A Study Assessing HMB-002 in Participants With Von Willebrand Disease
Condition(s) studied
Investigational drug(s) / intervention(s)
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.
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.
HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US): HMB-002 will be administered as a single dose with a concomitant single dose of factor concentrate. The planned duration of study participants in Part C will be approximately 17 weeks.
Study summary
This is a first-in-human (FIH), Phase 1/2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) regimen 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. Part C will evaluate the safety, PK, and PD of a single concomitant dose of HMB-002 and factor concentrate with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII who use factor concentrate as prophylaxis.
Eligibility
Primary outcome measure(s)
- Incidence of Treatment emergent adverse events (TEAE) — up to Day 113
Trial sites (25)
| Facility | City | Region | Status |
|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | Not Yet Recruiting |
| Arkansas Children's Hospital | Little Rock | Arkansas | Not Yet Recruiting |
| Children's Hospital of Los Angeles | Los Angeles | California | Not Yet Recruiting |
| University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center | Miami | Florida | Not Yet Recruiting |
| Emory Children's Center | Atlanta | Georgia | Not Yet Recruiting |
| Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana | Recruiting |
| Tulane University School of Medicine | New Orleans | Louisiana | Not Yet Recruiting |
| University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders | Ann Arbor | Michigan | Not Yet Recruiting |
| Mayo Clinic - Rochester | Rochester | Minnesota | Not Yet Recruiting |
| Oregon Health & Science University | Portland | Oregon | Not Yet Recruiting |
| Hemophilia Center of Western Pennsylvania | Pittsburgh | Pennsylvania | Not Yet Recruiting |
| The University of Texas Southwestern Medical Center | Dallas | Texas | Not Yet Recruiting |
| Washington Institute For Coagulation (WIC) | Seattle | Washington | Not Yet Recruiting |
| Fiona Stanley Hospital | Murdoch | Perth | Not Yet Recruiting |
| Royal Prince Alfred Hospital | Camperdown | Sydney | Recruiting |
| The Alfred Hospital | Melbourne | Victoria | Recruiting |
| Basingstoke and North Hampshire Hospital | Basingstoke | Hampshire | Recruiting |
| St George's Hospital | Tooting | London | Not Yet Recruiting |
| Royal London Hospital | Whitechapel | London | Not Yet Recruiting |
| University Hospitals Birmingham NHS Foundation Trust | Birmingham | United Kingdom | Not Yet Recruiting |
| University Hospital of Wales | Cardiff | United Kingdom | Recruiting |
| St James's University Hospital, Leeds Haemophilia Centre | Leeds | United Kingdom | Not Yet Recruiting |
| Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre | Liverpool | United Kingdom | Not Yet Recruiting |
| Richmond Pharmacology | London | United Kingdom | Recruiting |
| St Thomas' Hospital | London | United Kingdom | Not Yet Recruiting |
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06754852 on ClinicalTrials.gov ↗ ← All trials in Australia