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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

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ClinicalTrials.gov Identifier: NCT03548818
Recruitment Status : Recruiting
First Posted : June 7, 2018
Last Update Posted : January 18, 2020
Sponsor:
Information provided by (Responsible Party):
University of Colorado, Denver

Tracking Information
First Submitted Date March 20, 2018
First Posted Date June 7, 2018
Last Update Posted Date January 18, 2020
Actual Study Start Date May 16, 2018
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 24, 2018)
  • 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. [ Time Frame: 1 week off drug, 12 hours after a week of treatment ]
    Determine changes in neutrophil function in neutrophils from patients with CGD
  • 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. [ Time Frame: 1 week off drug, 12 hours after a week of treatment ]
    Determine changes in gene expression in neutrophils from patients with CGD.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: May 24, 2018)
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. [ Time Frame: 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.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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
Official Title 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
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 Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population The Investigators plan to enroll children and adults, aged 5-60 years with CGD defined by functional and biochemical or genetic testing. Enrollment will be stratified by the four main variants: X-linked, gp91phox deficient patients, and autosomal recessive CGD (p47phox deficient, p67phox deficient, and p22phox deficient patients).
Condition Chronic Granulomatous Disease
Intervention Drug: Interferon Gamma-1B
Study function and biochemistry of neutrophils off the drug for one week and then 12 hours after first dose and fourth dose.
Other Name: None Noted
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: May 24, 2018)
20
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2020
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study: All
Ages 5 Years to 60 Years   (Child, Adult)
Accepts Healthy Volunteers Not Provided
Contacts
Contact: Wendy Moore, MPH, CCRP 720-777-6353 wendy.moore@childrenscolorado.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03548818
Other Study ID Numbers 17-1676
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party University of Colorado, Denver
Study Sponsor University of Colorado, Denver
Collaborators Not Provided
Investigators
Principal Investigator: Daniel R. Ambruso, MD University of Colorado, Denver
PRS Account University of Colorado, Denver
Verification Date January 2020