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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
Sponsor:
Collaborators:
Texas Children's Hospital
Shire
Information provided by (Responsible Party):
Lakshmi Srivaths, Baylor College of Medicine

Tracking Information
First Submitted Date October 10, 2016
First Posted Date October 14, 2016
Last Update Posted Date January 15, 2019
Actual Study Start Date January 5, 2017
Estimated Primary Completion Date November 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 12, 2016)
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
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02933411 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: November 14, 2017)
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)
Original Secondary Outcome Measures
 (submitted: October 12, 2016)
Number of adolescents with Low VWF and HMB with genetic variations and bleeding phenotype (including high 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)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Low VW Activity in Adolescent HMB
Official Title Genotypic and Phenotypic Analysis of Adolescents With Heavy Menstrual Bleeding and Low Von Willebrand Activity
Brief Summary

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.

Detailed Description

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
Study Design Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood sample will be drawn to assess the number of participants enrolled that have heavy menstrual bleeding and low von willebrand activity disease causing sequence variation in the von willebrand factor gene and other genes affecting bleeding, clotting and blood vessel biology.
Sampling Method Non-Probability Sample
Study Population Females under the age of 21 years old with heavy menstrual bleeding and low von willebrand factor
Condition Von Willebrand Factor Deficiency
Intervention
  • 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
Study Groups/Cohorts Group A
Adolescent women with heavy menstrual bleeding and low von willebrand factor activity.
Interventions:
  • Other: Genetic Analysis
  • Other: Medical Record Data Abstraction
  • Other: Pictorial Blood Assessment Chart (PBAC) score
  • Other: Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 12, 2018)
120
Original Estimated Enrollment
 (submitted: October 12, 2016)
125
Estimated Study Completion Date November 2025
Estimated Primary Completion Date November 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

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)
Sex/Gender
Sexes Eligible for Study: Female
Ages up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Lakshmi V Srivaths, MD 832-822-4242 lvsrivat@txch.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02933411
Other Study ID Numbers H-39295 LOW VWF
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Lakshmi Srivaths, Baylor College of Medicine
Study Sponsor Baylor College of Medicine
Collaborators
  • Texas Children's Hospital
  • Shire
Investigators
Principal Investigator: Lakshmi V Srivaths, MD Texas Children's Hospital, an affiliate of Baylor College of Medicine
PRS Account Baylor College of Medicine
Verification Date January 2019