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

NCT ID: NCT06610201

Last Updated: 2026-01-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-08-30

Study Completion Date

2026-06-30

Brief Summary

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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)

Detailed Description

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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.

Conditions

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Von Willebrand Disease (VWD) Von Willebrand Disease (VWD), Type 1 Von Willebrand Disease (VWD), Type 2 Von Willebrand Disease (VWD), Type 3 Von Willebrand Disease, Type 2A Von Willebrand Disease, Type 2M Von Willebrand Disease, Type 2N

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL)

Clinical outcomes of patients with VWD, Type 1

Intervention Type OTHER

Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1

VWD Type 2A, Type 2M, Type 2N, or Type 3

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

Intervention Type OTHER

Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2A, Type 2M, Type 2N and Type 3.

Interventions

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Clinical outcomes of patients with VWD, Type 1

Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1

Intervention Type OTHER

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

Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2A, Type 2M, Type 2N and Type 3.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
3. ≥ 16 years at the time of screening.
5. Has symptomatic disease as defined by a history of bruising or bleeding events, with an expected minimum of 3 bleeding episodes (including heavy menstrual bleeding) per year that require treatment to control bleeding symptoms, and/or has recurrent and ongoing episodes of heavy menstrual bleeding at the time of enrollment.

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 degree relatives.
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, or abnormality in clinical laboratory tests which may impact on the participant's bleeding symptoms or affect their ability to complete the study, in the Investigator's opinion.
7. Has received any investigational product within 30 days prior to screening. If the participant was enrolled and dosed in Velora Pioneer (study HMB-002-102; NCT06754852), they must have completed their End of Study Visit.
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hemab ApS

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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VP of Clinical Research

Role: STUDY_DIRECTOR

Hemab ApS

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status RECRUITING

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status NOT_YET_RECRUITING

University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center

Miami, Florida, United States

Site Status RECRUITING

Emory Children's Center

Atlanta, Georgia, United States

Site Status RECRUITING

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, United States

Site Status RECRUITING

Tulane University School of Medicine

New Orleans, Louisiana, United States

Site Status RECRUITING

University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders

Ann Arbor, Michigan, United States

Site Status RECRUITING

Mayo Clinic - Rochester

Rochester, Minnesota, United States

Site Status RECRUITING

Oregon Health & Science University

Portland, Oregon, United States

Site Status RECRUITING

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Washington Institute For Coagulation (WIC)

Seattle, Washington, United States

Site Status RECRUITING

Fiona Stanley Hospital

Murdoch, Perth, Australia

Site Status RECRUITING

Royal Prince Alfred Hospital

Camperdown, Sydney, Australia

Site Status RECRUITING

The Alfred Hospital

Melbourne, Victoria, Australia

Site Status RECRUITING

Richmond Pharmacology

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Australia United Kingdom

Central Contacts

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Clinical Trials (USA; UK)

Role: CONTACT

+1 888 493 8148; 080 8304 6409

Clinical Trials (Australia)

Role: CONTACT

+611800875216

Facility Contacts

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Taylor Janiec

Role: primary

602-933-0091

Maggie McDuffie

Role: primary

501-364-5436

Sibgha Zaheer

Role: primary

323-361-4908

Fernando Corrales-Medina

Role: primary

305-243-8652

Gabrille Dean

Role: primary

404-785-3153

Sanjay Ahuja

Role: primary

317-871-0000

Melody S. Benton

Role: primary

504-988-3596

Angela Weyand

Role: primary

734-764-9336

Rajiv Pruthi

Role: primary

507-266-1944

Kristina Haley

Role: primary

503-494-8716

Nicoletta Manchin

Role: primary

412-209-7280

Yu-Min Shen

Role: primary

214-648-1929

Abigail deLa Cruz

Role: primary

206-821-2594

Marina Goruppi

Role: primary

+61 (08) 6152 3420

Lindsey Kirby

Role: primary

+61 (02) 9515 7682

Christine Mazis

Role: primary

+61 (03) 9076 0890

Volunteer Recruitment Team

Role: primary

+44 (0) 20 7042 5800

Other Identifiers

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HMB-002-101_SCR

Identifier Type: -

Identifier Source: org_study_id

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