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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00936845
Recruitment Status : Completed
First Posted : July 10, 2009
Last Update Posted : December 10, 2012
CSL Behring
University of Pennsylvania
Information provided by (Responsible Party):
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.

Condition or disease
Hemophilia A Hemophilia B

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 Type : Observational
Actual Enrollment : 22 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study
Study Start Date : April 2005
Actual Primary Completion Date : January 2010
Actual Study Completion Date : January 2010

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Females with Hemophilia
Females with severe or moderate Hemophilia A or B.

Primary Outcome Measures :
  1. The prevalence of females with severe or moderate hemophilia A or B in the United States. [ Time Frame: Study Duration ]

Secondary Outcome Measures :
  1. Collect existing data on the genetic and/or chromosomal causes for severe and moderate hemophilia A and B in females in the United States. [ Time Frame: Study Duration ]
  2. 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. [ Time Frame: Study Duration ]
  3. Collect and analyze data on the health-related quality of life and psychosocial adaptation to disease of females in the United States. [ Time Frame: Study Duration ]

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Females with severe or moderate (less than 5%) hemophilia A or B.

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.

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 identifier (NCT number): NCT00936845

United States, California
City of Hope National Medical Center
Duarte, California, United States, 91010
United States, Colorado
Mountain States Regional Hemophilia and Thrombosis Center
Aurora, Colorado, United States, 80045
United States, District of Columbia
Georgetown University Hospital
Washington, District of Columbia, United States, 20007
United States, Georgia
Emory University Hemophilia Program Office
Atlanta, Georgia, United States, 30322
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Kentucky
University of Kentucky Hemophilia Treatment Center
Lexington, Kentucky, United States, 40536
United States, Massachusetts
Boston Hemophilia Center- Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
Henry Ford Hospital Adult Hemophilia and Thrombosis Treatment Center
Detroit, Michigan, United States, 48202
United States, New Jersey
Newark Beth Israel Medical Center
Newark, New Jersey, United States, 07112
United States, New York
Hemophilia Center of Western New York - Pediatric
Buffalo, New York, United States, 14222
Long Island Jewish Medical Center
New Hyde Park, New York, United States, 11040
Weill Cornell Medical College
New York, New York, United States, 10065
Mary M. Gooley Hemophilia Center, Inc.
Rochester, New York, United States, 14621
United States, Ohio
Children's Hospital Medical Center of Akron
Akron, Ohio, United States, 44308
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Oklahoma
Oklahoma Center for Bleeding Disorders
Oklahoma City, Oklahoma, United States, 73104
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Gulf States Hemophilia and Thrombophilia Center
Houston, Texas, United States, 77030
Puerto Rico
Puerto Rico Hemophilia Treatment Center
San Juan, Puerto Rico, 00936
Sponsors and Collaborators
Weill Medical College of Cornell University
CSL Behring
University of Pennsylvania
Principal Investigator: William B Mitchell, MD Weill Medical College of Cornell University

Responsible Party: Weill Medical College of Cornell University Identifier: NCT00936845     History of Changes
Other Study ID Numbers: Females with Hemophilia
First Posted: July 10, 2009    Key Record Dates
Last Update Posted: December 10, 2012
Last Verified: December 2012

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Factor VIII