Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

Completed

• Conditions: Hemophilia A; Hemophilia B

• Registration Number: NCT00936845
• Lead Sponsor: Weill Medical College of Cornell University

Brief Summary

To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.

Detailed Description

This study is designed as an epidemiological study with retrospective clinical and genetic data collection and prospective quality of life (QOL) data collections and genotyping of subjects without current molecular diagnosis. Data on the genetic/chromosomal etiology of the condition will be collected through chart review. For those subjects who have not had molecular or cytogenetic analysis, molecular and cytogenetic testing will be offered after genetic counseling. Those subjects who agree to have genetic testing will have 5-10 ml of blood drawn and sent to the cytogenetic laboratory at Weill Cornell Medical College for testing to determine the chromosomal complement, and 5 ml of blood sent to Dr. Arupa Ganguly at the University of Pennsylvania for the identification of a factor VIII or factor IX mutation, and X-chromosome inactivation studies.

Study Timeline

• Study Start: 2005-04
• Study First Submitted: 2009-07-09
• Study First Submitted QC: 2009-07-09
• Study First Posted: 2009-07-10
• Primary Completion: 2010-01
• Study Completion: 2010-01
• Status Verified: 2012-12
• Last Update Submitted: 2012-12-06
• Last Update Posted: 2012-12-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Female
• Target Recruitment: 22

Inclusion Criteria

• Females with severe (FVIII<0.01u/dl) or moderate (FVIII-0.01<0.06 u/dl) hemophilia A
• Females with severe (FIX<0.01u/dl) or moderate (FIX-0.01<0.06u/dl) hemophilia B
• Willingness to participate in the study.

Exclusion Criteria

• Subjects who do not meet the inclusion criteria with respect to gender or hemophilia severity.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The prevalence of females with severe or moderate hemophilia A or B in the United States.	Study Duration

Secondary Outcome Measures

Name	Time	Method
Collect existing data on the genetic and/or chromosomal causes for severe and moderate hemophilia A and B in females in the United States.	Study Duration
Collect and analyze data on the health-related quality of life and psychosocial adaptation to disease of females in the United States.	Study Duration
To determine the natural history of hemophilia in females by collecting and analyzing data on the symptoms, treatment patterns, and complications of hemophilia in this cohort.	Study Duration

Trial Locations

Locations (19)

City of Hope National Medical Center; 🇺🇸 Duarte, California, United States

Mountain States Regional Hemophilia and Thrombosis Center; 🇺🇸 Aurora, Colorado, United States

Georgetown University Hospital; 🇺🇸 Washington, District of Columbia, United States

Emory University Hemophilia Program Office; 🇺🇸 Atlanta, Georgia, United States

Northwestern University; 🇺🇸 Chicago, Illinois, United States

University of Kentucky Hemophilia Treatment Center; 🇺🇸 Lexington, Kentucky, United States

Boston Hemophilia Center- Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Henry Ford Hospital Adult Hemophilia and Thrombosis Treatment Center; 🇺🇸 Detroit, Michigan, United States

Newark Beth Israel Medical Center; 🇺🇸 Newark, New Jersey, United States

Hemophilia Center of Western New York - Pediatric; 🇺🇸 Buffalo, New York, United States

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