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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.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

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
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
Are there results?
Not yet: the study is under way

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.
Show all 27Show fewer
  • 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.

Clinical Trials contact

080 8304 6409

clinicaltrials@hemab.com

25 sites in 3 countries

United States13 sites, 13 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

    Not yet recruiting

  • Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center

    Indianapolis, Indiana, 46260

    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

    Not yet recruiting

Other trials in United States

United Kingdom9 sites, 9 recruiting

  • Basingstoke and North Hampshire Hospital

    Basingstoke, Hampshire, RG24 9NA

    Recruiting

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B15 2TH

    Not yet recruiting

  • University Hospital of Wales

    Cardiff, CF14 4XW

    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

    Not yet recruiting

  • Richmond Pharmacology

    London, SE1 1YR

    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

    Not yet recruiting

Other trials in United Kingdom

Australia3 sites, 3 recruiting

  • Royal Prince Alfred Hospital

    Camperdown, Sydney, NSW 2050

    Recruiting

  • The Alfred Hospital

    Melbourne, Victoria, VIC 3004

    Recruiting

  • Fiona Stanley Hospital

    Murdoch, Perth, WA 6150

    Not yet recruiting

Other trials in Australia

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.

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 groups in NCT06754852
Study groupRoleWhat they receive
Part A Single Ascending Dose DesignExperimentalA 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 AssessmentExperimentalA 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 ConcentrateExperimentalA 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.

  1. Incidence of Treatment emergent adverse events (TEAE)

    Measured: up to Day 113

Secondary outcomes (9)

  1. Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax)

    Measured: Day 1 to Day 113

  2. Pharmacokinetic Parameter: Area under the curve from time zero to last quantifiable concentration (AUClast)

    Measured: Day 1 to Day 113

  3. Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf)

    Measured: Day 1 to Day 113

  4. Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax)

    Measured: Day 1 to Day 113

  5. Pharmacodynamics Parameters: Assessment of VWF antigen (VWF:Ag)

    Measured: Day 1 to Day 113

  6. Pharmacodynamics Parameters: Assessment of VWF activity

    Measured: Day 1 to Day 113

  7. Pharmacodynamics Parameters: Assessment of FVIII activity

    Measured: Day 1 to Day 113

  8. Annualized Bleeding Rate Assessments

    Measured: Day 1 to Day 113

  9. 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.

  1. First posted1 Jan 2025
  2. Study start6 Feb 2025
  3. Primary completionJul 2027 (estimated)
  4. Study completionJul 2027 (estimated)

Resources

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)
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

Questions about this trial

What is this clinical trial studying?

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.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 27 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in United States, United Kingdom, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Clinical Trials (080 8304 6409, clinicaltrials@hemab.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment emergent adverse events (TEAE) (time frame: up to Day 113). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06754852.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jun 2026.

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