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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00936312
• Other study ID #: Intl Female Hemophilia Study
• Status: Completed
• Phase: N/A
• First received: July 9, 2009
• Last updated: December 6, 2012
• Start date: March 2008
• Est. completion date: August 2011

Study information

• Verified date: December 2012
• Source: Weill Medical College of Cornell University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 200
• Est. completion date: August 2011
• Est. primary completion date: August 2011
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A and older

Eligibility

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

Observational Model: Cohort, Time Perspective: Retrospective

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Locations

Country	Name	City	State
Australia	Princess Margaret Hospital for Children	Perth
Germany	University Clinic Bonn	Bonn
Germany	University Clinic Munich	Munich
Israel	Sheba Medical Center	Tel Hashomer
Italy	Instituto G. Gaslini	Genova
Italy	A. Bianchi Bonomi Hemophilia Center	Milan
Japan	Nara Medical University	Nara
Netherlands	University Hospital Utrecht	Utrecht
Sweden	Malmo University Hospital	Malmo
Taiwan	Changhua Christian Hospital	Chunghua City	Changhua
United States	Weill Cornell Medical College	New York	New York

Sponsors (3)

Lead Sponsor	Collaborator
Weill Medical College of Cornell University	Bayer, University Hospital, Bonn

Countries where clinical trial is conducted

United States,  Australia,  Germany,  Israel,  Italy,  Japan,  Netherlands,  Sweden,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No