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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00936312

Enrollment

200

Registered

2009-07-10

Start date

2008-03-31

Completion date

2011-08-31

Last updated

2012-12-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A, Hemophilia B

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

FEMALE

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
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

Countries

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

Outcome results