Actively Recruiting

Phase 1
Phase 2
Age: 18Years - 64Years
All Genders
NCT06754852

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

Led by Hemab ApS · Updated on 2025-12-09

108

Participants Needed

4

Research Sites

125 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 18Years - 64Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Ability to provide informed consent according to regulations
  • Willingness and ability to comply with study procedures and restrictions
  • Age 18 to under 65 years
  • Weight between 50 and 110 kg
  • Diagnosed with congenital Type 1, Type 1C, or Type 2A Von Willebrand Disease confirmed by lab results
  • Vital signs within specified ranges: resting pulse rate ≤105 bpm; systolic BP 90-140 mmHg; diastolic BP 40-90 mmHg
  • Female participants of childbearing potential must have a negative pregnancy test within 72 hours before first dose
  • Women of childbearing potential must use two medically acceptable contraceptive methods during the study; men with female partners of childbearing potential must use condoms plus one additional contraceptive method
  • Adequate baseline organ function shown by lab results: eGFR ≥45 ml/min/1.73m²; AST, ALT, and total bilirubin ≤1.5 times upper normal limit (or ≤2 times for Gilbert's Syndrome); hemoglobin >85 g/L and platelet count >120 x 10^9/L
  • Part B only: symptomatic participants with at least 3 treated bleeding events reported in observational study or medical record
  • Part B only: participants may enroll if they completed Part A follow-up
Not Eligible

You will not qualify if you...

  • History of severe allergic reactions to monoclonal antibody therapies
  • Personal history of venous or arterial thrombosis or thromboembolic disease, except superficial venous thrombosis related to catheter
  • High risk thrombophilia conditions including homozygous Factor V Leiden, compound heterozygous FVL/Prothrombin mutation, antithrombin <50%, congenital Protein C or S deficiency <50%
  • Need for ongoing hemostatic treatment to prevent bleeding except before procedures or surgery
  • Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV antibody with detectable RNA at screening
  • Receipt of any live vaccine within 28 days before consent or planned live vaccine during study
  • Planned major surgery during study
  • Body mass index over 35 kg/m² (adjusted for ethnicity)
  • Other conditions increasing thrombosis risk as judged by investigator
  • Pregnant or breastfeeding
  • Clinically significant cardiovascular disease
  • Current smokers unable to abstain from smoking throughout study
  • Other conditions increasing cardiovascular event risk as judged by investigator
  • Other congenital or acquired bleeding disorders besides specified VWD types
  • Concurrent diseases, treatments, or lab abnormalities that increase risk per investigator
  • Hypersensitivity to study drug or its components
  • Use of investigational medication within 5 half-lives before study drug
  • Use of drugs affecting hemostasis that cannot be stopped 14 days before and during study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 4 locations

1

Fiona Stanley Hospital

Murdoch, Perth, Australia, WA 6150

Not Yet Recruiting

2

Royal Prince Alfred Hospital

Camperdown, Sydney, Australia, NSW 2050

Actively Recruiting

3

The Alfred Hospital

Melbourne, Victoria, Australia, VIC 3004

Actively Recruiting

4

Richmond Pharmacology

London, United Kingdom, SE1 1YR

Actively Recruiting

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Research Team

C

Clinical Trials

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

2

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