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NCT06754852PHASE1, PHASE2Recruiting

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

Hemab ApS

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

Key Inclusion Criteria: 1. Weight 50 to 120 kg, inclusive. 2. Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH/ASH) diagnostic guidelines). 3. Vital signs are within normal ranges at Screening. 4. Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening: 1. Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL/min/1.73m\^2. 2. Hepatic: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total bilirubin ≤1.5 upper limit of normal (ULN) at Screening. For participants with a history of Gilbert's Syndrome, total bilirubin ≤2 × ULN. 3. Hematology \>85 g/L and platelet count \>120 x 10\^9/L. Part A Only: 5. Age: ≥18 and \<70 years of age at the time of informed consent. 6. VWD Subtype Eligibility: * Cohorts A1 and A2: Participants with Type 1 VWD, only. * Cohorts A3 and A4: Participants with Type 1 VWD (including Type 1C) and Type 2A VWD 7. Residual VWF activity of ≤ 50 IU/dL and FVIII activity ≤ 70 IU/dL during screening. Part B Only: 8. Age: ≥16 and \<70 years of age at the time of informed consent. 9. VWD Subtype Eligibility: Participants with Type 1 VWD (including Type 1C) and Type 2A. 10. Residual VWF activity of ≤50 IU/dL and FVIII activity ≤70 IU/dL during screening. 11. Symptomatic Disease: Participants must be symptomatic, typically reporting bleeding events on a monthly basis. 12. Bleeding History (must meet one of the following): 1. Prior Observational Study Participation: The participant must have participated in the observational study HMB-002-101\_SCR (VELORA Discover), have a minimum annualized treated bleeding event (ATBR) of 3; OR 2. Medical Record-Documented Bleeding History: The Investigator confirms that ≥3 treated bleeding events have been documented in the participant's medical record within the preceding 12 months. Part C Only: 13. Age: ≥18 and \<70 years of age at the time of informed consent. 14. Participants with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII activity levels (VWF activity \<5 IU/dL and FVIII activity \<10 IU/dL). 15. Receives regular VWF concentrate (at least 1/week) as part of their routine care (usual dose ≤50 IU/kg). Key Exclusion Criteria: 1. Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis. 2. High risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/Prothrombin gene mutation, Antithrombin deficiency with activity \<50%. Congenital Protein C and Protein S deficiency with levels \<50%. 3. Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity). 4. Presence of other conditions that substantially increase risk of thrombosis either individually (for participants \>65 years of age) or in combination (for participants ≤65 years of age), at the discretion of the Investigator or Medical Monitor. 5. Clinically significant cardiovascular disease. 6. Other known severe bleeding disorder(s) other than VWD. 7. Requirement for concomitant medications that affect hemostasis (including, but not limited to anticoagulation, antiplatelet agents, certain non-steroidal anti-inflammatory drugs) and cannot refrain from use for 14 days prior to the first dose of study drug and throughout the study. Exclusion Criteria for Part A and Part B Only 8. Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.

Interventions

DRUG

HMB-002 (Part A)

DRUG

HMB-002 (Part B)

DRUG

HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)

Interested in This Trial?

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Conditions

Von Willebrand Disease (VWD)Von Willebrand Disease (VWD), Type 1Von Willebrand Disease (VWD), Type 2Von Willebrand Disease (VWD), Type 3

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