Actively Recruiting
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
Led by Baxalta now part of Shire · Updated on 2025-04-30
31
Participants Needed
46
Research Sites
438 weeks
Total Duration
On this page
Sponsors
B
Baxalta now part of Shire
Lead Sponsor
T
Takeda Development Center Americas, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (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 vonicog alfa 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.
CONDITIONS
Official Title
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of severe von Willebrand disease (VWD) with von Willebrand factor ristocetin cofactor (VWF:RCo) less than 20% for types 1, 2A, 2B, 2N, 2M, or type 3 with VWF:Ag ≤3 IU/dL
- Age 0 to less than 18 years at screening
- Participant has provided assent if appropriate and legally authorized representative has provided informed consent
- For females of childbearing potential, negative serum pregnancy test
- Agreement to use adequate birth control if applicable during the study
- Willing and able to comply with study protocol requirements
- Unable to tolerate or not responsive to DDAVP or not a good candidate for DDAVP
- At least 1 documented bleed requiring VWF replacement therapy in the past 12 months
- At least 3 exposure days to VWF replacement therapy historically
- For previously untreated participants, no prior VWF replacement therapy
You will not qualify if you...
- Diagnosis of pseudo-VWD or other hereditary/acquired coagulation disorders
- Presence or history of VWF or Factor VIII inhibitors above specified levels
- Documented VWF:RCo half-life less than 6 hours
- Known hypersensitivity to study drug components such as mouse or hamster proteins
- History of immunological disorders excluding common allergies
- History of thromboembolic events
- HIV positive with absolute CD4 count less than 200/mm3
- Other clinically significant diseases posing risk, such as uncontrolled hypertension or cancer
- Significant liver disease indicated by high ALT, hypoalbuminemia, portal vein hypertension, or advanced liver cirrhosis
- Renal disease with serum creatinine ≥2.5 mg/dL
- Recent immunomodulatory drug treatment excluding antiretroviral therapy
- Pregnant or lactating females at consent
- Participation in another investigational study within 30 days prior or planned during this study
- Legal representative is a family member or employee of the Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 46 locations
1
University of Colorado Hemophilia & Thrombosis Center
Aurora, Colorado, United States, 80045
Actively Recruiting
2
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Completed
3
University of Florida College of Medicine
Jacksonville, Florida, United States, 32610
Actively Recruiting
4
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States, 61615
Actively Recruiting
5
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States, 46260
Completed
6
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198
Completed
7
St. Jude Affiliate Clinic at Novant Health
Charlotte, North Carolina, United States, 28204
Completed
8
Comprehensive Cancer Center of Wake Forest Unversity
Winston-Salem, North Carolina, United States, 27157
Completed
9
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
10
Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States, 44106
Actively Recruiting
11
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
12
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Completed
13
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
14
Texas Children's Cancer and Hematology Center
Houston, Texas, United States, 77030
Actively Recruiting
15
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
16
Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, United States, 53225
Completed
17
Medizinische Universität Innsbruck
Innsbruck, Austria, 6020
Completed
18
AKH - Medizinische Universität Wien
Vienna, Austria, 1090
Actively Recruiting
19
UZ Leuven
Leuven, Belgium, 3000
Actively Recruiting
20
Fakultni nemocnice Brno
Brno, Czechia, 613 00
Completed
21
Hôpital Morvan
Brest, Finistere, France, 29609
Completed
22
Groupe Hospitalier Pellegrin - Hôpital Pellegrin
Bordeaux, France, 33000
Actively Recruiting
23
Groupement Hospitalier Est- Hôpital Louis Pradel
Bron, France, 69677
Actively Recruiting
24
CHU CAEN - Hôpital de la Côte de Nacre
Caen, France, 14033
Actively Recruiting
25
Groupement Hospitalier Sud - Hôpital Bicêtre
Le Kremlin-Bicêtre, France, 94270
Actively Recruiting
26
Hopital Cardiologique - CHU Lille
Lille, France, 59037
Actively Recruiting
27
CHU de Nantes Site Hotel Dieu
Nantes, France, 44093
Completed
28
Hôpital Necker - Enfants Malades
Paris, France, 75743
Actively Recruiting
29
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, Germany, 20246
Completed
30
Werlhof-Institut GmbH
Hanover, Germany, 30159
Completed
31
Medizinische Hochschule Hannover
Hanover, Germany, 30625
Completed
32
Azienda Ospedaliera Universitaria Careggi
Florence, Italy, 50134
Actively Recruiting
33
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, Italy, 20122
Actively Recruiting
34
Azienda Ospedaliera Pediatrica Santobono Pausillipon
Naples, Italy, 80122
Actively Recruiting
35
Ospedale Pediatrico Bambino Gesù
Roma, Italy, 00165
Actively Recruiting
36
Erasmus Medisch Centrum
Rotterdam, Netherlands, 3015 CN
Completed
37
SBEI HPE Altai State Medical University of MoH and SD
Barnaul, Russia, 656038
Completed
38
SAIH "Kemerovo Regional Clinical Hospital"
Kemerovo, Russia, 650066
Completed
39
FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA
Kirov, Russia, 610027
Active, Not Recruiting
40
Hospital General Universitario de Alicante
Alicante, Spain, 03010
Actively Recruiting
41
Hospital Universitari i Politecnic La Fe
Valencia, Spain, 46026
Actively Recruiting
42
Istanbul University Cerrahpasa Medical Faculty
Istanbul, Turkey (Türkiye), 34098
Actively Recruiting
43
Ege University Medical Faculty
Izmir, Turkey (Türkiye), 35100
Actively Recruiting
44
Ondokuz Mayis Univ. Med. Fac.
Samsun, Turkey (Türkiye), 55139
Actively Recruiting
45
SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
Lviv, Ukraine, 79044
Completed
46
Royal Manchester Children's Hospital
Manchester, Greater Manchester, United Kingdom, M13 9WL
Completed
Research Team
T
Takeda Contact
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here