWD), pseudoVWD, or another hereditary or acquired coagulation disorder other than VWD (egqualitative and quantitative platelet disorders or prothrombin time [PT]/internationalnormalized ratio [INR] greater than [>]1.4).
- The participant is currently receiving prophylactic treatment with more than 5infusions per week.
- The participant is currently receiving prophylactic treatment with a weekly doseexceeding 240 IU/kg.
- The participant has a history or presence of a VWF inhibitor at screening.
- The participant has a history or presence of a Factor VIII (FVIII) inhibitor with atiter ≥0.4 Bethesda units (BU) (by Nijmegen modified Bethesda assay) or > or = 0.6Bethesda Unit (BU) (by Bethesda assay).
- The participant has a known hypersensitivity to any of the components of the studydrugs, such as to mouse or hamster proteins.
- The participant has a medical history of immunological disorders, excluding seasonalallergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
- The participant has a medical history of a thromboembolic event.
- The participant is human immunodeficiency virus (HIV) positive with an absolute HelperT cell (CD4) count <200/ cubic millimeter (mm^3).
- The participant has been diagnosed with significant liver disease per investigator'smedical assessment of the participant's current condition or medical history or asevidenced by any of the following: serum alanine aminotransferase (ALT) greater than 5times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g.,presence of otherwise unexplained splenomegaly, history of esophageal varices).
- The participant has been diagnosed with renal disease, with a serum creatinine (CR)level > or = 2.5 milligram per deciliter (mg/dL).
- The participant has a platelet count <100,000/ milliliter (mL) at screening.
- The participant has been treated with an immunomodulatory drug, excluding topicaltreatment (e.g., ointments, nasal sprays), within 30 days prior to signing theinformed consent.
- The participant is pregnant or lactating at the time of enrollment.
- Patient has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
- The participant has participated in another clinical study involving anotherInvestigational product (IP) or investigational device within 30 days prior toenrollment or is scheduled to participate in another clinical study involving an IP orinvestigational device during the course of this study.
- The participant has a progressive fatal disease and/or life expectancy of less than 15months.
- The participant is scheduled for a surgical intervention.
- The participant is identified by the investigator as being unable or unwilling tocooperate with study procedures.
- The participant has a mental condition rendering him/her unable to understand thenature, scope and possible consequences of the study and/or evidence of anuncooperative attitude.
- The participant is in prison or compulsory detention by regulatory and/or juridicalorder.
- The participant is member of the study team or in a dependent relationship with one ofthe study team members which includes close relatives (i.e., children, partner/spouse,siblings and parents) as well as employees.
Study Design
Connect with a study center
Hamilton Health Sciences Centre
Hamilton, L8N 3Z5
CanadaSite Not Available
Hôpital Morvan
Brest, Finistere 29609
FranceSite Not Available
Hôpital Morvan
Brest Cedex, Finistere 29609
FranceSite Not Available
Groupement Hospitalier Est- Hôpital Louis Pradel
Bron cedex, 69677
FranceSite Not Available
CHU CAEN - Hôpital de la Côte de Nacre
Caen cedex 9, 14033
FranceSite Not Available
CHU Dijon - Hopital du Bocage
Dijon cedex, 21079
FranceSite Not Available
Hopital Cardiologique - CHU Lille
Lille Cedex, 59037
FranceSite Not Available
Coagulation Research Centre GmbH
Duisburg, 47051
GermanySite Not Available
Klinikum der Johann Wolfgang Goethe-Universitaet
Frankfurt, 60590
GermanySite Not Available
Medizinische Hochschule Hannover
Hannover, 30625
GermanySite Not Available
Werlhof-Institut GmbH
Hannover, 30159
GermanySite Not Available
Medizinische Hochschule Hannover
Marburg, 30625
GermanySite Not Available
Ospedale Pediatrico Bambino Gesù
Rome, Roma 165
ItalySite Not Available
Azienda Ospedaliera Universitaria Careggi
Firenze, 50134
ItalySite Not Available
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milano, 20122
ItalySite Not Available
Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza
Roma, 00161
ItalySite Not Available
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168
ItalySite Not Available
Ospedale Pediatrico Bambino Gesù
Roma, 00165
ItalySite Not Available
Erasmus Medisch Centrum
Rotterdam, 3015 AA
NetherlandsSite Not Available
SBEI HPE Altai State Medical University of MoH and SD
Barnaul, 656038
Russian FederationSite Not Available
SAIH "Kemerovo Regional Clinical Hospital"
Kemerovo, 650066
Russian FederationSite Not Available
FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA
Kirov, 610017
Russian FederationSite Not Available
Hospital General Universitario de Alicante
Alicante, 03010
SpainSite Not Available
Hospital Universitario La Paz
Madrid, 28046
SpainSite Not Available
Hospital Universitari i Politecnic La Fe
Valencia, 46026
SpainSite Not Available
Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty
Istanbul, 34098
TurkeySite Not Available
Ege University Medical Faculty
Izmir, 35040
TurkeySite Not Available
Ondokuz Mayis Univ. Med. Fac.
Samsun, 55139
TurkeySite Not Available
University of Colorado Health
Loveland, Colorado 80045
United StatesSite Not Available
University of Florida College of Medicine
Gainesville, Florida 32610
United StatesSite Not Available
University of Florida College of Medicine
Jacksonville, Florida 32610
United StatesSite Not Available
Bleeding and Clotting Disorders Institute
Peoria, Illinois 61615
United StatesSite Not Available
University of Colorado Health
Aurora, Indiana 80045
United StatesSite Not Available
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana 46260
United StatesSite Not Available
Henry Ford Hospital
Detroit, Michigan 48202
United StatesSite Not Available
Comprehensive Cancer Center of Wake Forest Unversity
Winston-Salem, North Carolina 27157
United StatesSite Not Available

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