Clinical Research Directory

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)

Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate

Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting. Children with VWD may experience frequent nosebleeds, easy bruising, prolonged bleeding after injuries or surgeries, and, in adolescent girls, heavy menstrual bleeding. The severity of symptoms varies widely depending on the type of the disease and the level of the clotting factor. Despite its clinical importance, data about the frequency, clinical presentation, and treatment outcomes of von Willebrand disease among children in Upper Egypt are limited. Early recognition and appropriate management are crucial to prevent complications, reduce hospital visits, and improve quality of life. This observational study aims to assess the frequency of von Willebrand disease among children attending Assiut University Children's Hospital, describe the different disease subtypes, and evaluate the clinical bleeding patterns and management strategies used in routine practice. The study will include children aged 0-18 years with suspected or confirmed VWD. Information will be collected from medical records and clinical evaluations, including bleeding symptoms, laboratory test results, disease classification, and treatment approaches. The results of this study are expected to improve understanding of von Willebrand disease in children in this region and support better diagnostic and therapeutic planning for affected patients.

Endothelial Colony-Forming Cells in Patients With VWD, AVWS and Healthy Subjects

The goal of this observational study is to learn how endothelial colony-forming cells (ECFCs) behave in people with von Willebrand disease (VWD), acquired von Willebrand syndrome (AVWS), and in healthy individuals.

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). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)

A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD)

The main aim of this study is to find out if VONVENDI is safe for adult Chinese participants with VWD. The study will also check how well VONVENDI helps control bleeding with or without product ADVATE in the participants who may need elective surgery or dental procedures. In addition, the study will also examine how VONVENDI is processed by the body (known as pharmacokinetic \[PK\]) and how the drug helps the body respond or improve a condition (pharmacodynamic \[PD\]). Participants will receive an initial dose of VONVENDI of 40 to 80 international units per kilogram (IU/kg) of body weight. If a participant's baseline factor VIII (FVIII) level is not high enough to help stop bleeding, VONVENDI will be given along with 30 to 45 IU/kg of ADVATE rFVIII. Participants will be in the study for approximately 14 months. During the study, participants will be followed up at clinics or over telephone calls.

A Study Assessing HMB-002 in Participants With Von Willebrand Disease

This is a first-in-human (FIH), Phase 1/2, open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study of HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) design to establish safety, tolerability, PK, and PD effect. In Part B of the study, the safety and tolerability of repeat dosing will be established prior to cohort expansion to explore efficacy.

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. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) product. In this study, participants will be treated with vonicog alfa (rVWF) for 12 months. During the study, participants will visit the study clinic 5 times after treatment initiation.

Algorithm-based Management to Reduce the Recurrence of GI Bleeding and Severe Epistaxis in Von Willebrand Disease

WILL MANAGE is a prospective multicenter controlled open label randomized trial comparing an algorithm-based multidisciplinary management to a standard of care to reduce the incidence of bleeding (GI bleeding or severe epistaxis) recurrence in von Willebrand disease.