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

Completed

• Conditions: Hemophilia A; Hemophilia B

• Registration Number: NCT00936312
• Lead Sponsor: Weill Medical College of Cornell University

Brief 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.

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.

Study Timeline

• Study Start: 2008-03
• Study First Submitted: 2009-07-09
• Study First Submitted QC: 2009-07-09
• Study First Posted: 2009-07-10
• Primary Completion: 2011-08
• Study Completion: 2011-08
• Status Verified: 2012-12
• Last Update Submitted: 2012-12-06
• Last Update Posted: 2012-12-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Female
• Target Recruitment: 200

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.	1-2 visits

Trial Locations

Locations (11)

Weill Cornell Medical College; 🇺🇸 New York, New York, United States

University Clinic Bonn; 🇩🇪 Bonn, Germany

A. Bianchi Bonomi Hemophilia Center; 🇮🇹 Milan, Italy

Sheba Medical Center; 🇮🇱 Tel Hashomer, Israel

Instituto G. Gaslini; 🇮🇹 Genova, Italy

University Clinic Munich; 🇩🇪 Munich, Germany

Princess Margaret Hospital for Children; 🇦🇺 Perth, Australia

University Hospital Utrecht; 🇳🇱 Utrecht, Netherlands

Nara Medical University; 🇯🇵 Nara, Japan

Changhua Christian Hospital; 🇨🇳 Chunghua City, Changhua, Taiwan

Malmo University Hospital; 🇸🇪 Malmo, Sweden