Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease

Stopped early · Phase 4

Conditions studied: IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease, Immunodeficiency Disease

In brief

Background: \- Chronic granulomatous disease (CGD) is an immunodeficiency disease in which white blood cells are unable to kill certain bacteria and fungi. People with CGD are more likely to develop recurrent life-threatening infections. Certain changes or mutations in genes contribute to the severity of CGD, and also appear to affect the success of treatment with interferon-gamma, a substance that is used to improve the immune system s ability to fight infection. Researchers are interested in studying changes in the immune system caused by interferon-gamma treatment of CGD in individuals with different mutations that cause CGD. Objectives: \- To compare changes in the immune system caused by interferon-gamma treatment for CGD in individuals with different mutations that cause CGD. Eligibility: \- Individuals of any age who have been diagnosed with CGD and have specific types of mutations that cause CGD (to be determined after testing). Design: * Participants will be screened with a medical history, physical examination, and blood and urine tests. Participants must weigh more than 11 kilograms (\~24 pounds) to participate in the study. * Participants will receive injections of interferon-gamma once weekly for 4 weeks, twice weekly for 4 weeks, and then three times weekly for 4 weeks (a total of 24 injections). * Blood will be drawn periodically during treatment and for 8 weeks after the treatment, for a total of 21 weeks on the study. Participants will regularly provide information on their symptoms and responses to treatment to the study researchers.

Key facts

Study ID

NCT01147042

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

2

Starts

2010-05-18

Expected to finish

2014-11-14

Last updated by the study team

2017-09-20

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Subjects are excluded from the study who:
• Have undergone successful bone marrow transplantation;
• Had a serious adverse reaction to IFN gamma in the past;
• Are pregnant or breast feeding;
• Weigh less than 11 kg;
• Are currently on therapy with INF gamma;
• Have any of the following medical conditions:
• Coronary artery disease;
• Hepatic disease and/or liver enzymes elevated above 3 times normal;
• Seizure disorder, or
• Severe myelosuppression (absolute neutrophil count less than1000 cells/mm(3)).
• Participation of Minors: minor patients will be invited to participant in this study.
• Participation of Women: Exposure to IFN gamma by the developing human fetus may be detrimental. For this reason, women of childbearing-age will have a pregnancy test prior to undergoing study procedures. Should a woman become pregnant or suspect that she is pregnant while participating in this study, she should immediately inform study staff and her primary care physician.
• Pregnancy and Lactation: The effects of IFN gamma therapy on the developing fetus and newborn infant have not been studied. Therefore, it is not recommended that subjects who are pregnant or breast-feeding receive IFN gamma and they will be excluded from this study.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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