BAX 111 rVWF in Pediatrics
Status: | Recruiting |
---|---|
Healthy: | No |
Age Range: | Any - 17 |
Updated: | 2/21/2018 |
Start Date: | December 18, 2017 |
End Date: | May 29, 2020 |
Contact: | Sanhita Abrol, PhD |
Email: | sanhita.abrol1@shire.com |
Phone: | +1 617-588-8550 |
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
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
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
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
We found this trial at
15
sites
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
Cincinnati, Ohio 45229
1-513-636-4200
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Rainbow Babies and Children's Hospital UH Rainbow Babies & Children’s Hospital is a 244-bed, full-service...
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Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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171 Ashley Avenue
Charleston, South Carolina 29425
Charleston, South Carolina 29425
843-792-1414
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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Univ of Nebraska Med Ctr A vital enterprise in the nation’s heartland, the University of...
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