Overview
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)
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.
Eligibility
Inclusion Criteria:
- 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 Criteria:
- 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.