A Study Assessing HMB-002 in Participants With Von Willebrand Disease
Start Date
2/6/2025
Completion Date
7/1/2027
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 Criteria
Age Range: 16 years to 69 years
Interventions
HMB-002 (Part A)
HMB-002 (Part B)
HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)
Conditions
Locations
Phoenix Children's Hospital
Phoenix, Arizona 85016
United States
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United States
Children's Hospital of Los Angeles
Los Angeles, California 90027
United States
University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida 33136
United States
Emory Children's Center
Atlanta, Georgia 30329
United States
Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana 46260
United States
Tulane University School of Medicine
New Orleans, Louisiana 70112
United States
University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
Ann Arbor, Michigan 48109
United States
Mayo Clinic - Rochester
Rochester, Minnesota 55905
United States
Oregon Health & Science University
Portland, Oregon 97239
United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania 15213
United States
The University of Texas Southwestern Medical Center
Dallas, Texas 75390
United States
Washington Institute For Coagulation (WIC)
Seattle, Washington 98101
United States
Fiona Stanley Hospital
Murdoch, Perth WA 6150
Australia
Royal Prince Alfred Hospital
Camperdown, Sydney NSW 2050
Australia
The Alfred Hospital
Melbourne, Victoria VIC 3004
Australia
Basingstoke and North Hampshire Hospital
Basingstoke, Hampshire RG24 9NA
United Kingdom
St George's Hospital
Tooting, London SW17 0QT
United Kingdom
Royal London Hospital
Whitechapel, London E1 1FR
United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2TH
United Kingdom
University Hospital of Wales
Cardiff, CF14 4XW
United Kingdom
St James's University Hospital, Leeds Haemophilia Centre
Leeds, LS9 7TF
United Kingdom
Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre
Liverpool, L7 8XP
United Kingdom
Richmond Pharmacology
London, SE1 1YR
United Kingdom
St Thomas' Hospital
London, SE1 7EH
United Kingdom