Idiopathic Pulmonary FibrosisPhase 2GKT137831NCT03865927
GKT137831 in IPF Patients With Idiopathic Pulmonary Fibrosis
A Phase 2 study testing GKT137831 in people with Idiopathic Pulmonary Fibrosis, run by University of Alabama at Birmingham at 4 sites in United States.
Trial summary
A placebo-controlled, multicenter, randomized trial to test GKT137831 in ambulatory patients with idiopathic pulmonary fibrosis. This drug is an inhibitor of nicotinamide adenine dinucleotide phosphate (NADPH) oxidase (NOX) isoforms. Read more about this study
- Who is it for?
- Ages 40 to 85 years
- Conditions the study is for
- Idiopathic Pulmonary Fibrosis
- What is being tested?
- GKT137831
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 58 participants
- Where
- 4 sitesUnited States
- Study dates
- 7 Sep 2020to 30 Nov 2024
- Phase
- Phase 2
- 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 40 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age between 40-85 years old.
- A diagnosis of IPF that fulfills current American Thoracic Society (ATS) Consensus Criteria.
- IPF duration <5 years, based on the date of definitive diagnosis.
- Ability and willingness to give informed consent and adhere to study requirements.
- Ratio of forced expiratory volume in 1 second to forced vital capacity (FEV1/FVC) >70% of predicted values
Who cannot join
- Diagnosis of major comorbidities expected to interfere with study participation
- History of malignancy, excluding basal or squamous cell skin cancer and low-risk prostate cancer, the latter defined as stage T1 or T2a, with prostate specific antigen <10 ng/dl. NOX inhibition is not known to promote cancer, and these criteria are within current guidelines.
- The occurrence of any acute infection requiring systemic antibiotic therapy within 2 weeks prior to Screening (Visit 1).
- Treatment for >14 days within the preceding month with >20 mg. prednisone (or equivalent) or any treatment during the last month with a cellular immunosuppressant (e.g., cyclophosphamide, methotrexate, calcineurin inhibitors, etc.), given increased risks of opportunistic infections.
- Treatment with any investigational agent within 4 weeks of Screening (Visit 1) or 5 half-lives of the investigational medicinal product (whichever is longer).
- Fertile women who do not agree to contraception or abstinence, or who are breast feeding. IPF is a disease of older adults, and male predominant, so this will not be a frequent consideration.
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- Subjects with known hypersensitivity to GKT137831 or its excipients (e.g. capsule "bulking" agents).
- A history of bone marrow disorder including aplastic anemia, or marked anemia defined as hemoglobin < 10.0 g/dL (or 6.2 mmol/L).
- Severe cardiovascular disease, defined as any of the following within the preceding 12 weeks: acute myocardial infarction or unstable angina, a coronary revascularization procedure, congestive heart failure (NYHA Class III or IV), or stroke, including a transient ischemic attack.
- Evidence of cardiac conducting abnormalities, defined as second or third degree atrial-ventricular (AV) block not successfully treated with a pacemaker, or a personal or family history of long QT syndrome (QTc interval >450 msec for males or 470 msec for females).
- End-stage renal disease requiring dialysis.
- Undergoing transplantation evaluation, or listed with the United Network for Organ Sharing (UNOS) as a lung transplantation candidate at the time of enrollment in this trial.
- Liver function tests (transaminases, alkaline phosphatase, direct and total bilirubin) >3x upper limit of normal values
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Michigan Medical Center
Ann Arbor, Michigan, 48109
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Tulane University Medical Center
New Orleans, Louisiana, 70112
Temple University Medical Center
Philadelphia, Pennsylvania, 19140
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Phase II Clinical Trial of GKT137831 in Patients With Idiopathic Pulmonary Fibrosis
A placebo-controlled, multicenter, randomized trial to test GKT137831 in ambulatory patients with idiopathic pulmonary fibrosis. This drug is an inhibitor of nicotinamide adenine dinucleotide phosphate (NADPH) oxidase (NOX) isoforms. The investigators hypothesize the drug will decrease pulmonary injury due to reactive oxygen species (ROS) generated by NOX enzymes, which are believed to play an important role in the development of IPF. Treatment with GKT137831 could result in significant benefit for a lung disease that has, until now, been almost invariably inexorable.
This clinical trial represents the bedside application of a series of NOX translational and basic studies and discoveries, over several years, from the laboratory of Dr. Victor Thannickal.
- Conditions
- Idiopathic Pulmonary Fibrosis
- Being tested
- Placebo Oral Tablet, GKT137831
- Keywords
- Reactive oxygen species (ROS), nicotinamide adenine dinucleotide phosphate (NADPH) oxidase
- Medical subject headings
- Idiopathic Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo Oral TabletOther
see Arm/Group description
GKT137831Drug
GKT137831 is a NOX enzyme inhibitor
| Study group | Role | What they receive |
|---|---|---|
| GKT137831 | Experimental | GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks. Receives: Drug: GKT137831 |
| Placebo Oral Tablet | Placebo comparator | Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks. Receives: Other: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Surrogate Biomarker of Oxidative Stress by Mass Spectroscopy
Changes in concentrations of circulating o,o'-dityrosine, as determined by mass spectroscopy in plasma, in terms of absolute concentrations and percentages of baseline, will be compared within and between treatment arm participants.
Measured: From baseline thru week 24
Secondary outcomes (4)
Collagen Degradation Product by Enzyme Linked Immunoabsorbant Assay
Changes in concentrations of the collagen degradation product, serum C1M measured by enzyme linked immunsorbent assays will be compared between baseline values and those at 24 weeks, and between experimental arm and control participants.
Measured: Baseline to week 24
Pulmonary Function by Spirometry
Forced vital capacity (FVC), measured by spirometer at baseline, will be compared to values at the conclusion of the study and between the two treatment arms.
Measured: Baseline to week 24
Ambulatory Ability by Measuring Walk Distance in Six Minutes
Six-minute walk distance (6MWD) will be compared at baseline and as changes from baseline among experimental arm participants and control subjects
Measured: Baseline to week 24
Evaluation of Safety by Adverse Events
The number of participants who had adverse events will be compared between experimental arm participants and those in the control arm.
Measured: Up to week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2019
- Study start7 Sep 2020
- Primary completion30 Nov 2024
- Study completion30 Nov 2024
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.
- Other trials for Idiopathic Pulmonary FibrosisStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Following screening assessments, IPF patients who meet all inclusion/exclusion criteria will be randomly assigned to receive one of the following treatments in a ratio of 1:1: • Arm A (n=30) - GKT137831 Treatment: GKT137831 will be administered orally, at a dose of 400 mg bid, for a total of 24 weeks. • Arm B (n=30) - Placebo Treatment: Arm B subjects will receive matching placebo for the same duration. Participants will be followed in face-to-face visits with trial personnel every 6 weeks for 24 weeks to assess drug effects and monitor safety during their treatments, and by phone surveillances one month thereafter.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Collaborators
- Temple University, Tulane University, University of Michigan
- Responsible party
- Principal investigator, Steven R. Duncan, MD, University of Alabama at Birmingham
- Investigators
- Steven R Duncan, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03865927
- Sponsor's protocol ID
- IRB-300001635
- Acronym
- GKT137831
- Status last verified
- Apr 2026
- First submitted
- 1 Mar 2019
- Last updated
- 11 May 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Phase II Clinical Trial of GKT137831 in Patients With Idiopathic Pulmonary Fibrosis
- ClinicalTrials.gov ID
- NCT03865927
- Acronym / sponsor ID
- GKT137831 / IRB-300001635
- Conditions
- Idiopathic Pulmonary Fibrosis
- Keywords
- Reactive oxygen species (ROS), nicotinamide adenine dinucleotide phosphate (NADPH) oxidase
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- Temple University, Tulane University, University of Michigan
- Study start
- 7 Sep 2020 (actual)
- Primary completion
- 30 Nov 2024 (actual)
- Study completion
- 30 Nov 2024 (actual)
- First posted
- 7 Mar 2019 (actual)
- Results
- Posted 11 May 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A placebo-controlled, multicenter, randomized trial to test GKT137831 in ambulatory patients with idiopathic pulmonary fibrosis. This drug is an inhibitor of nicotinamide adenine dinucleotide phosphate (NADPH) oxidase (NOX) isoforms. The investigators hypothesize the drug will decrease pulmonary injury due to reactive oxygen species (ROS) generated by NOX enzymes, which are believed to play an important role in the development of IPF. Treatment with GKT137831 could result in significant benefit for a lung disease that has, until now, been almost invariably inexorable.
This clinical trial represents the bedside application of a series of NOX translational and basic studies and discoveries, over several years, from the laboratory of Dr. Victor Thannickal.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Following screening assessments, IPF patients who meet all inclusion/exclusion criteria will be randomly assigned to receive one of the following treatments in a ratio of 1:1: • Arm A (n=30) - GKT137831 Treatment: GKT137831 will be administered orally, at a dose of 400 mg bid, for a total of 24 weeks. • Arm B (n=30) - Placebo Treatment: Arm B subjects will receive matching placebo for the same duration. Participants will be followed in face-to-face visits with trial personnel every 6 weeks for 24 weeks to assess drug effects and monitor safety during their treatments, and by phone surveillances one month thereafter.
| Arm | Type | Receives |
|---|---|---|
| GKT137831 | Experimental | Drug: GKT137831GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks. |
| Placebo Oral Tablet | Placebo comparator | Other: Placebo Oral TabletIdentically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks. |
Who may qualify
- Ages
- 40 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age between 40-85 years old.
- A diagnosis of IPF that fulfills current American Thoracic Society (ATS) Consensus Criteria.
- IPF duration <5 years, based on the date of definitive diagnosis.
- Ability and willingness to give informed consent and adhere to study requirements.
- Ratio of forced expiratory volume in 1 second to forced vital capacity (FEV1/FVC) >70% of predicted values
Key exclusion criteria (13 listed)
- Diagnosis of major comorbidities expected to interfere with study participation
- History of malignancy, excluding basal or squamous cell skin cancer and low-risk prostate cancer, the latter defined as stage T1 or T2a, with prostate specific antigen <10 ng/dl. NOX inhibition is not known to promote cancer, and these criteria are within current guidelines.
- The occurrence of any acute infection requiring systemic antibiotic therapy within 2 weeks prior to Screening (Visit 1).
- Treatment for >14 days within the preceding month with >20 mg. prednisone (or equivalent) or any treatment during the last month with a cellular immunosuppressant (e.g., cyclophosphamide, methotrexate, calcineurin inhibitors, etc.), given increased risks of opportunistic infections.
- Treatment with any investigational agent within 4 weeks of Screening (Visit 1) or 5 half-lives of the investigational medicinal product (whichever is longer).
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 |
|---|---|---|
| Placebo Oral Tabletsee Arm/Group description | Other | Placebo Oral Tablet |
| GKT137831GKT137831 is a NOX enzyme inhibitor | Drug | GKT137831 |
Primary outcome
Surrogate Biomarker of Oxidative Stress by Mass Spectroscopy
Time frame: From baseline thru week 24
Changes in concentrations of circulating o,o'-dityrosine, as determined by mass spectroscopy in plasma, in terms of absolute concentrations and percentages of baseline, will be compared within and between treatment arm participants.
4 secondary outcomes
Collagen Degradation Product by Enzyme Linked Immunoabsorbant Assay
Time frame: Baseline to week 24
Pulmonary Function by Spirometry
Time frame: Baseline to week 24
Ambulatory Ability by Measuring Walk Distance in Six Minutes
Time frame: Baseline to week 24
Evaluation of Safety by Adverse Events
Time frame: Up to week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Steven R Duncan, MD principal investigator
University of Alabama at Birmingham
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GKT137831 in people with Idiopathic Pulmonary Fibrosis, run by University of Alabama at Birmingham at 4 sites 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 5 inclusion and 13 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 11 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 11 May 2026). 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?
Surrogate Biomarker of Oxidative Stress by Mass Spectroscopy (time frame: From baseline thru week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 May 2026.