Von Willebrand Factor Deficiency Clinical Trial
— Low VWFOfficial title:
Genotypic and Phenotypic Analysis of Adolescents With Heavy Menstrual Bleeding and Low Von Willebrand Activity
Verified date | April 2022 |
Source | Baylor College of Medicine |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
This is a research study for patients diagnosed with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF). Menstruation, also known as a period, is the regular discharge of blood and tissues from the uterus. HMB is having a heavier amount of discharge during menstrual period. Low Von Willebrand Factor means that the participant has lower level of a blood protein that is important for clotting of blood and so, the participant is at a higher risk for bleeding. The purpose of this project is to study the genetic differences of adolescent females with HMB and low VWF activity and compare the genetic differences with their bleeding manifestations, response to medications and outcome.
Status | Active, not recruiting |
Enrollment | 120 |
Est. completion date | November 2025 |
Est. primary completion date | June 2020 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | N/A to 21 Years |
Eligibility | Inclusion Criteria: - Post-menarchal females less than 21 years of age - HMB defined as PBAC score greater than 100 - VWF:Activity more than or equal to 30 and less than or equal to 50 IU/dL x 2 - VWF: Activity /VWF:Ag ratio greater than or equal to 0.6 - Normal VW multimers, if performed Exclusion Criteria: - Post menarchal females age greater than or equal to 21 years - VWF: Activity less than 30 or greater than 50 IU/dL consistently, type 2 or type 3 VWD - Presence of other bleeding disorders (thrombocytopenia, platelet function defect, coagulation factor deficiency, fibrinogen defect or deficiency) |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital of Atlanta | Atlanta | Georgia |
United States | Hemophilia Center of Western New York | Buffalo | New York |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Nationwide Children's Hospital | Columbus | Ohio |
United States | University of Texas Southwestern Medical Center- Children's Medical Center | Dallas | Texas |
United States | Michigan State University | East Lansing | Michigan |
United States | Joseph M Sanzari Children's Hospital | Hackensack | New Jersey |
United States | Texas Children's Hospital | Houston | Texas |
United States | Children's Mercy Hospital | Kansas City | Missouri |
United States | Vanderbilt University Medical Center | Nashville | Tennessee |
United States | University of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Mary M. Gooley Hemophilia Center | Rochester | New York |
Lead Sponsor | Collaborator |
---|---|
Baylor College of Medicine | Shire |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of adolescents with Low VWF and HMB with genetic variations in VWF gene and other genes affecting bleeding, clotting and blood vessel biology | The genetic variations of adolescent females with heavy menstrual bleeding and low von Willebrand factor activity in VWF gene and other genes affecting bleeding, clotting and blood vessel biology | 3 years | |
Secondary | Number of adolescents with Low VWF and HMB with genetic variations and bleeding phenotype (including PBAC score and ISTH-BAT score, response to DDAVP challenge, HMB therapy) | The correlation of subjects with and without genetic variations with bleeding phenotype (including PBAC score, ISTH BAT score, response to DDAVP challenge and HMB therapy) | 3 years |