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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 : June 24, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Chronic Granulomatous Disease Drug: Interferon Gamma-1B

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
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
Actual Study Start Date : May 16, 2018
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2019



Intervention Details:
  • 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


Primary Outcome Measures :
  1. 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

  2. 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.


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   5 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
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).
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03548818


Contacts
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Contact: Wendy Moore, MPH, CCRP 720-777-6353 wendy.moore@childrenscolorado.org

Locations
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United States, Colorado
University of Colorado Anschutz Medical Campus Recruiting
Aurora, Colorado, United States, 80045
Contact: Alison Lakin RN L LLM    303-724-1010    alison.lakin@ucdenver.edu   
Sponsors and Collaborators
University of Colorado, Denver
Investigators
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Principal Investigator: Daniel R. Ambruso, MD University of Colorado, Denver

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Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT03548818     History of Changes
Other Study ID Numbers: 17-1676
First Posted: June 7, 2018    Key Record Dates
Last Update Posted: June 24, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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

Additional relevant MeSH terms:
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Granuloma
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Interferons
Interferon-gamma
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents