A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants with Von Willebrand Disease (NCT06754852)

This study is for participants who have Von Willibrand Disease (VWD) and involves the use of a subcutaneous prophylactic treatment (HMB-002) to prevent and reduce the frequency and/or severity of bleeding events. The study is conducted in two parts. Part A: A single dose will be administered to determine the safety and tolerability of HMB-002 in participants with VWB. Part B: Three doses of HMB-002 will be given, one dose every four weeks, to determine safety and tolerability of the IP and capture information regarding any bleeding events experienced.

Trial phase

Phase 1

Trial participation type

This trial has been designed for the Clinical Research of a Drug.

Principal investigator

Prof Huyen Tran

Key inclusion data

Congenital Type 1 VWD, Type 1C and Type 2A VWD, as follows: Part A (Cohorts A1 and A2) only Type 1 VWD disease. Part A (Cohorts A3 and A4) and Part B Type 1 VWD disease, Type 1C, and Type 2A VWD. All participants must have: Documented laboratory results confirming their diagnosis (consistent with International Society on Thrombosis and Hemostasis /American Society of Hematology diagnostic guidelines); Residual VWF activity of ?40 IU/dL measured during screening; FVIII:C measured via chromogenic assay ?70 IU/dL measured during screening. VWF antigen, activity and FVIII activity levels may transiently be elevated for example due to exercise. Repeat samples (up to 3 within the screening period) may be performed to confirm levels of VWF activity and FVIII activity meeting inclusion requirements at screening. Part B only: Participants must be symptomatic (typically reporting bleeding events every month) plus must have participated in the Velora Discover study.