Low VW Activity in Adolescent HMB (Low VWF)
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|ClinicalTrials.gov Identifier: NCT02933411|
Recruitment Status : Recruiting
First Posted : October 14, 2016
Last Update Posted : January 15, 2019
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.
|Condition or disease||Intervention/treatment|
|Von Willebrand Factor Deficiency||Other: Genetic Analysis Other: Medical Record Data Abstraction Other: Pictorial Blood Assessment Chart (PBAC) score Other: Complete Bleeding Symptom ISTH Bleeding Assessment Tool|
One hundred and twenty subjects will be enrolled. Adolescent females with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF) will be recruited.
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.
|Study Type :||Observational|
|Estimated Enrollment :||120 participants|
|Official Title:||Genotypic and Phenotypic Analysis of Adolescents With Heavy Menstrual Bleeding and Low Von Willebrand Activity|
|Actual Study Start Date :||January 5, 2017|
|Estimated Primary Completion Date :||November 2019|
|Estimated Study Completion Date :||November 2025|
Adolescent women with heavy menstrual bleeding and low von willebrand factor activity.
Other: 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.
Other: 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.
Other: 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.
Other Name: PBAC Score
Other: 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 Name: ISTH BAT Assessment
- Number of adolescents with Low VWF and HMB with genetic variations in VWF gene and other genes affecting bleeding, clotting and blood vessel biology [ Time Frame: 3 years ]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
- 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) [ Time Frame: 3 years ]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)
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02933411
|Contact: Lakshmi V Srivaths, MDfirstname.lastname@example.org|
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|Principal Investigator:||Lakshmi V Srivaths, MD||Texas Children's Hospital, an affiliate of Baylor College of Medicine|