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, 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
- 26 sites in United States, Belgium and 12 more
- Volunteers
- 44 people
- Sponsor
- Hoffmann-La Roche
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 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
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), 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, 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
Von Willebrand Disease (VWD), Type 2
Efficacy and Safety of BT200 (Rondaptivon Pegol) in Patients With Type 2B Von Willebrand Disease
This randomized clinical trial with a cross-over design is being conducted at the Department of Clinical Pharmacology at the Medical University of Vienna, and a total of 4-6 patients with type 2B von Willebrand disease (VWD) will…
- Trial name
- BT200-VWD2B (NCT07273721)
- Testing
- BT200
- Where
- Austria
- Volunteers
- 6 people
- Sponsor
- Medical University of Vienna
Von Willebrand Disease, Type 1, Heavy Menstrual Bleeding
Delivering Transcutaneous Auricular Neurostimulation to Reduce Heavy Menstrual Bleeding in Patients With and Without Von Willebrand Disease
The objectives of this study are to determine if transcutaneous auricular neurostimulation (tAN) can modulate hemostasis, improve perceived quality of life, and improve pain during the menstrual cycle of von Willebrand Disease…
- Trial name
- VWD/HMB (NCT06064851)
- Testing
- Volta System
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Five Liters, Inc.
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, Type 2B
Impact of Von Willebrand Factor-platelet Aggregates in Patients With Type 2B Disease
The working hypothesis is that patients with Von Willebrand Disease-type 2B may have neurological symptoms due to the chronic formation of platelets/Von Willebrand Factor aggregates.
- Trial name
- Von2B (NCT05015244)
- Testing
- 3T (Tesla) Magnetic Resonance Imaging (MRI)
- Where
- France
- Volunteers
- 5 people
- Sponsor
- University Hospital, Caen
Von Willebrand Disease, Type 1
Evaluation of Bleeding Score in Egyptian Patients With vWD Type I and Correlate it With Laboratory Parameters
Applying the ISTH-BAT questionnaire on Egyptian patients with type I VWD aiming to correlate the BS with the laboratory findings
- Trial name
- NCT03915873
- Testing
- The von Willebrand Antigen level
- Volunteers
- 20 people
- Sponsor
- Assiut University
Purpura, Thrombotic Thrombocytopenic, Von Willebrand Disease Type-2b
ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders
To evaluate the overall safety and tolerability of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with three types of von Willebrand Factor related platelet disorders.
- Trial name
- NCT00632242
- Testing
- ARC1779
- Where
- Austria
- Volunteers
- 28 people
- Sponsor
- Archemix Corp.