Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease

Completed

• Conditions: Chronic Granulomatous Disease

• Registration Number: NCT03548818
• Lead Sponsor: University of Colorado, Denver

Brief Summary

The overall goal of the study is to investigate the functional, biochemical, and gene expression effects of Interferon-gamma 1-b (IFN-γ) on the neutrophils of patients with Chronic Granulomatous Disease (CGD). The investigators hypothesize that the clinical effects demonstrated in patients with CGD treated with IFN-γ (decreased number and severity of infections) are the result of biochemical processes and upregulation of specific genes, which lead to enhanced functionality of this immune cell population.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-03-20
• Study Start: 2018-05-16
• Study First Submitted QC: 2018-05-24
• Study First Posted: 2018-06-07
• Primary Completion: 2020-07-15
• Study Completion: 2020-07-15
• Status Verified: 2021-01
• Last Update Submitted: 2021-01-20
• Last Update Posted: 2021-01-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

• Current treatment with IFN-γ or intent to treat with this drug

• Acute infections resolved

• Subject is off treatment medications

• Women of childbearing potential must have:

  • a negative urine beta-HCG, and
  • be willing to practice two forms of effective contraception for the duration of study participation

Exclusion Criteria

• Pregnancy,

• Breast-feeding, or

• Unwillingness to use effective contraception

• Recent vaccination (within the last fourteen days)

• Current infection or acute health condition requiring treatment

• Chronic inflammatory complications of CGD including:

  • gastric outlet or urinary tract obstruction,
  • pleuritis,
  • pericarditis,
  • inflammatory bowel disease, or
  • colitis requiring treatment.

• Un-associated chronic lung, liver, kidney, or cardiac disorders requiring active treatment

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Changes in Gene expression as measured with RNA and affimetrics gene chips in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ.	1 week off drug, 12 hours after a week of treatment	Determine changes in gene expression in neutrophils from patients with CGD.
Changes in Neutrophil bacterialcidal activity in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ.	1 week off drug, 12 hours after a week of treatment	Determine changes in neutrophil function in neutrophils from patients with CGD

Secondary Outcome Measures

Name	Time	Method
Differences in Neutrophil biochemical studies in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ.	1 week off drug, 12 hours after first dose of IFN-gamma; 12 hours after a week of treatment	Determine changes in biochemical tests in neutrophils from CGD patients.

Trial Locations

Locations (1)

University of Colorado Anschutz Medical Campus; 🇺🇸 Aurora, Colorado, United States