Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder. The study is designed to determine whether there are problems and issues unique to females with hemophilia.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia B Hemorrhagic Disorders

Sex eligibility

Female

Study type

Observational

Primary location

Princess Margaret Hospital for Children, Perth, Australia

About this study

The study involves two questionnaires: one on the diagnosis, symptoms, complications and treatment of each participant to be completed by a staff member; the other is a questionnaire to be completed by the participant on how the disease has affected her life. Finally, for those participants who have not previously had genetic testing, the third part of the study is an optional blood test to determine the genetic cause, what change in the factor VIII or factor IX gene, caused the hemophilia. The test results will be available to those participants who wish to learn their results. With the data we collect we will compile a database to examine the connection between the genetic cause of hemophilia and the course and symptoms of the disease.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Females with severe (FVIII<0.01u/ml) or moderate (FVIII 0.01≤0.05 u/ml) hemophilia A
Females with severe (FIX<0.01u/ml) or moderate (FIX 0.01≤0.05u/ml) 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.

Treatment and study plan

Primary outcomes

The molecular and cytogenetic etiology of the condition will be compiled. Clinical manifestation demonstrated by the female hemophiliacs will be compared to published data available. Genotype and phenotype will be correlated.

Time frame: 1-2 visits

Sponsors and collaborators

Lead sponsor

Weill Medical College of Cornell University

Other

Collaborators

Bayer
University Hospital, Bonn

Registry information

Important dates

Study start: 2008
Primary completion: 2011
Study completion: 2011
First posted: Jul 10, 2009
Registry last updated: Dec 10, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed