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

Recruiting

• Conditions: 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

• Registration Number: NCT06610201
• Lead Sponsor: Hemab ApS

Brief 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)

Detailed 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.

Study Timeline

• Study Start: 2024-08-30
• Study First Submitted: 2024-09-13
• Study First Submitted QC: 2024-09-23
• Study First Posted: 2024-09-24
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-11
• Last Update Posted: 2025-08-14
• Primary Completion: 2026-06
• Study Completion: 2026-06-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 200

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Annualized treated bleeding rate	4.5 to 12.5 months
Annualized heavy menstrual bleed rate	4.5 to 12.5 months
Number of overnight admissions	4.5 to 12.5 months	Hospitalization monitoring of bleeding events.
Annualized bleeding event rate	4.5 to 12.5 months

Secondary Outcome Measures

Name	Time	Method
Prophylactic and on demand treatment	4.5 to 12.5 months	Details of treatment used for bleeding events
Menstrual Bleeding Questionnaire (MBQ)	4.5 to 12.5 months	Measure the effect of heavy menstrual bleeding on a self-assessment of menstrual blood loss, limitations in social and leisure activities, physical activities, and work activities.
Iron status	4.5 to 12.5 months	Blood samples will be collected for the determination of ferritin and hemoglobin.
Patient-Reported Outcomes Measurement Information System (PROMIS)-29	4.5 to 12.5 months	An assessment which evaluates four items from each of the seven PROMIS domains. Each question is rated on a scale of 1 to 5. In addition, the PROMIS-29 includes one pain intensity question which is rated on a scale of 1 to 10. Higher scores on the PROMIS-29 indicate worse symptoms.
Epistaxis Severity Score (ESS)	4.5 to 12.5 months	Assessment of nose bleed severity
Oral Bleeding Experience	4.5 to 12.5 months	Questionnaire to evaluate bleeding symptoms

Trial Locations

Locations (15)

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center; 🇺🇸 Miami, Florida, United States

Emory Children's Center; 🇺🇸 Atlanta, Georgia, United States

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center; 🇺🇸 Indianapolis, Indiana, United States

Tulane University School of Medicine; 🇺🇸 New Orleans, Louisiana, United States

University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders; 🇺🇸 Ann Arbor, Michigan, United States

Mayo Clinic - Rochester; 🇺🇸 Rochester, Minnesota, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

The University of Texas Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States

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