A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Seve… (NCT03879135) | Clinical Trial Compass
CompletedPhase 3
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
United States38 participantsStarted 2019-04-01
Plain-language summary
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants during the first 12 months on study treatment.
The participants will be treated with rVWF for a maximum of 3 years. Their von Willebrand Disease will be treated according to Investigational product (IP) dosing directions.
Who can participate
Age range0 Years
SexALL
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Inclusion Criteria:
The participant will not be considered eligible for the study without meeting all of the criteria below.
Participants who have completed Study 071301 or Study 071102 (or participants who have completed the surgery arm treatment in Study 071102 and want to continue to receive on-demand (OD) treatment) and are willing to immediately transition into this study, must meet the following 2 criteria to be eligible for this study:
* If female of childbearing potential, has a negative blood/urine pregnancy test at screening and agrees to employ highly effective birth control measures for the duration of the study.
* Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
New participants (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study:
\- Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline von Willebrand factor: Ristocetin cofactor (VWF:RCo) \<20 International Units per deciliter \[IU/dL\]) with a history of requiring substitution therapy with von Willebrand factor (vWF) 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 (\<=) 3 IU/dL).
Diagnosis is confirmed by genetic testing and multimer analysis, documented in participant hi…