A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

Last updated: January 12, 2026
Sponsor: Hemab ApS
Overall Status: Active - Recruiting

Phase

N/A

Condition

Von Willebrand Disease

Treatment

Clinical outcomes of patients with VWD, Type 1 (less than 40 IU per dL)

Clinical outcomes of patients with VWD, Type 1

Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3

Clinical Study ID

NCT06610201
HMB-002-101_SCR
  • Ages > 16
  • All Genders

Study Summary

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Has the ability to provide informed consent to participate in the study, inaccordance with applicable regulations.

  2. Has an understanding, ability, and willingness to comply with Study procedures andrestrictions.

  3. ≥ 16 years at the time of screening.

  4. Has congenital Type 1 VWD with a residual VWF antigen and/or activity <30 IU/dLand/or meets the bleeding event rate inclusion criteria. Other congenital VWDsubtypes may be enrolled with Sponsor approval.

  5. Has symptomatic disease as defined by a history of bruising or bleeding events, withan expected minimum of 3 bleeding episodes (including heavy menstrual bleeding) peryear that require treatment to control bleeding symptoms, and/or has recurrent andongoing episodes of heavy menstrual bleeding at the time of enrollment.

Exclusion

Exclusion Criteria:

  1. Has a personal history of venous or arterial thrombosis or thromboembolic disease,except for catheter-associated, superficial vein thrombosis events.

  2. Has a significant family history of unprovoked thromboembolic events in first degreerelatives.

  3. Has a congenital or acquired bleeding disorder other than VWD.

  4. Has planned major surgery within the next 6 months.

  5. Is pregnant or plans to become pregnant within the next 6 months.

  6. Has any concurrent disease, treatment (including ongoing anticoagulation,antiplatelet, or non-steroidal anti-inflammatory drugs), condition, medication, orabnormality in clinical laboratory tests which may impact on the participant'sbleeding symptoms or affect their ability to complete the study, in theInvestigator's opinion.

  7. Has received any investigational product within 30 days prior to screening. If theparticipant was enrolled and dosed in Velora Pioneer (study HMB-002-102;NCT06754852), they must have completed their End of Study Visit.

Study Design

Total Participants: 200
Treatment Group(s): 6
Primary Treatment: Clinical outcomes of patients with VWD, Type 1 (less than 40 IU per dL)
Phase:
Study Start date:
August 30, 2024
Estimated Completion Date:
June 30, 2026

Study Description

This is a prospective, screening study in participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with confirmed Type 1 VWD and associated bleeding symptoms will be enrolled. The study may also be opened to participants with Type 2 and Type 3 VWD with Sponsor approval. Up to a total of 200 participants may be enrolled in the study.

The study includes screening, a baseline evaluation, and an approximately 4 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary entries and bleeding event treatments. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.

Connect with a study center

  • Fiona Stanley Hospital

    Murdoch, Perth WA 6150
    Australia

    Site Not Available

  • Fiona Stanley Hospital

    Murdoch 8349091, Perth WA 6150
    Australia

    Active - Recruiting

  • Royal Prince Alfred Hospital

    Camperdown, Sydney NSW 2050
    Australia

    Site Not Available

  • Royal Prince Alfred Hospital

    Camperdown 2172563, Sydney NSW 2050
    Australia

    Active - Recruiting

  • The Alfred Hospital

    Melbourne, Victoria VIC 3004
    Australia

    Site Not Available

  • The Alfred Hospital

    Melbourne 2158177, Victoria 2145234 VIC 3004
    Australia

    Active - Recruiting

  • Richmond Pharmacology

    London, SE1 1YR
    United Kingdom

    Site Not Available

  • Richmond Pharmacology

    London 2643743, SE1 1YR
    United Kingdom

    Active - Recruiting

  • Phoenix Children's Hospital

    Phoenix, Arizona 85016
    United States

    Site Not Available

  • Phoenix Children's Hospital

    Phoenix 5308655, Arizona 5551752 85016
    United States

    Active - Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202-3591
    United States

    Site Not Available

  • Arkansas Children's Hospital

    Little Rock 4119403, Arkansas 4099753 72202-3591
    United States

    Active - Recruiting

  • Children's Hospital of Los Angeles

    Los Angeles 5368361, California 5332921 90027
    United States

    Site Not Available

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

    Miami, Florida 33136
    United States

    Site Not Available

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

    Miami 4164138, Florida 4155751 33136
    United States

    Active - Recruiting

  • Emory Children's Center

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • Emory Children's Center

    Atlanta 4180439, Georgia 4197000 30329
    United States

    Active - Recruiting

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

    Indianapolis, Indiana 46260
    United States

    Site Not Available

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

    Indianapolis 4259418, Indiana 4921868 46260
    United States

    Active - Recruiting

  • Tulane University School of Medicine

    New Orleans, Louisiana 70112-2699
    United States

    Site Not Available

  • Tulane University School of Medicine

    New Orleans 4335045, Louisiana 4331987 70112-2699
    United States

    Active - Recruiting

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

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

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

    Ann Arbor 4984247, Michigan 5001836 48109
    United States

    Active - Recruiting

  • Mayo Clinic - Rochester

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • Mayo Clinic - Rochester

    Rochester 5043473, Minnesota 5037779 55905
    United States

    Active - Recruiting

  • Oregon Health & Science University

    Portland, Oregon 97239-3098
    United States

    Site Not Available

  • Oregon Health & Science University

    Portland 5746545, Oregon 5744337 97239-3098
    United States

    Active - Recruiting

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh 5206379, Pennsylvania 6254927 15213
    United States

    Active - Recruiting

  • The University of Texas Southwestern Medical Center

    Dallas, Texas 75390
    United States

    Site Not Available

  • The University of Texas Southwestern Medical Center

    Dallas 4684888, Texas 4736286 75390
    United States

    Active - Recruiting

  • Washington Institute For Coagulation (WIC)

    Seattle, Washington 98101
    United States

    Site Not Available

  • Washington Institute For Coagulation (WIC)

    Seattle 5809844, Washington 5815135 98101
    United States

    Active - Recruiting

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