Low VW Activity in Adolescent HMB

Active, not recruitingOBSERVATIONAL
Enrollment

120

Participants

Timeline

Start Date

January 5, 2017

Primary Completion Date

June 30, 2020

Study Completion Date

November 30, 2025

Conditions
Von Willebrand Factor Deficiency
Interventions
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

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.

Trial Locations (12)

14209

Hemophilia Center of Western New York, Buffalo

14621

Mary M. Gooley Hemophilia Center, Rochester

15213

University of Pittsburgh, Pittsburgh

30322

Children's Hospital of Atlanta, Atlanta

37232

Vanderbilt University Medical Center, Nashville

43205

Nationwide Children's Hospital, Columbus

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

48823

Michigan State University, East Lansing

64108

Children's Mercy Hospital, Kansas City

75235

University of Texas Southwestern Medical Center- Children's Medical Center, Dallas

77030

Texas Children's Hospital, Houston

07601

Joseph M Sanzari Children's Hospital, Hackensack

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Shire

INDUSTRY

lead

Baylor College of Medicine

OTHER