Clinical trials: Von Willebrand Disease, Type 3
Von Willebrand Disease, Type 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been…
- Trial name
- WILL-EMI (NCT06998524)
- Testing
- Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates
- Where
- 29 sites in United States, Belgium and 12 more
- Volunteers
- 75 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
This is a first-in-human (FIH), Phase 1/2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD.
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Trial name
- NCT06754852
- Testing
- HMB-002 (Part A), HMB-002 (Part B), HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)
- Where
- 25 sites in United States, Australia and 1 more
- Volunteers
- 108 people
- Sponsor
- Hemab ApS
Type 3 Von Willebrand's Disease
Type 3 Von Willebrand International Registries Inhibitor Prospective Study
International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand…
- Trial name
- 3WINTERS-IPS (NCT02460458)
- Testing
- Von Willebrand Factor
- Where
- 25 sites in Finland, France and 8 more
- Volunteers
- 265 people
- Sponsor
- Fondazione Angelo Bianchi Bonomi
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
VWD - Von Willebrand's Disease
The Severe Von Willebrand Disease (sVWD) Patient Registry
A web-based registry will be created by the sponsor, VWD Connect Foundation (VCF), to collect data on patients with severe Von Willebrand Disease (sVWD).
- Trial name
- NCT05437536
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- VWD Connect Foundation
Von Willebrand Disease (VWD)
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
- Trial name
- VIVID-6 (NCT07115004)
- Testing
- VGA039
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 60 people
- Sponsor
- Vega Therapeutics, Inc
Von Willebrand Disease, Type 3, Concomitant VWD and Hemophilia
Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges.
- Trial name
- BCDI-XII (NCT05500807)
- Testing
- Emicizumab
- Where
- 12 sites in United States
- Volunteers
- 40 people
- Sponsor
- Bleeding and Clotting Disorders Institute Peoria, Illinois
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD).
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Trial name
- NCT06610201
- Testing
- Clinical outcomes of patients with VWD, Type 1, Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 200 people
- Sponsor
- Hemab ApS
Von Willebrand Disease, Type 3
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants…
- Trial name
- WILL-EMI NIS (NCT06883240)
- Testing
- Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates
- Where
- 25 sites in United States, Belgium and 11 more
- Volunteers
- 44 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Disease
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
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…
- Trial name
- NCT02932618
- Testing
- Vonicog alfa, Antihemophilic Factor (Recombinant)
- Where
- 46 sites in United States, Austria and 11 more
- Volunteers
- 47 people
- Sponsor
- Baxalta now part of Shire
Von Willebrand Disease (VWD)
A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor [rVWF]) in children.
- Trial name
- NCT05582993
- Testing
- Vonicog Alfa, ADVATE
- Where
- 23 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Takeda