Safety, tolerability, PK, PD, and efficacy of HMB-002 in participants with Von Willebrand Disease
Research type
Research Study
Full title
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants with Von Willebrand Disease
IRAS ID
1010089
Contact name
Catherine Rea
Contact email
Sponsor organisation
Hemab Aps
Research summary
Hemab ApS is funding this research to evaluate the ability of a new drug HMB-002, to help patients with Von Willebrand Disease (VWD). HMB-002 is a medicine intended to prevent and reduce bleeding events in patients with VWD.
Von Willebrand disease is an inherited bleeding disorder caused by a reduced amount of a plasma protein called Von Willebrand factor (VWF), causing an inability to properly form blood clots to stop bleeding. This leads to frequent bleeding episodes.
Many patients experience daily low volume bleeding that impacts their quality of life, while others may face more severe, potentially life-threatening bleeding. While there are drugs currently available to help stop bleeding once it has started, there are limited options to prevent bleeding from occurring in the first place. Current guidelines support preventative treatment with up to 3 times a week intravenous VWF concentrate infusion for only those with the most frequent, clinically severe bleeding events.
HMB-002 is being developed as a prophylactic treatment option to prevent and reduce the severity of bleeding events in patients with VWD when administered using a weekly to monthly subcutaneous injection regimen.
The main goal of this study is to look at the safety and tolerability of HMB-002. In addition, this clinical trial will also look at how long the drug remains in the body (pharmacokinetics), how it effects the body (pharmacodynamics), and whether it works for preventing bleeding (efficiency).
This is a multicentre clinical trial conducted in the UK, AUS and USA. This trial is comprised of two parts. Part A and Part B. Part A will include up to 48 participants with VWD and will evaluate a single dose of HMB-002. Part B will include up to 60 participants and will evaluate multiple doses of HMB-002. The planned duration for participation in Part A is approximately 12 weeks and Part B approximately 20 weeks.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
24/SC/0325
Date of REC Opinion
13 Jan 2025
REC opinion
Further Information Favourable Opinion