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

This study has been completed.
Sponsor:
Collaborators:
Bayer
University Hospital, Bonn
Information provided by (Responsible Party):
Weill Medical College of Cornell University
ClinicalTrials.gov Identifier:
NCT00936312
First received: July 9, 2009
Last updated: December 6, 2012
Last verified: December 2012
  Purpose

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.


Condition
Hemophilia A
Hemophilia B

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

Resource links provided by NLM:


Further study details as provided by Weill Medical College of Cornell University:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 200
Study Start Date: March 2008
Study Completion Date: August 2011
Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Groups/Cohorts
Females with Hemophilia
Females with severe or moderate Hemophilia A or B
Control group
Male subjects with severe Hemophilia A or B and female subjects with mild (20-60%) Hemophilia A or B.

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

Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Females with severe or moderate Hemophilia A (less than or equal to 5%) Females with severe or moderate Hemophilia B (less than or equal to 5%)

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00936312

Locations
United States, New York
Weill Cornell Medical College
New York, New York, United States, 10065
Australia
Princess Margaret Hospital for Children
Perth, Australia
Germany
University Clinic Bonn
Bonn, Germany
University Clinic Munich
Munich, Germany
Israel
Sheba Medical Center
Tel Hashomer, Israel
Italy
Instituto G. Gaslini
Genova, Italy
A. Bianchi Bonomi Hemophilia Center
Milan, Italy
Japan
Nara Medical University
Nara, Japan
Netherlands
University Hospital Utrecht
Utrecht, Netherlands
Sweden
Malmo University Hospital
Malmo, Sweden
Taiwan
Changhua Christian Hospital
Chunghua City, Changhua, Taiwan
Sponsors and Collaborators
Weill Medical College of Cornell University
Bayer
University Hospital, Bonn
Investigators
Principal Investigator: William B Mitchell, MD Weill Medical College of Cornell University
  More Information

No publications provided

Responsible Party: Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT00936312     History of Changes
Other Study ID Numbers: Intl Female Hemophilia Study
Study First Received: July 9, 2009
Last Updated: December 6, 2012
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 14, 2014