Chronic Granulomatous DiseaseNCT03548818
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
An observational study of Chronic Granulomatous Disease, run by University of Colorado, Denver at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 5 to 60 years
- Conditions the study is for
- Chronic Granulomatous Disease
- What is being tested?
- Interferon Gamma-1B
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 16 May 2018to 15 Jul 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 60 years
- All sexes
- Child, Adult
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).
Who may be able to join
- 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
Who cannot join
- 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:
Show all 12Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
About this study
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.
- Conditions
- Chronic Granulomatous Disease
- Being tested
- Interferon Gamma-1B
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Interferon Gamma-1BDrug
Also called None Noted
Study function and biochemistry of neutrophils off the drug for one week and then 12 hours after first dose and fourth dose.
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Determine changes in neutrophil function in neutrophils from patients with CGD
Measured: 1 week off drug, 12 hours after a week of treatment
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.
Determine changes in gene expression in neutrophils from patients with CGD.
Measured: 1 week off drug, 12 hours after a week of treatment
Secondary outcomes (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.
Determine changes in biochemical tests in neutrophils from CGD patients.
Measured: 1 week off drug, 12 hours after first dose of IFN-gamma; 12 hours after a week of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2018
- Study start16 May 2018
- Primary completion15 Jul 2020
- Study completion15 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Daniel R. Ambruso, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03548818
- Sponsor's protocol ID
- 17-1676
- Status last verified
- Jan 2021
- First submitted
- 20 Mar 2018
- Last updated
- 22 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ambruso DR, Briones NJ, Tran AD, Sanford B, Childs C, Katz BZ, Ellison M, Johnston RB, Jones KL. Interferon-gamma 1b-induced gene expression alters neutrophil function in patients with chronic granulomatous disease. PLoS One. 2025 Sep 8;20(9):e0331657. doi: 10.1371/journal.pone.0331657. eCollection 2025.
PubMed 40920799
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03548818
- Acronym / sponsor ID
- 17-1676
- Conditions
- Chronic Granulomatous Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Study start
- 16 May 2018 (actual)
- Primary completion
- 15 Jul 2020 (actual)
- Study completion
- 15 Jul 2020 (actual)
- First posted
- 7 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 5 to 60 years
- Sex
- All
- Age groups
- Child, Adult
- 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).
Key inclusion criteria (6 listed)
- 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:
Key exclusion criteria (12 listed)
- Pregnancy,
- Breast-feeding, or
- Unwillingness to use effective contraception
- Recent vaccination (within the last fourteen days)
- Current infection or acute health condition requiring treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Interferon Gamma-1BAlso: None NotedStudy function and biochemistry of neutrophils off the drug for one week and then 12 hours after first dose and fourth dose. | Drug | – |
Primary outcome
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.
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2021.
- Derived from this study: PubMed 40920799Ambruso DR, Briones NJ, Tran AD, Sanford B, Childs C, Katz BZ, Ellison M, Johnston RB, Jones KL. Interferon-gamma 1b-induced gene expression alters neutrophil function in patients with chronic granulomatous disease. PLoS One. 2025 Sep 8;20(9):e0331657. doi: 10.1371/journal.pone.0331657. eCollection 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel R. Ambruso, MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Chronic Granulomatous Disease, run by University of Colorado, Denver at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03548818, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jan 2021.