Phase 2
N=58
GKT137831 in IPF Patients With Idiopathic Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT03865927 ↗Enrolled (actual)
58
Serious AEs
17.2%
Results posted
May 2026
Primary outcome: Primary: Surrogate Biomarker of Oxidative Stress by Mass Spectroscopy — -79.9; 13.3 micromole/M tyrosine
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Placebo Oral Tablet (Other); GKT137831 (Drug)
- Age
- Adult, Older Adult · 40+ yrs
- Sex
- All
- Sponsor
- University of Alabama at Birmingham
- Primary completion
- Nov 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Surrogate Biomarker of Oxidative Stress by Mass Spectroscopy |
-79.9; 13.3 | — |
| SECONDARY Collagen Degradation Product by Enzyme Linked Immunoabsorbant Assay |
— | — |
| SECONDARY Pulmonary Function by Spirometry |
2.8; 2.7; 2.8; 2.6 | — |
| SECONDARY Ambulatory Ability by Measuring Walk Distance in Six Minutes |
1190; 1191; 1168; 1249 | — |
| SECONDARY Evaluation of Safety by Adverse Events |
19; 17 | — |
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. 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.
Eligibility Criteria
Inclusion Criteria
- Age between 40-85 years old.
- A diagnosis of IPF that fulfills current American Thoracic Society (ATS) Consensus Criteria.
- IPF duration 70% of predicted values
Exclusion Criteria
- 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 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.
- 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 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
Data sourced from ClinicalTrials.gov (NCT03865927). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.