Protocol Summary

Protocol No.: OCR15577

Sponsor Protocol No.: 071102

Protocol Title.: A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

Principal Investigator: Wynn, Tung

Objective: The main aim of the study is to check effectiveness, side effects, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with rVWF for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.

Phase: Phase III

Age Group: Children

Age: N/A - 17 Years

Gender: All

Scope: National

Treatment:

Experimental: On-demand Treatment
Participants will receive recombinant von Willebrand factor (rVWF) treatment for non-surgical bleeding episodes over a 12 to 18-month period.

Experimental: Elective Surgery
12-24 hours prior to surgery and within 3 hours of surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing.

Experimental: Emergency Surgery
Within 3 hours prior to surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing.

Detailed Eligibility:

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 ] 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 =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 rVWF 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 rVWF or investigational device during the course of this study. - Participant's legal representative is a family member or employee of the Investigator.

Applicable Conditions:

  • Pediatric von Willebrand Disease
  • Participation Institution:

  • UF Gainesville : Melissa Lingis
  • Contact:
    Melissa Lingis
    Phone: +1 352-294-8556
    Email: melissa.lingis@peds.ufl.edu

    More Information: View study listing on ClinicialTrials.gov http://www.clinicaltrials.gov/ct2/show/NCT02932618