A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Purpose
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)
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
Eligibility
- Eligible Ages
- Over 16 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL) |
|
|
| VWD Type 2A, Type 2M, Type 2N, or Type 3 |
|
Recruiting Locations
Phoenix 5308655, Arizona 5551752 85016
Little Rock 4119403, Arkansas 4099753 72202-3591
Miami 4164138, Florida 4155751 33136
Atlanta 4180439, Georgia 4197000 30329
Indianapolis 4259418, Indiana 4921868 46260
New Orleans 4335045, Louisiana 4331987 70112-2699
Ann Arbor 4984247, Michigan 5001836 48109
Rochester 5043473, Minnesota 5037779 55905
Portland 5746545, Oregon 5744337 97239-3098
Pittsburgh 5206379, Pennsylvania 6254927 15213
Dallas 4684888, Texas 4736286 75390
Seattle 5809844, Washington 5815135 98101
More Details
- NCT ID
- NCT06610201
- Status
- Recruiting
- Sponsor
- Hemab ApS
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.