A Study Assessing HMB-002 in Participants With Von Willebrand Disease
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)
1 other identifier
interventional
108
3 countries
25
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Feb 2025
Typical duration for phase_1
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 19, 2024
CompletedFirst Posted
Study publicly available on registry
January 1, 2025
CompletedStudy Start
First participant enrolled
February 6, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 1, 2027
June 30, 2026
June 1, 2026
2.4 years
December 19, 2024
June 26, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incidence of Treatment emergent adverse events (TEAE)
up to Day 113
Secondary Outcomes (9)
Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax)
Day 1 to Day 113
Pharmacokinetic Parameter: Area under the curve from time zero to last quantifiable concentration (AUClast)
Day 1 to Day 113
Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf)
Day 1 to Day 113
Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax)
Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of VWF antigen (VWF:Ag)
Day 1 to Day 113
- +4 more secondary outcomes
Study Arms (3)
Part A Single Ascending Dose Design
EXPERIMENTALA multicenter study to evaluate the safety, tolerability, PK, and PD effect of single dose HMB-002 in participants with Type 1 VWD.
Part B Multiple Dose Assessment
EXPERIMENTALA multicenter study to evaluate the safety, tolerability, PK, and PD effect of repeat doses of HMB-002, as well as the preliminary prophylactic effects on bleeding events.
Part C HMB-002 with Concomitant Factor Concentrate
EXPERIMENTALA multicenter study to evaluate the safety and tolerability of a single dose of HMB-002, administered to patients concurrently receiving regular factor concentrate as standard of care.
Interventions
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 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 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.
Eligibility Criteria
You may qualify if:
- Weight 50 to 120 kg, inclusive.
- Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH/ASH) diagnostic guidelines).
- Vital signs are within normal ranges at Screening.
- Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening:
- Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL/min/1.73m\^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.
- Hematology \>85 g/L and platelet count \>120 x 10\^9/L.
- Part A Only:
- Age: ≥18 and \<70 years of age at the time of informed consent.
- 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
- Residual VWF activity of ≤ 50 IU/dL and FVIII activity ≤ 70 IU/dL during screening.
- Part B Only:
- Age: ≥16 and \<70 years of age at the time of informed consent.
- +12 more criteria
You may not qualify if:
- Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis.
- 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%.
- Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity).
- 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.
- Clinically significant cardiovascular disease.
- Other known severe bleeding disorder(s) other than VWD.
- 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.
- Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Hemab ApSlead
Study Sites (25)
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
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 19, 2024
First Posted
January 1, 2025
Study Start
February 6, 2025
Primary Completion (Estimated)
July 1, 2027
Study Completion (Estimated)
July 1, 2027
Last Updated
June 30, 2026
Record last verified: 2026-06