Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
Information source: Weill Medical College of Cornell University
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hemophilia A; Hemophilia B
Phase: N/A
Status: Completed
Sponsored by: Weill Medical College of Cornell University Official(s) and/or principal investigator(s): William B Mitchell, MD, Principal Investigator, Affiliation: Weill Medical College of Cornell University
Summary
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.
Clinical Details
Official title: Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
Study design: Observational Model: Cohort, Time Perspective: Retrospective
Primary outcome: 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.
Detailed description:
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.
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Female.
Criteria:
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.
Locations and Contacts
Princess Margaret Hospital for Children, Perth, Australia
University Clinic Bonn, Bonn, Germany
University Clinic Munich, Munich, Germany
Sheba Medical Center, Tel Hashomer, Israel
Instituto G. Gaslini, Genova, Italy
A. Bianchi Bonomi Hemophilia Center, Milan, Italy
Nara Medical University, Nara, Japan
University Hospital Utrecht, Utrecht, Netherlands
Malmo University Hospital, Malmo, Sweden
Changhua Christian Hospital, Chunghua City, Changhua, Taiwan
Weill Cornell Medical College, New York, New York 10065, United States
Additional Information
Starting date: March 2008
Last updated: December 6, 2012
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