A Study Assessing HMB-002 in Participants With Von Willebrand Disease (NCT06754852)
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)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Phoenix Children's Hospital — Phoenix, Arizona
Age range: 16 Years to 69 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (25 Sites)
- Phoenix Children's Hospital — Phoenix, Arizona
- Arkansas Children's Hospital — Little Rock, Arkansas
- Children's Hospital of Los Angeles — Los Angeles, California
- University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center — Miami, Florida
- Emory Children's Center — Atlanta, Georgia
- Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center — Indianapolis, Indiana
- Tulane University School of Medicine — New Orleans, Louisiana
- University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders — Ann Arbor, Michigan
- Mayo Clinic - Rochester — Rochester, Minnesota
- Oregon Health & Science University — Portland, Oregon
- Hemophilia Center of Western Pennsylvania — Pittsburgh, Pennsylvania
- The University of Texas Southwestern Medical Center — Dallas, Texas
- Washington Institute For Coagulation (WIC) — Seattle, Washington
- Fiona Stanley Hospital — Murdoch, Perth
- Royal Prince Alfred Hospital — Camperdown, Sydney
- The Alfred Hospital — Melbourne, Victoria
- Basingstoke and North Hampshire Hospital — Basingstoke, Hampshire
- St George's Hospital — Tooting, London
- Royal London Hospital — Whitechapel, London
- University Hospitals Birmingham NHS Foundation Trust — Birmingham,
- University Hospital of Wales — Cardiff,
- St James's University Hospital, Leeds Haemophilia Centre — Leeds,
- Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre — Liverpool,
- Richmond Pharmacology — London,
- St Thomas' Hospital — London,
📞 Contact the Study Coordinator
Phone: 080 8304 6409
Email: clinicaltrials@hemab.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Von Willebrand Disease (VWD)
- A Study of Bleeding and Treatment in Participants With Von Willebrand Disease — Phoenix, Arizona
- Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD) — Little Rock, Arkansas
- A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD) — Birmingham, Alabama
- HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB — Los Angeles, California
Explore by condition: All Von Willebrand Disease (VWD) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.