Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
120 participants
OBSERVATIONAL
2017-01-05
2025-11-30
Brief Summary
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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.
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Detailed Description
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Data collection will occur from participant's medical records in regards to their low VWF activity and HMB medical history.
Participants will be asked to complete symptom questionnaires in regards to their HMB.
A blood sample will be collected to analyze how many participants have the disease causing sequence variation in the VWF gene and other genes affecting bleeding, clotting and blood vessel biology and correlated with their bleeding history.
The blood sample will be deidentified and stored indefinitely for future research.
Conditions
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Study Design
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OTHER
PROSPECTIVE
Study Groups
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Group A
Adolescent women with heavy menstrual bleeding and low von willebrand factor activity.
Genetic Analysis
Blood sample will be drawn to assess the number of participants enrolled that have sequence variation in the von willebrand factor gene and other genes affecting bleeding, clotting and blood vessel biology.
Medical Record Data Abstraction
The subject's response to intranasal or intravenous desmopressin (DDAVP) challenge performed as part of standard of care will be recorded. The desmopressin is a drug that increases the clotting factor in blood to prevent bleeding.
Medical and family history including and not limited to age, diagnoses, race/ethnicity, lab values, HMB and low VWF activity diagnoses, and treatment history and outcome will also be recorded.
Pictorial Blood Assessment Chart (PBAC) score
PBAC is a pictorial tool to assess menstrual blood loss. Study team will complete the PBAC assessment with each study participant evaluating and measure response to different treatments for menstrual blood loss in the clinic setting between those with VWD versus other bleeding disorders.
Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Study participants will complete the ISTH BAT assessment. The ISTH BAT is a questionnaire to aid in the standardized evaluation of the presence and severity of bleeding symptoms.
Interventions
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Genetic Analysis
Blood sample will be drawn to assess the number of participants enrolled that have sequence variation in the von willebrand factor gene and other genes affecting bleeding, clotting and blood vessel biology.
Medical Record Data Abstraction
The subject's response to intranasal or intravenous desmopressin (DDAVP) challenge performed as part of standard of care will be recorded. The desmopressin is a drug that increases the clotting factor in blood to prevent bleeding.
Medical and family history including and not limited to age, diagnoses, race/ethnicity, lab values, HMB and low VWF activity diagnoses, and treatment history and outcome will also be recorded.
Pictorial Blood Assessment Chart (PBAC) score
PBAC is a pictorial tool to assess menstrual blood loss. Study team will complete the PBAC assessment with each study participant evaluating and measure response to different treatments for menstrual blood loss in the clinic setting between those with VWD versus other bleeding disorders.
Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Study participants will complete the ISTH BAT assessment. The ISTH BAT is a questionnaire to aid in the standardized evaluation of the presence and severity of bleeding symptoms.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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)
21 Years
FEMALE
No
Sponsors
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Shire
INDUSTRY
Baylor College of Medicine
OTHER
Responsible Party
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Sarah Sartain
Assistant Professor
Principal Investigators
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Sarah Sartain, MD
Role: PRINCIPAL_INVESTIGATOR
Texas Children's Hospital, an affiliate of Baylor College of Medicine
Locations
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Children's Hospital of Atlanta
Atlanta, Georgia, United States
Michigan State University
East Lansing, Michigan, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Joseph M Sanzari Children's Hospital
Hackensack, New Jersey, United States
Hemophilia Center of Western New York
Buffalo, New York, United States
Mary M. Gooley Hemophilia Center
Rochester, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Texas Southwestern Medical Center- Children's Medical Center
Dallas, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Countries
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Other Identifiers
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H-39295 LOW VWF
Identifier Type: -
Identifier Source: org_study_id
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