Phase
Condition
Von Willebrand Disease
Treatment
Factor VIII (FVIII) Concentrates
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Bypassing Agents
Clinical Study ID
Ages > 1 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
Preexisting medical record verifying the status of von Willebrand factor (VWF)inhibitor (positive or negative, including titer if available)
Adequate hematologic, hepatic, and renal function
For participants of childbearing potential: agreement to remain abstinent or adhereto the contraception requirements
Additional Inclusion Criteria for Arms A and B:
Age ≥1 month at the time of signing Informed Consent/Assent Form
Documented previous use of on-demand therapy with intermittent (less than once aweek) on-demand SOC therapy for VWD
Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24weeks prior to enrollment
Additional Inclusion Criteria for Arm C:
Age ≥2 years at the time of signing Informed Consent/Assent Form
Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 timesweekly, as per prescribed dose) as described in the eligibility of Study WP45335
Have completed all study requirements as defined in the WP45335 protocol for atleast 24 weeks
Exclusion
Exclusion Criteria:
Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
History of gastrointestinal bleeding within 18 months prior to enrollment, or anyprevious diagnosis of angiodysplasia
History of intracranial hemorrhage
Previous or current treatment for thromboembolic disease or signs of thromboembolicdisease
Other conditions (e.g., certain autoimmune diseases) that may increase risk ofbleeding or thrombosis
History of clinically significant hypersensitivity associated with monoclonalantibody therapies or components of the emicizumab injection
Use of systemic immunomodulators (e.g., interferon) at enrollment or planned useduring the study, with the exception of anti-retroviral therapy
Study Design
Connect with a study center
UZ Leuven Gasthuisberg
Leuven, 3000
BelgiumActive - Recruiting
UZ Leuven Gasthuisberg
Leuven 2792482, 3000
BelgiumSite Not Available
The Hospital for Sick Children
Toronto, Ontario M5G 1X8
CanadaActive - Recruiting
The Hospital for Sick Children
Toronto 6167865, Ontario 6093943 M5G 1X8
CanadaSite Not Available
McGill University Health Center
Montreal, Quebec H4A 3J1
CanadaActive - Recruiting
McGill University Health Center
Montreal 6077243, Quebec 6115047 H4A 3J1
CanadaSite Not Available
IPS SURA Industriales Medellín
Medellín,
ColombiaActive - Recruiting
IPS SURA Industriales Medellín
Medellín 3674962,
ColombiaSite Not Available
Hopital Claude Huriez - CHU Lille
Lille, 59037
FranceActive - Recruiting
Hopital Claude Huriez - CHU Lille
Lille 2998324, 59037
FranceSite Not Available
Groupe Hospitalier Necker Enfants Malades
Paris, 75015
FranceActive - Recruiting
Groupe Hospitalier Necker Enfants Malades
Paris 2988507, 75015
FranceSite Not Available
Universitätsklinikum Bonn
Bonn, 53127
GermanyActive - Recruiting
Universitätsklinikum Bonn
Bonn 2946447, 53127
GermanySite Not Available
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg, 47051
GermanyActive - Recruiting
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg 2934691, 47051
GermanySite Not Available
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M., 60590
GermanyActive - Recruiting
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome, Lazio 00161
ItalyActive - Recruiting
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome 3169070, Lazio 3174976 00161
ItalySite Not Available
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy 20122
ItalyActive - Recruiting
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan 3173435, Lombardy 3174618 20122
ItalySite Not Available
AOU Careggi
Firenze, Toscana 50134
ItalySite Not Available
AOU Careggi
Florence, Tuscany 50134
ItalyActive - Recruiting
AOU Careggi
Florence 3176959, Tuscany 3165361 50134
ItalySite Not Available
Nagoya University Hospital
Aichi, 466-8560
JapanSite Not Available
Nagoya University Hospital
Aichi 11192139, 466-8560
JapanSite Not Available
Kurume University Hospital
Fukuoka, 830-0011
JapanActive - Recruiting
Kurume University Hospital
Fukuoka 1863967, 830-0011
JapanSite Not Available
Nagoya University Hospital
Nagoya, 466-8560
JapanActive - Recruiting
Nagoya University Hospital
Nagoya 1856057, 466-8560
JapanSite Not Available
Erasmus MC
Rotterdam, 3015 GD
NetherlandsActive - Recruiting
Erasmus MC
Rotterdam 2747891, 3015 GD
NetherlandsSite Not Available
Instytut Hematologii i Transfuzjologii
Warsaw, 02-776
PolandActive - Recruiting
Instytut Hematologii i Transfuzjologii
Warsaw 756135, 02-776
PolandSite Not Available
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, 2193
South AfricaActive - Recruiting
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg 993800, 2193
South AfricaSite Not Available
Hospital Universitario la Paz
Madrid, 28046
SpainActive - Recruiting
Hospital Universitario la Paz
Madrid 3117735, 28046
SpainSite Not Available
Hospital Universitario Virgen del Rocio
Sevilla, 41013
SpainSite Not Available
Hospital Universitario Virgen del Rocio
Seville, 41013
SpainActive - Recruiting
Hospital Universitario Virgen del Rocio
Seville 2510911, 41013
SpainSite Not Available
Sahlgrenska Universitetssjukhuset
Gothenburg, S-413 45
SwedenActive - Recruiting
Sahlgrenska Universitetssjukhuset
Gothenburg 2711537, S-413 45
SwedenSite Not Available
Great Ormond Street Hospital
London, WC1N 3JH
United KingdomActive - Recruiting
St Thomas' Hospital
London, SE1 7EH
United KingdomActive - Recruiting
Great Ormond Street Hospital
London 2643743, WC1N 3JH
United KingdomSite Not Available
St Thomas' Hospital
London 2643743, SE1 7EH
United KingdomActive - Recruiting
Manchester Royal Infirmary
Manchester, M13 9WL
United KingdomActive - Recruiting
Manchester Royal Infirmary
Manchester 2643123, M13 9WL
United KingdomSite Not Available
UC Davis
Sacramento, California 95817
United StatesActive - Recruiting
UC Davis
Sacramento 5389489, California 5332921 95817
United StatesSite Not Available
University of Florida
Gainesville, Florida 32610
United StatesActive - Recruiting
University of Florida
Gainesville 4156404, Florida 4155751 32610
United StatesSite Not Available
University of Minnesota Medical Center
Minneapolis, Minnesota 55455
United StatesActive - Recruiting
University of Minnesota Medical Center
Minneapolis 5037649, Minnesota 5037779 55455
United StatesSite Not Available
Washington University School of Medicine
St Louis, Missouri 63110
United StatesActive - Recruiting
Washington University School of Medicine
St Louis 4407066, Missouri 4398678 63110
United StatesSite Not Available

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