NCT06754852

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

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 108 (estimated)
• Sponsor: Hemab ApS · Industry
• Sex: All
• Age: 18 Years – 64 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Von Willebrand Disease (VWD)
• Von Willebrand Disease (VWD), Type 1
• Von Willebrand Disease (VWD), Type 2

Interventions

Type	Name	Description
DRUG	HMB-002 (Part A)	HMB-002 will be administered subcutaneously. Part A will utilize sentinel dosing. The planned duration of study participants in Part A is approximately 12 weeks.
DRUG	HMB-002 (Part B)	HMB-002 will be administered subcutaneously. Part B dosing intervals will be determined following evaluation of Part A results. The planned duration of study participants in Part B will be approximately 21 weeks.

Timeline

• Start date: 2025-02-06
• Primary completion: 2027-07-01
• Completion: 2027-07-01
• First posted: 2025-01-01
• Last updated: 2025-12-09

Locations

4 sites across 2 countries: Australia, United Kingdom

Regulatory

• FDA-regulated drug study