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)
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
200
Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants 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 2A, Type 2M, Type 2N and Type 3.
Phoenix Children's Hospital
Phoenix, Arizona, United States
RECRUITINGArkansas Children's Hospital
Little Rock, Arkansas, United States
RECRUITINGChildren's Hospital of Los Angeles
Los Angeles, California, United States
NOT_YET_RECRUITINGUniversity of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida, United States
RECRUITINGEmory Children's Center
Atlanta, Georgia, United States
RECRUITINGInnovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
RECRUITINGTulane University School of Medicine
New Orleans, Louisiana, United States
RECRUITINGUniversity of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, United States
RECRUITINGMayo Clinic - Rochester
Rochester, Minnesota, United States
RECRUITINGOregon Health & Science University
Portland, Oregon, United States
RECRUITING...and 7 more locations
Annualized bleeding event rate
Time frame: 4.5 to 12.5 months
Annualized treated bleeding rate
Time frame: 4.5 to 12.5 months
Annualized heavy menstrual bleed rate
Time frame: 4.5 to 12.5 months
Number of overnight admissions
Hospitalization monitoring of bleeding events.
Time frame: 4.5 to 12.5 months
Prophylactic and on demand treatment
Details of treatment used for bleeding events
Time frame: 4.5 to 12.5 months
Iron status
Blood samples will be collected for the determination of ferritin and hemoglobin.
Time frame: 4.5 to 12.5 months
Patient-Reported Outcomes Measurement Information System (PROMIS)-29
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.
Time frame: 4.5 to 12.5 months
Menstrual Bleeding Questionnaire (MBQ)
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.
Time frame: 4.5 to 12.5 months
Epistaxis Severity Score (ESS)
Assessment of nose bleed severity
Time frame: 4.5 to 12.5 months
Oral Bleeding Experience
Questionnaire to evaluate bleeding symptoms
Time frame: 4.5 to 12.5 months
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