Von Willebrand Disease (VWD)Phase 1/2NCT06754852
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
A Phase 1/2 study testing HMB-002 (Part A), HMB-002 (Part B) and HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US) in people with Von Willebrand Disease (VWD) and Von Willebrand Disease (VWD), Type 1, run by Hemab ApS at 25 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 69 years
- Conditions the study is for
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2
- What is being tested?
- HMB-002 (Part A), HMB-002 (Part B) and HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 108 participants
- Where
- 25 sitesin 3 countries
- Study dates
- 6 Feb 2025to Jul 2027
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 69 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
- VWD Subtype Eligibility: Participants with Type 1 VWD (including Type 1C) and Type 2A.
- Residual VWF activity of ≤50 IU/dL and FVIII activity ≤70 IU/dL during screening.
- Symptomatic Disease: Participants must be symptomatic, typically reporting bleeding events on a monthly basis.
- Bleeding History (must meet one of the following):
- Prior Observational Study Participation:
- The participant must have participated in the observational study HMB-002-101SCR (VELORA Discover), have a minimum annualized treated bleeding event (ATBR) of 3; OR
- 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:
- Age: ≥18 and <70 years of age at the time of informed consent.
- 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).
- Receives regular VWF concentrate (at least 1/week) as part of their routine care (usual dose ≤50 IU/kg).
Who cannot join
- 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.
Trial locations and contacts
25 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
25 sites in 3 countries
United States13 sites, 13 recruiting
- Not yet recruiting
University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, 48109
- Not yet recruiting
Emory Children's Center
Atlanta, Georgia, 30329
- Not yet recruiting
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
- Not yet recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
- Not yet recruiting
Children's Hospital of Los Angeles
Los Angeles, California, 90027
- Not yet recruiting
University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
- Not yet recruiting
Tulane University School of Medicine
New Orleans, Louisiana, 70112
- Not yet recruiting
Phoenix Children's Hospital
Phoenix, Arizona, 85016
- Not yet recruiting
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
- Not yet recruiting
Oregon Health & Science University
Portland, Oregon, 97239
- Not yet recruiting
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
- Not yet recruiting
Washington Institute For Coagulation (WIC)
Seattle, Washington, 98101
United Kingdom9 sites, 9 recruiting
- Recruiting
Basingstoke and North Hampshire Hospital
Basingstoke, Hampshire, RG24 9NA
- Not yet recruiting
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2TH
- Recruiting
University Hospital of Wales
Cardiff, CF14 4XW
- Not yet recruiting
St James's University Hospital, Leeds Haemophilia Centre
Leeds, LS9 7TF
- Not yet recruiting
Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre
Liverpool, L7 8XP
- Recruiting
Richmond Pharmacology
London, SE1 1YR
- Not yet recruiting
St Thomas' Hospital
London, SE1 7EH
- Not yet recruiting
St George's Hospital
Tooting, London, SW17 0QT
- Not yet recruiting
Royal London Hospital
Whitechapel, London, E1 1FR
Australia3 sites, 3 recruiting
- Recruiting
Royal Prince Alfred Hospital
Camperdown, Sydney, NSW 2050
- Recruiting
The Alfred Hospital
Melbourne, Victoria, VIC 3004
- Not yet recruiting
Fiona Stanley Hospital
Murdoch, Perth, WA 6150
About this study
Official title: 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)
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.
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Being tested
- HMB-002 (Part A), HMB-002 (Part B), HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)
- Keywords
- Von Willebrand Disease (VMD), Type 1 VWD, Type 1 with low residual VWF and FVIII who use factor concentrate as prophylaxis, Type 2 VWD, Type 3 VWD, Von Willebrand Factor (VWF)
- Medical subject headings
- von Willebrand Diseases, von Willebrand Disease, Type 3
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HMB-002 (Part A)Drug
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)Drug
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)Drug
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 group | Role | What they receive |
|---|---|---|
| Part A Single Ascending Dose Design | Experimental | A multicenter study to evaluate the safety, tolerability, PK, and PD effect of single dose HMB-002 in participants with Type 1 VWD. Receives: Drug: HMB-002 (Part A) |
| Part B Multiple Dose Assessment | Experimental | A 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. Receives: Drug: HMB-002 (Part B) |
| Part C HMB-002 with Concomitant Factor Concentrate | Experimental | A 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. Receives: Drug: HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment emergent adverse events (TEAE)
Measured: up to Day 113
Secondary outcomes (9)
Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax)
Measured: Day 1 to Day 113
Pharmacokinetic Parameter: Area under the curve from time zero to last quantifiable concentration (AUClast)
Measured: Day 1 to Day 113
Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf)
Measured: Day 1 to Day 113
Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax)
Measured: Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of VWF antigen (VWF:Ag)
Measured: Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of VWF activity
Measured: Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of FVIII activity
Measured: Day 1 to Day 113
Annualized Bleeding Rate Assessments
Measured: Day 1 to Day 113
Pharmacokinetic Parameter: Terminal elimination half-life (t1/2)
Measured: Day 1 to Day 113
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jan 2025
- Study start6 Feb 2025
- Primary completionJul 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 108 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hemab ApS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06754852
- Sponsor's protocol ID
- HMB-002-102
- Status last verified
- Jun 2026
- First submitted
- 19 Dec 2024
- Last updated
- 30 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No