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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00936845
• Other study ID #: Females with Hemophilia
• Status: Completed
• Phase: N/A
• First received: July 9, 2009
• Last updated: December 6, 2012
• Start date: April 2005
• Est. completion date: January 2010

Study information

• Verified date: December 2012
• Source: Weill Medical College of Cornell University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: January 2010
• Est. primary completion date: January 2010
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 1 Month and older

Eligibility

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

Observational Model: Cohort, Time Perspective: Retrospective

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Locations

Country	Name	City	State
Puerto Rico	Puerto Rico Hemophilia Treatment Center	San Juan
United States	Children's Hospital Medical Center of Akron	Akron	Ohio
United States	Emory University Hemophilia Program Office	Atlanta	Georgia
United States	Mountain States Regional Hemophilia and Thrombosis Center	Aurora	Colorado
United States	Boston Hemophilia Center- Children's Hospital	Boston	Massachusetts
United States	Hemophilia Center of Western New York - Pediatric	Buffalo	New York
United States	Northwestern University	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Henry Ford Hospital Adult Hemophilia and Thrombosis Treatment Center	Detroit	Michigan
United States	City of Hope National Medical Center	Duarte	California
United States	Gulf States Hemophilia and Thrombophilia Center	Houston	Texas
United States	University of Kentucky Hemophilia Treatment Center	Lexington	Kentucky
United States	Long Island Jewish Medical Center	New Hyde Park	New York
United States	Weill Cornell Medical College	New York	New York
United States	Newark Beth Israel Medical Center	Newark	New Jersey
United States	Oklahoma Center for Bleeding Disorders	Oklahoma City	Oklahoma
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Mary M. Gooley Hemophilia Center, Inc.	Rochester	New York
United States	Georgetown University Hospital	Washington	District of Columbia

Sponsors (3)

Lead Sponsor	Collaborator
Weill Medical College of Cornell University	CSL Behring, University of Pennsylvania

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The prevalence of females with severe or moderate hemophilia A or B in the United States.		Study Duration	No
Secondary	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	No
Secondary	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	No
Secondary	Collect and analyze data on the health-related quality of life and psychosocial adaptation to disease of females in the United States.		Study Duration	No