Status:
RECRUITING
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Lead Sponsor:
Hemab ApS
Conditions:
Von Willebrand Disease (VWD)
Von Willebrand Disease (VWD), Type 1
Eligibility:
All Genders
16+ years
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 (V...
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 enr...
Eligibility Criteria
Inclusion
- Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
- Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
- ≥ 16 years at the time of screening.
- 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.
- 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
- Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
- Has a significant family history of unprovoked thromboembolic events in first degree relatives.
- Has a congenital or acquired bleeding disorder other than VWD.
- Has planned major surgery within the next 6 months.
- Is pregnant or plans to become pregnant within the next 6 months.
- 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.
- 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.
Key Trial Info
Start Date :
August 30 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 1 2026
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT06610201
Start Date
August 30 2024
End Date
June 1 2026
Last Update
December 3 2025
Active Locations (17)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202-3591
3
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
4
University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida, United States, 33136