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

April 16, 2026 updated by: Hemab ApS

Velora Discover: A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Australia
    • Perth
      • Murdoch, Perth, Australia, WA 6150
    • Sydney
      • Camperdown, Sydney, Australia, NSW 2050
    • Victoria
      • Melbourne, Victoria, Australia, VIC 3004
        • Recruiting
        • The Alfred Hospital
        • Contact:
  • United Kingdom
      • London, United Kingdom, SE1 1YR
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
    • Arkansas
      • Little Rock, Arkansas, United States, 72202-3591
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital of Los Angeles
        • Contact:
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Recruiting
        • Emory Children's Center
        • Contact:
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Recruiting
        • Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
        • Contact:
    • Louisiana
      • New Orleans, Louisiana, United States, 70112-2699
        • Recruiting
        • Tulane University School of Medicine
        • Contact:
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
        • Contact:
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic - Rochester
        • Contact:
    • Oregon
      • Portland, Oregon, United States, 97239-3098
        • Recruiting
        • Oregon Health & Science University
        • Contact:
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • Hemophilia Center of Western Pennsylvania
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • The University of Texas Southwestern Medical Center
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98101
        • Recruiting
        • Washington Institute For Coagulation (WIC)
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants ≥16 years of age with VWD.

Description

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. Is 16 years and < 70 years at the time of screening.
  4. Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) <38.5 kg/m*2.
  5. Has Von Willebrand Disease: Type 1 VWD (including Type 1C VWD) or Type 2A VWD. All participants must have: Documented lab results confirming their diagnosis consistent with ISTH/ASH diagnostic guidelines; VWF Activity ≤30 IU/dL and FVIII activity ≤70 IU/dL during Screening.
  6. 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 history of clinically significant hypersensitivity associated with monoclonal antibody therapies.
  2. Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
  3. Has a high-risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/prothrombin gene mutation, antithrombin <50%, congenital protein C and protein S deficiency with levels <50%.
  4. Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding
  5. Has other known severe bleeding disorder(s) other than VWD.
  6. Planned major surgery during the study period.
  7. Has other conditions that substantially increase the risk of thrombosis either individually or in combination, at the discretion of the Investigator, including but not limited to: significant family history; BMI >30 and ≤38.5 kg/m² (moderately obese, adjusted for ethnicity and increased central adiposity); reduced mobility; active malignancy; major surgery within 6 weeks preceding Screening; or postpartum within 12 weeks preceding Screening.
  8. Is pregnant or plans to become pregnant within the next 6 months following informed consent sign off.
  9. Has clinically significant cardiovascular disease including, but not limited to: NYHA Class III or IV heart failure, coronary artery disease, uncontrolled arrythmia, moderate to severe valvular heart disease, peripheral vascular disease, and ischemic stroke.
  10. Has other combinations of conditions that substantially increase the risk of cardiovascular events at the discretion of the Investigator including, but not limited to, smoking, uncontrolled hyperlipidemia, and uncontrolled hypertension.
  11. Has any concurrent disease, treatment, medication (including but not limited to ongoing anticoagulation, antiplatelet therapy, or non-steroidal anti-inflammatory drugs or other drugs that affect hemostasis), 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.
  12. 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL)
Other: 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
VWD Type 2A, Type 2M, Type 2N, or Type 3
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized bleeding event rate
Time Frame: 4.5 to 12.5 months
4.5 to 12.5 months
Annualized treated bleeding rate
Time Frame: 4.5 to 12.5 months
4.5 to 12.5 months
Annualized heavy menstrual bleed rate
Time Frame: 4.5 to 12.5 months
4.5 to 12.5 months
Number of overnight admissions
Time Frame: 4.5 to 12.5 months
Hospitalization monitoring of bleeding events.
4.5 to 12.5 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Prophylactic and on demand treatment
Time Frame: 4.5 to 12.5 months
Details of treatment used for bleeding events
4.5 to 12.5 months
Patient-Reported Outcomes Measurement Information System (PROMIS)-29
Time Frame: 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.
4.5 to 12.5 months
Menstrual Bleeding Questionnaire (MBQ)
Time Frame: 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.
4.5 to 12.5 months
Epistaxis Severity Score (ESS)
Time Frame: 4.5 to 12.5 months
Assessment of nose bleed severity
4.5 to 12.5 months
Oral Bleeding Experience
Time Frame: 4.5 to 12.5 months
Questionnaire to evaluate bleeding symptoms
4.5 to 12.5 months
Iron status
Time Frame: 4.5 to 12.5 months
Blood samples will be collected for the determination of ferritin and hemoglobin levels.
4.5 to 12.5 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hemab ApS

Collaborators

PSI CRO

Investigators

  • Study Director: VP of Clinical Research, Hemab ApS

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 30, 2024

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2026

Study Registration Dates

First Submitted

September 13, 2024

First Submitted That Met QC Criteria

September 23, 2024

First Posted (Actual)

September 24, 2024

Study Record Updates

Last Update Posted (Actual)

April 21, 2026

Last Update Submitted That Met QC Criteria

April 16, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HMB-002-101_SCR

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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