IFN-Gamma TherapyPhase 4NCT01147042

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

A Phase 4 study testing IFN-gamma in people with IFN-Gamma Therapy and CGD Gene Mutation, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

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. Read more about this study

Who is it for?: Any age
Conditions the study is for: IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg
What is being tested?: IFN-gamma
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 2 participants
Where: 1 siteUnited States
Study dates: 18 May 2010to 14 Nov 2014
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Subjects may be enrolled if they are:
Already are enrolled on an existing CGD protocol at the Clinical Center (and will remain enrolled on their existing protocol);
Are included in one of the study cohorts listed below;
Male or female;
Able to comply with self-administration of a subcutaneous injection; and
Willing to have their blood samples stored for the duration of this study and for future research.

Show all 12Show fewer

Study Groups/Cohorts:
X-linked CGD Nonsense/Frameshift/RNA Processing/Deletion Mutations Cohort: Subjects in this cohort must have X-linked CGD resulting from a documented nonsense, frameshift, RNA processing, or deletion gene mutation. Subjects with other gene defects or for whom the specific genetic defect has not been determined are not eligible for inclusion in this cohort.
X-linked CGD Missense Mutation with Low Baseline Superoxide Production (less than or equal to 2.5 nmol/10(6) cells/hr) Cohort: Subjects in this cohort must have X-linked CGD resulting from a documented missense gene and superoxide production by cytochrome c reduction assay at baseline of less than or equal to 2.5 nmol/10(6) cells/hr. Subjects with other gene defects or for whom the specific genetic defect has not been determined are not eligible for inclusion in this cohort.
X-linked CGD Missense Mutation with Higher Baseline Superoxide Production (greater than 2.5 nmol/10(6) cells/hr) Cohort: Subjects in this cohort must have X-linked CGD resulting from a documented missense gene and superoxide production by cytochrome c reduction assay at baseline of greater than 2.5 nmol/10(6) cells/hr. Subjects with other gene defects or for whom the specific genetic defect has not been determined are not eligible for inclusion in this cohort.
Autosomal Recessive p47phox CGD Cohort: Subjects in this cohort must have autosomal recessive CGD resulting from a documented p47phox gene mutation. Subjects with other gene defects or for whom the specific genetic defect has not been determined are not eligible for inclusion in this cohort.
Autosomal Recessive p67phox CGD Cohort: Subjects in this cohort must have autosomal recessive CGD resulting from a documented p67phox gene mutation. Subjects with other gene defects or for whom the specific genetic defect has not been determined are not eligible for inclusion in this cohort.

Who cannot join

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;

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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.

Trial locations and contacts

1 site in 1 country.

United States1 site

National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Assessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease

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.

Read the detailed description

Chronic Granulomatous Disease (CGD) is caused by mutations of 1 of the 4 proteins comprising the NADPH oxidase that result in decreased or absent production of superoxide by phagocytes, and predisposes CGD subjects to life-threatening infection. Intensive management with antibiotics and antifungal agents has dramatically increased the life expectancy of subjects with CGD. Interferon-gamma (IFN gamma), which increases superoxide production by neutrophils and enhances their antimicrobial activity, is an FDA approved therapy for CGD and is now the standard of care. However, there is substantial variability in the biochemical and clinical response to IFN gamma treatment. Recently, the specific mutations of the genes responsible for causing CGD in most of the subjects followed at the NIH have been characterized. Because of this, it is now known that the severity of the disease is correlated not only with inheritance pattern, but also with the specific underlying mutation. It is not known, however, if the biochemical response to IFN gamma therapy correlates with the specific mutation as well.

Since treatment with IFN gamma is expensive, requires frequent injections, and in some subjects results in systemic side effects, it would be useful to determine whether the biochemical response and systemic side effects correlate with the underlying mutation GCD.

We hypothesize that subjects with X-linked CGD due to nonsense/frameshift/RNA processing/deletion mutations of the gp91phox component of the NADPH oxidase will generate a smaller biochemical response to IFN gamma therapy compared to subjects with missense gp91phox mutations or the autosomal recessive form of CGD that results from mutations of the p47phox or p67phox components.

The primary objective of this study is to assess the predictability of IFN gamma responsiveness in CGD based on mutational analysis. compare the change in function of the NADPH oxidase during treatment with an escalating dose of IFN gamma in subjects with CGD resulting from missense or nonsense/frameshift/RNA processing/deletion gp91phox mutations or mutations of p47phox or p67phox. The secondary objectives are to assess changes in superoxide production, expression of NADPH oxidase components, neutrophil bactericidal capacity for Staphyloccus aureus, cytokines, cell surface markers, antibodies, lymphocyte subsets, constitutional symptoms , and gene expression in leukocytes from subjects with missense gp91phox mutations, nonsense/frameshift/RNA processing/deletion gp91phox mutations, p47phox mutations, and p67phox mutations after treatment with IFN gamma to assess changes in the expression of NADPH oxidase components, cytokines, cell surface markers, antibody production, production of various lymphocyte subsets, constitutional symptoms and gene expression in leukocytes from these subjects following treatment with IFN gamma. This knowledge will assist physicians in determining which subjects are likely to respond to full dose and alternative dose therapy with IFN gamma and provide information about biochemical responses of to these regimens in subjects with specific CDG gene mutations enabling them to better counsel and manage subjects with CGD.

Conditions: IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease, Immunodeficiency Disease
Being tested: IFN-gamma
Keywords: NADPH Oxidase-Nicotinamide Adenine Dinucleotide Phosphate, Chronic Granulomatous Disease, Gene Mutation, Superoxide by Phagocytes, Interferon-Gamma
Medical subject headings: Granulomatous Disease, Chronic, Immunologic Deficiency Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IFN-gammaDrug
Also called Interferon-gamma
Following a washout period subjects have a subcutaneous injection of 50 mcg per meter squared once per week, Monday, for 4 weeks, twice per week, Monday and Thursday, for 4 weeks, then thrice per week, Monday, Wednesday, Friday, for 4 weeks. Total treatment period is 12 weeks.

Study groups in NCT01147042
Study group	Role	What they receive
gp91 CGD with relatively high baseline superoxide	Active comparator	Patients with X-linked Chronic Granulomatous Disease (CGD) with a missense gp91phox mutation and relatively high baseline superoxide production. IFN-gamma was the administered intervention. Receives: Drug: IFN-gamma
Autosomal Recessive CGD with p47	Active comparator	Patients with Autosomal Recessive Chronic Granulomatous Disease (CGD) with p47 phox mutation. IFN-gamma was the administered intervention. Receives: Drug: IFN-gamma

What is being measured

The primary outcome is the main question the study is designed to answer.

Basal and PMA-stimulated O2 Production Detected by Ferricytochrome c Reduction in Neutrophils
Measured: 21 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 May 201014 Nov 2014

First posted22 Jun 2010 (estimated)
Study start18 May 2010
Primary completion14 Nov 2014
Study completion14 Nov 2014

Why it stopped: Early termination due to only 2 subjects completing trial.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (4)Papers the sponsor has linked to this study.
Other trials for IFN-Gamma TherapyStudies of the same condition, open and under way.
More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The 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: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: Dose frequency increased from once per week to three times per week, the recommended dose frequency.
Masking: None (open label)
Enrolment: 2 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01147042
Sponsor's protocol ID: 100123
Other IDs: 10-I-0123
Status last verified: Aug 2017
First submitted: 17 Jun 2010
Last updated: 20 Sep 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Winkelstein JA, Marino MC, Johnston RB Jr, Boyle J, Curnutte J, Gallin JI, Malech HL, Holland SM, Ochs H, Quie P, Buckley RH, Foster CB, Chanock SJ, Dickler H. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore). 2000 May;79(3):155-69. doi: 10.1097/00005792-200005000-00003.
PubMed 10844935
Nathan CF, Murray HW, Wiebe ME, Rubin BY. Identification of interferon-gamma as the lymphokine that activates human macrophage oxidative metabolism and antimicrobial activity. J Exp Med. 1983 Sep 1;158(3):670-89. doi: 10.1084/jem.158.3.670.
PubMed 6411853
Berton G, Zeni L, Cassatella MA, Rossi F. Gamma interferon is able to enhance the oxidative metabolism of human neutrophils. Biochem Biophys Res Commun. 1986 Aug 14;138(3):1276-82. doi: 10.1016/s0006-291x(86)80421-1.
PubMed 3092821
International Chronic Granulomatous Disease Cooperative Study Group. A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease. N Engl J Med. 1991 Feb 21;324(8):509-16. doi: 10.1056/NEJM199102213240801.
PubMed 1846940

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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RecruitingPhase 3
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All IFN-Gamma Therapy trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Assessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease
ClinicalTrials.gov ID: NCT01147042
Acronym / sponsor ID: 100123
Conditions: IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease, Immunodeficiency Disease
Keywords: NADPH Oxidase-Nicotinamide Adenine Dinucleotide Phosphate, Chronic Granulomatous Disease, Gene Mutation, Superoxide by Phagocytes, Interferon-Gamma
Phase: Phase 4
Study type: Interventional
Recruitment status: TerminatedReason stopped: Early termination due to only 2 subjects completing trial.
Enrolment: 2 participants (actual)
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
Study start: 18 May 2010
Primary completion: 14 Nov 2014 (actual)
Study completion: 14 Nov 2014 (actual)
First posted: 22 Jun 2010 (estimated)
Results: Posted 20 Sep 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2
Model description: Dose frequency increased from once per week to three times per week, the recommended dose frequency.

Arm	Type	Receives
gp91 CGD with relatively high baseline superoxide	Active comparator	Drug: IFN-gammaPatients with X-linked Chronic Granulomatous Disease (CGD) with a missense gp91phox mutation and relatively high baseline superoxide production. IFN-gamma was the administered intervention.
Autosomal Recessive CGD with p47	Active comparator	Drug: IFN-gammaPatients with Autosomal Recessive Chronic Granulomatous Disease (CGD) with p47 phox mutation. IFN-gamma was the administered intervention.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (12 listed)

Already are enrolled on an existing CGD protocol at the Clinical Center (and will remain enrolled on their existing protocol);
Are included in one of the study cohorts listed below;
Male or female;
Able to comply with self-administration of a subcutaneous injection; and
Willing to have their blood samples stored for the duration of this study and for future research.

Key exclusion criteria (14 listed)

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;

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
IFN-gammaAlso: Interferon-gammaFollowing a washout period subjects have a subcutaneous injection of 50 mcg per meter squared once per week, Monday, for 4 weeks, twice per week, Monday and Thursday, for 4 weeks, then thrice per week, Monday, Wednesday, Friday, for 4 weeks. Total treatment period is 12 weeks.	Drug	Autosomal Recessive CGD with p47, gp91 CGD with relatively high baseline superoxide

Primary outcome

Basal and PMA-stimulated O2 Production Detected by Ferricytochrome c Reduction in Neutrophils
Time frame: 21 weeks

Results and safety

Results posted to the registry on 20 Sep 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
gp91 CGD With HIGH Baseline Superoxide	2	2
Autosomal Recessive CGD With p47	0	0

Primary outcome: Basal and PMA-stimulated O2 Production Detected by Ferricytochrome c Reduction in Neutrophils

Time frame: 21 weeks

Population analysed: Data were not collected.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
gp91 CGD With Relatively High Baseline Superoxide	0 of 2 (0%)	0 of 2 (0%)	0 of 2

Limitations and caveats (from the sponsor): Early termination due to only 2 subjects completing trial.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 10844935Winkelstein JA, Marino MC, Johnston RB Jr, Boyle J, Curnutte J, Gallin JI, Malech HL, Holland SM, Ochs H, Quie P, Buckley RH, Foster CB, Chanock SJ, Dickler H. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore). 2000 May;79(3):155-69. doi: 10.1097/00005792-200005000-00003.
Background: PubMed 6411853Nathan CF, Murray HW, Wiebe ME, Rubin BY. Identification of interferon-gamma as the lymphokine that activates human macrophage oxidative metabolism and antimicrobial activity. J Exp Med. 1983 Sep 1;158(3):670-89. doi: 10.1084/jem.158.3.670.
Background: PubMed 3092821Berton G, Zeni L, Cassatella MA, Rossi F. Gamma interferon is able to enhance the oxidative metabolism of human neutrophils. Biochem Biophys Res Commun. 1986 Aug 14;138(3):1276-82. doi: 10.1016/s0006-291x(86)80421-1.
Background: PubMed 1846940International Chronic Granulomatous Disease Cooperative Study Group. A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease. N Engl J Med. 1991 Feb 21;324(8):509-16. doi: 10.1056/NEJM199102213240801.

Related trials for IFN-Gamma Therapy

RecruitingPhase 2
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Active, not recruitingPhase 2
Interferon-gamma as Adjunctive Therapy in Chronic Pulmonary Aspergillosis: a Randomised Feasibility Study
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RecruitingPhase 2/3
Effect of Interferon Gamma as a Treatment for Post-aggressive Immunosuppression in Intensive Care Units, a Randomized Bayesian Double-blind Controlled Trial Versus Placebo
Post-aggressive Immunosuppression, Adult Patients Admitted to Intensive Care With Initial Severity (SOFA of the First 24 Hours Post-admission ≥ 6
RecruitingPhase 3
Human Recombinant Interferon Gamma in the Treatment of Ventilator-acquired Pneumonia in ICU Patients
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RecruitingPhase 2
Pulsed Dose Chemotherapy Plus Pembrolizumab in Recurrent/Metastatic HNSCC
Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)

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.

John I Gallin, M.D. principal investigator

National Institute of Allergy and Infectious Diseases (NIAID)

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing IFN-gamma in people with IFN-Gamma Therapy and CGD Gene Mutation, run by National Institute of Allergy and Infectious Diseases (NIAID) 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 12 inclusion and 14 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: stopped early, as of the sponsor's last update on 20 Sep 2017. 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.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Sep 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Basal and PMA-stimulated O2 Production Detected by Ferricytochrome c Reduction in Neutrophils (time frame: 21 weeks). 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 NCT01147042, which also lists 4 linked publications.

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 20 Sep 2017.