Protocol Summary

Protocol No.: OCR15577

Sponsor Protocol No.: 071102

Protocol Title.: BAX 111 rVWF in Pediatrics

Principal Investigator: Wynn, Tung

Objective: The purpose of this study in pediatric participants (<18 years of age) with severe hereditary von Willebrand disease (VWD) is: 1. To assess the efficacy, safety, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events 2. To assess the efficacy and safety of rVWF with ADVATE during elective or emergency surgery 3. To determine the pharmacokinetic (PK) profile of rVWF

Phase: Phase III

Age Group: Children

Age: N/A - 17 Years

Gender: All

Scope: National

Treatment:

Experimental: On-demand Treatment
Participants will receive treatment for non-surgical bleeding episodes.

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. Oral Surgery: infuse at least once within first 8-12 hours post-surgery. Major Surgery: infuse every 12-24 hours for at least first 72 hours post-surgery.

Experimental: Emergency Surgery
Within 3 hours prior to surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours. Oral Surgery: infuse at least once within first 8-12 hours post-surgery. Major Surgery: infuse every 12-24 hours for at least first 72 hours post-surgery.

Detailed Eligibility:

Inclusion Criteria:
1. Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor [VWF:RCo] <20%):
1. Type 1 (VWF:RCo <20 IU/dL); or
2. 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
3. Type 3 (VWF:Ag ≤3 IU/dL).
2. Age 0 to <18 years at the time of Screening
3. The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent
4. If female of childbearing potential, participant presents with a negative serum pregnancy test
5. If applicable, participant agrees to employ adequate birth control measures for the duration of the study
6. The participant and/or the legal representative is willing and able to comply with the requirements of the protocol
Additional inclusion criteria for previously treated participants and participants undergoing surgery are as follows:
1. Unable to tolerate or are inadequately responsive to deamino-delta-D-arginine vasopressin
2. The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to plasma-derived VWF
Additional inclusion criterion for previously untreated participants are as follows:
1. The participant has not received prior VWF coagulation factor replacement therapy
Exclusion Criteria:
1. 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] >1.4)
2. History or presence of a VWF inhibitor at Screening
3. History or presence of a Factor VIII (FVIII) inhibitor with a titer ≥0.4 Bethesda units (BU) (by Nijmegen assay) or ≥0.6 BU (by Bethesda assay)
4. Documented history of a VWF:RCo half-life <6 hours
5. Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins
6. Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/asthma, food allergies, or animal allergies
7. Medical history of a thromboembolic event
8. Human immunodeficiency virus (HIV) positive, with an absolute CD4 count <200/mm^3
9. In the judgment of the Investigator, the participant has another clinically significant concomitant disease (eg, uncontrolled hypertension, cancer) that may pose additional risks for the participant
10. 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 (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C
11. Diagnosis of renal disease, with a serum creatinine level ≥2.5 mg/dL
12. Immunomodulatory drug treatment other than anti-retroviral chemotherapy (eg, α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day (excluding topical treatment [eg, ointments, nasal sprays]), within 30 days prior to signing the informed consent (or assent, if appropriate)
13. If female, participant is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained
14. Participant has participated in another clinical study involving an investigational product (IP), other than recombinant von Willebrand Factor (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 or investigational device during the course of this study

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