rVWF IN PROPHYLAXIS (NCT02973087) | Clinical Trial Compass
CompletedPhase 3
rVWF IN PROPHYLAXIS
United States29 participantsStarted 2017-11-16
Plain-language 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).
Who can participate
Age range18 Years
SexALL
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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 substitution therapy 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 \[\< or =\] 3 IU/dL).
✓. Diagnosis is confirmed by genetic testing and multimer analysis, documented in patient history or at screening.
✓. For on-demand patient group, participant currently receiving on-demand treatment for whom 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 less than 12 months prior to screening.
✓. For on-demand patient group, participant has greater than or equal to (\>or=) 3 documented spontaneous bleeds (not including menorrhagia) requiring von Willebrand factor (VWF) treatment during the past 12 months.
Exclusion criteria
✕. The participant has been diagnosed with Type 2N Von Willebrand disease (VWD), pseudo VWD, or another hereditary or acquired coagulation disorder other than VWD (eg qualitative and quantitative platelet disorders or prothrombin time \[PT\]/international normalized ratio \[INR\] greater than \[\>\]1.4).
What they're measuring
1
Ratio of Annualized Bleeding Rate (ABR) for Spontaneous Bleeding Episodes (BEs) (On-study ABR / Historical ABR) Assessed by Investigator During Prophylactic Treatment With rVWF Through Month 12
✕. The participant is currently receiving prophylactic treatment with more than 5 infusions per week.
✕. The participant is currently receiving prophylactic treatment with a weekly dose exceeding 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 a titer ≥0.4 Bethesda units (BU) (by Nijmegen modified Bethesda assay) or \> or = 0.6 Bethesda Unit (BU) (by Bethesda assay).
✕. The participant has a known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins.
✕. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
✕. The participant has a medical history of a thromboembolic event.