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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

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.


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

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.


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Study Type : Observational
Estimated Enrollment : 120 participants
Observational Model: Other
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Group A
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




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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
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.


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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Females under the age of 21 years old with heavy menstrual bleeding and low von willebrand factor
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)

Information from the National Library of Medicine

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


Contacts
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Contact: Lakshmi V Srivaths, MD 832-822-4242 lvsrivat@txch.org

Locations
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United States, Georgia
Children's Hospital of Atlanta Recruiting
Atlanta, Georgia, United States, 30322
Contact: Robert Sidonio, MD         
United States, Michigan
Michigan State University Recruiting
East Lansing, Michigan, United States, 48823
Contact: Roshni Kulkarni, MD         
United States, Missouri
Children's Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64108
Contact: Mukta Sharma, MD         
United States, New Jersey
Joseph M Sanzari Children's Hospital Recruiting
Hackensack, New Jersey, United States, 07601
Contact: Frances Flug, MD, MBA         
United States, New York
Hemophilia Center of Western New York Recruiting
Buffalo, New York, United States, 14209
Contact: Shilpa Jain, MD, MPH         
Mary M. Gooley Hemophilia Center Recruiting
Rochester, New York, United States, 14621
Contact: Peter Kouides, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Eric Mullins, MD         
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Sarah O'Brien, MD         
United States, Pennsylvania
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Margaret Ragni, MD         
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
Contact: Allison Wheeler, MD         
United States, Texas
University of Texas Southwestern Medical Center- Children's Medical Center Recruiting
Dallas, Texas, United States, 75235
Contact: Ayesha Zia, MD         
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Lakshmi Srivaths, MD         
Sponsors and Collaborators
Baylor College of Medicine
Texas Children's Hospital
Shire
Investigators
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Principal Investigator: Lakshmi V Srivaths, MD Texas Children's Hospital, an affiliate of Baylor College of Medicine

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Responsible Party: Lakshmi Srivaths, Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02933411     History of Changes
Other Study ID Numbers: H-39295 LOW VWF
First Posted: October 14, 2016    Key Record Dates
Last Update Posted: January 15, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Lakshmi Srivaths, Baylor College of Medicine:
Bleeding Disorders
Heavy Menstrual Bleeding

Additional relevant MeSH terms:
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Von Willebrand Diseases
Uterine Hemorrhage
Uterine Diseases
Genital Diseases, Female
Hemorrhage
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Menorrhagia
Pathologic Processes
Menstruation Disturbances