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

This study has been completed.
Sponsor:
Collaborators:
CSL Behring
University of Pennsylvania
Information provided by (Responsible Party):
Weill Medical College of Cornell University
ClinicalTrials.gov Identifier:
NCT00936845
First received: July 9, 2009
Last updated: December 6, 2012
Last verified: December 2012
  Purpose

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
Hemophilia A
Hemophilia B

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

Resource links provided by NLM:


Further study details as provided by Weill Medical College of Cornell University:

Primary Outcome Measures:
  • The prevalence of females with severe or moderate hemophilia A or B in the United States. [ Time Frame: Study Duration ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 22
Study Start Date: April 2005
Study Completion Date: January 2010
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts
Females with Hemophilia
Females with severe or moderate Hemophilia A or 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.

  Eligibility

Ages Eligible for Study:   1 Month and older
Genders 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.

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00936845

Locations
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
Investigators
Principal Investigator: William B Mitchell, MD Weill Medical College of Cornell University
  More Information

No publications provided

Responsible Party: Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT00936845     History of Changes
Other Study ID Numbers: Females with Hemophilia
Study First Received: July 9, 2009
Last Updated: December 6, 2012
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on October 29, 2014