rVWF IN PROPHYLAXIS

Last updated: August 5, 2021
Sponsor: Takeda
Overall Status: Completed

Phase

3

Condition

Von Willebrand Disease

Dysfunctional Uterine Bleeding

Thrombosis

Treatment

N/A

Clinical Study ID

NCT02973087
071301
2016-001478-14
  • Ages > 18
  • All Genders

Study Summary

The purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity, thrombogenicity and hypersensitivity reactions, as well as pharmacokinetics (PK), health related quality of life (HRQoL) and pharmacoeconomics of prophylactic treatment with recombinant von Willebrand factor (rVWF) (vonicog alfa) in adult participants with severe von Willebrand disease (VWD).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. 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
  2. Type 1 (VWF:RCo <20 IU/dL) or,
  3. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype),Type 2M or,
  4. Type 3 (Von Willebrand factor antigen (VWF:Ag) less than or equal to [< or =] 3IU/dL).
  5. Diagnosis is confirmed by genetic testing and multimer analysis, documented in patienthistory or at screening.
  6. For on-demand patient group, participant currently receiving on-demand treatment forwhom prophylactic treatment is recommended by the investigator.
  7. 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.
  8. 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.
  9. 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.
  10. 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).
  11. 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.
  12. Participant is willing and able to comply with the requirements of the protocol.

Exclusion

Exclusion Criteria:

  1. 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).
  2. The participant is currently receiving prophylactic treatment with more than 5infusions per week.
  3. The participant is currently receiving prophylactic treatment with a weekly doseexceeding 240 IU/kg.
  4. The participant has a history or presence of a VWF inhibitor at screening.
  5. 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).
  6. The participant has a known hypersensitivity to any of the components of the studydrugs, such as to mouse or hamster proteins.
  7. The participant has a medical history of immunological disorders, excluding seasonalallergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
  8. The participant has a medical history of a thromboembolic event.
  9. The participant is human immunodeficiency virus (HIV) positive with an absolute HelperT cell (CD4) count <200/ cubic millimeter (mm^3).
  10. 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).
  11. The participant has been diagnosed with renal disease, with a serum creatinine (CR)level > or = 2.5 milligram per deciliter (mg/dL).
  12. The participant has a platelet count <100,000/ milliliter (mL) at screening.
  13. The participant has been treated with an immunomodulatory drug, excluding topicaltreatment (e.g., ointments, nasal sprays), within 30 days prior to signing theinformed consent.
  14. The participant is pregnant or lactating at the time of enrollment.
  15. Patient has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
  16. 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.
  17. The participant has a progressive fatal disease and/or life expectancy of less than 15months.
  18. The participant is scheduled for a surgical intervention.
  19. The participant is identified by the investigator as being unable or unwilling tocooperate with study procedures.
  20. 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.
  21. The participant is in prison or compulsory detention by regulatory and/or juridicalorder.
  22. 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

Total Participants: 29
Study Start date:
November 16, 2017
Estimated Completion Date:
July 06, 2020

Connect with a study center

  • Hamilton Health Sciences Centre

    Hamilton, L8N 3Z5
    Canada

    Site Not Available

  • Hôpital Morvan

    Brest, Finistere 29609
    France

    Site Not Available

  • Hôpital Morvan

    Brest Cedex, Finistere 29609
    France

    Site Not Available

  • Groupement Hospitalier Est- Hôpital Louis Pradel

    Bron cedex, 69677
    France

    Site Not Available

  • CHU CAEN - Hôpital de la Côte de Nacre

    Caen cedex 9, 14033
    France

    Site Not Available

  • CHU Dijon - Hopital du Bocage

    Dijon cedex, 21079
    France

    Site Not Available

  • Hopital Cardiologique - CHU Lille

    Lille Cedex, 59037
    France

    Site Not Available

  • Coagulation Research Centre GmbH

    Duisburg, 47051
    Germany

    Site Not Available

  • Klinikum der Johann Wolfgang Goethe-Universitaet

    Frankfurt, 60590
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover

    Hannover, 30625
    Germany

    Site Not Available

  • Werlhof-Institut GmbH

    Hannover, 30159
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover

    Marburg, 30625
    Germany

    Site Not Available

  • Ospedale Pediatrico Bambino Gesù

    Rome, Roma 165
    Italy

    Site Not Available

  • Azienda Ospedaliera Universitaria Careggi

    Firenze, 50134
    Italy

    Site Not Available

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milano, 20122
    Italy

    Site Not Available

  • Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza

    Roma, 00161
    Italy

    Site Not Available

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 00168
    Italy

    Site Not Available

  • Ospedale Pediatrico Bambino Gesù

    Roma, 00165
    Italy

    Site Not Available

  • Erasmus Medisch Centrum

    Rotterdam, 3015 AA
    Netherlands

    Site Not Available

  • SBEI HPE Altai State Medical University of MoH and SD

    Barnaul, 656038
    Russian Federation

    Site Not Available

  • SAIH "Kemerovo Regional Clinical Hospital"

    Kemerovo, 650066
    Russian Federation

    Site Not Available

  • FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA

    Kirov, 610017
    Russian Federation

    Site Not Available

  • Hospital General Universitario de Alicante

    Alicante, 03010
    Spain

    Site Not Available

  • Hospital Universitario La Paz

    Madrid, 28046
    Spain

    Site Not Available

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026
    Spain

    Site Not Available

  • Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty

    Istanbul, 34098
    Turkey

    Site Not Available

  • Ege University Medical Faculty

    Izmir, 35040
    Turkey

    Site Not Available

  • Ondokuz Mayis Univ. Med. Fac.

    Samsun, 55139
    Turkey

    Site Not Available

  • University of Colorado Health

    Loveland, Colorado 80045
    United States

    Site Not Available

  • University of Florida College of Medicine

    Gainesville, Florida 32610
    United States

    Site Not Available

  • University of Florida College of Medicine

    Jacksonville, Florida 32610
    United States

    Site Not Available

  • Bleeding and Clotting Disorders Institute

    Peoria, Illinois 61615
    United States

    Site Not Available

  • University of Colorado Health

    Aurora, Indiana 80045
    United States

    Site Not Available

  • Indiana Hemophilia and Thrombosis Center

    Indianapolis, Indiana 46260
    United States

    Site Not Available

  • Henry Ford Hospital

    Detroit, Michigan 48202
    United States

    Site Not Available

  • Comprehensive Cancer Center of Wake Forest Unversity

    Winston-Salem, North Carolina 27157
    United States

    Site Not Available

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