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New hope for bleeding disorder: first human trial of HMB-002 begins

NCT ID NCT06754852

Disease control Sponsor: Hemab ApS Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times

Summary

This study tests a new medicine, HMB-002, in about 108 adults with von Willebrand disease (types 1 and 2). The goal is to check safety, how the drug moves in the body, and whether it helps control bleeding. It is the first time this drug is being tested in humans, starting with single doses and then repeated doses.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 108 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2025

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 to 69 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

von Willebrand disease (hereditary or acquired) von Willebrand disease 1 von Willebrand disease 2 von Willebrand disease 3 von Willebrand disease type 2A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Von Willebrand disease (VWD) Von Willebrand disease (VWD), type 1 Von Willebrand disease (VWD), type 2 Von Willebrand disease (VWD), type 3

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    25 sites in 3 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Arkansas Children's Hospital

    NOT_YET_RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Basingstoke and North Hampshire Hospital

    RECRUITING

    Basingstoke, Hampshire, RG24 9NA, United Kingdom

  • Children's Hospital of Los Angeles

    NOT_YET_RECRUITING

    Los Angeles, California, 90027, United States

  • Emory Children's Center

    NOT_YET_RECRUITING

    Atlanta, Georgia, 30329, United States

  • Fiona Stanley Hospital

    NOT_YET_RECRUITING

    Murdoch, Perth, WA 6150, Australia

  • Hemophilia Center of Western Pennsylvania

    NOT_YET_RECRUITING

    Pittsburgh, Pennsylvania, 15213, United States

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

    RECRUITING

    Indianapolis, Indiana, 46260, United States

  • Mayo Clinic - Rochester

    NOT_YET_RECRUITING

    Rochester, Minnesota, 55905, United States

  • Oregon Health & Science University

    NOT_YET_RECRUITING

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    NOT_YET_RECRUITING

    Phoenix, Arizona, 85016, United States

  • Richmond Pharmacology

    RECRUITING

    London, SE1 1YR, United Kingdom

  • Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre

    NOT_YET_RECRUITING

    Liverpool, L7 8XP, United Kingdom

  • Royal London Hospital

    NOT_YET_RECRUITING

    Whitechapel, London, E1 1FR, United Kingdom

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, Sydney, NSW 2050, Australia

  • St George's Hospital

    NOT_YET_RECRUITING

    Tooting, London, SW17 0QT, United Kingdom

  • St James's University Hospital, Leeds Haemophilia Centre

    NOT_YET_RECRUITING

    Leeds, LS9 7TF, United Kingdom

  • St Thomas' Hospital

    NOT_YET_RECRUITING

    London, SE1 7EH, United Kingdom

  • The Alfred Hospital

    RECRUITING

    Melbourne, Victoria, VIC 3004, Australia

  • The University of Texas Southwestern Medical Center

    NOT_YET_RECRUITING

    Dallas, Texas, 75390, United States

  • Tulane University School of Medicine

    NOT_YET_RECRUITING

    New Orleans, Louisiana, 70112, United States

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • University Hospitals Birmingham NHS Foundation Trust

    NOT_YET_RECRUITING

    Birmingham, B15 2TH, United Kingdom

  • University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center

    NOT_YET_RECRUITING

    Miami, Florida, 33136, United States

  • University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders

    NOT_YET_RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Washington Institute For Coagulation (WIC)

    NOT_YET_RECRUITING

    Seattle, Washington, 98101, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.