Home › Trials › NCT02932618

Treatment of severe Von Willebrand Disease in children using recombinant factor

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

Phase 3 Interventional Takeda · NCT02932618

This study is testing a new treatment called vonicog alfa to see if it helps children with severe von Willebrand disease stop bleeding better than current methods.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment31 (estimated)
AgesN/A to 17 Years
SexAll
SponsorTakeda Industry-sponsored
Drugs / interventionschemotherapy
Locations46 sites (Aurora, Colorado and 45 other locations)
Trial IDNCT02932618 on ClinicalTrials.gov

What this trial studies

This study evaluates the effectiveness, side effects, and tolerability of vonicog alfa, a recombinant von Willebrand factor, in treating nonsurgical bleeding events in children under 18 with severe hereditary von Willebrand disease. Participants will receive treatment for 12-18 months, with their condition managed according to standard clinical practices. Follow-up will occur through clinic visits or telephone calls to monitor progress and outcomes.

Who should consider this trial

Good fit: Ideal candidates are children aged 0 to less than 18 years diagnosed with severe Von Willebrand Disease.

Not a fit: Patients with mild or moderate forms of Von Willebrand Disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of bleeding events in children with severe Von Willebrand Disease.

How similar studies have performed: Other studies have shown promise in using recombinant factors for bleeding disorders, indicating potential success for this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]):

  * Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or
  * Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity \[FVIII:C\] \<10 % and historically documented genetics), Type 2M; or
  * Type 3 (VWF:Ag less than or equal to \[=\<\] 3 IU/dL).
* Age 0 to \<18 years at the time of Screening.
* The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
* If female of childbearing potential, participant presents with a negative serum pregnancy test.
* If applicable, participant agrees to employ adequate birth control measures for the duration of the study.
* The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements.

Additional inclusion criteria for both previously treated participants and participants undergoing surgery are as follows:

* Unable to tolerate are inadequately responsive to, or not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of participants who are not good candidates for DDAVP include participants with type 2B or type 3 VWD.
* The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (i.e. treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.

Additional inclusion criterion for previously untreated participants are as follows:

\- The participant has not received prior VWF coagulation factor replacement therapy.

Exclusion Criteria:

* Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time \[PT\]/international normalized ratio \[INR\] greater than \[\>\] 1.4).
* History or presence of a VWF inhibitor at Screening.
* History or presence of a Factor VIII (FVIII) inhibitor with a titer greater than or equal \[\>=\] 0.4 Bethesda units (BU) (by Nijmegen assay) or \>=0.6 BU (by Bethesda assay).
* Documented history of a VWF: RCo half-life \<6 hours.
* Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins.
* Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/asthma, food allergies, or animal allergies.
* Medical history of a thromboembolic event.
* Human immunodeficiency virus (HIV) positive, with an absolute CD4 count \<200/ cubic millimeter (mm\^3).
* In the judgment of the Investigator, the participant has another clinically significant concomitant disease (e.g. uncontrolled hypertension, cancer) that may pose additional risks for the participant.
* Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) of 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C.
* Diagnosis of renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL).
* Immunomodulatory drug treatment other than anti-retroviral chemotherapy (e.g. α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram per day \[mg/day\] (excluding topical treatment \[e.g. ointments, nasal sprays\]), within 30 days prior to signing the informed consent (or assent, if appropriate).
* If female, participant is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained.
* Participant has participated in another clinical study involving an investigational product (IP), other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study.
* Participant's legal representative is a family member or employee of the Investigator.

Where this trial is running

Aurora, Colorado and 45 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Von Willebrand Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.