Genetic Susceptibility to Factor VIII Inhibitors

Hemophilia Clinical Trial

Official title:

Factor VIII Inhibitor Formation: Identifying Predisposing Genetic Factors

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00344435
• Other study ID #: 999905160
• Secondary ID: 05-C-N160
• Status: Completed
• First received: June 23, 2006
• Last updated: April 4, 2018
• Start date: May 24, 2005
• Est. completion date: April 5, 2016

Study information

• Verified date: April 5, 2016
• Source: National Institutes of Health Clinical Center (CC)
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This international study will identify genetic factors that may influence the development of inhibitory antibodies in patients with hemophilia A after treatment with factor VIII. Bleeding episodes in patients with inhibitors are often more difficult to treat. Previous research indicates that genetic factors play a role in the development of inhibitors. A better understanding of the influence of genes in this treatment complication may be helpful in predicting, treating or preventing inhibitors.

People in families in which one or more members have severe factor VIII deficiency and one or more have a history of an inhibitor may be eligible for this study. Participants fill out a form with questions about the person's relationship to other family members taking part in the study. Those with hemophilia provide a brief medical history, including hemophilia-related information, inhibitor history and the presence of other conditions such as hepatitis C and HIV. All participants have a blood sample taken for laboratory and research tests.

Description:

In collaboration with investigators of the Hemophilia Inhibitor Genetic Study (HIGS) multicenter study and the University of Lund, University Hospital, Malmo, Sweden, we propose to assess the role of genetic variants in the process of developing inhibitors to Factor VIII in persons with hemophilia. The discovery of genetic associations offers the potential to direct clinical management in order to prevent inhibitor development and improve clinical care in patients with inhibitors.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1187
• Est. completion date: April 5, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 100 Years

Eligibility

• INCLUSION CRITERIA

DNA and relevant clinical data from properly consented hemophiliac subjects and family members (maximum estimated = 3500) will be provided to the LGD for genotyping and analysis.

EXCLUSION CRITERIA

No available subjects will be excluded to maximize power.

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A

Locations

Country	Name	City	State
Sweden	University Hospital, University of Lund	Malmo

Sponsors (1)

Lead Sponsor	Collaborator
National Cancer Institute (NCI)

Country where clinical trial is conducted

Sweden, 

References & Publications (3)

Astermark J, Berntorp E, White GC, Kroner BL; MIBS Study Group. The Malmö International Brother Study (MIBS): further support for genetic predisposition to inhibitor development in hemophilia patients. Haemophilia. 2001 May;7(3):267-72. — View Citation

Frommel D, Allain JP. Genetic predisposition to develop factor VIII antibody in classic hemophilia. Clin Immunol Immunopathol. 1977 Jul;8(1):34-8. — View Citation

Vermylen J. How do some haemophiliacs develop inhibitors? Haemophilia. 1998 Jul;4(4):538-42. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Collection from 3500 donors		Every six months