Trial Outcomes & Findings for GKT137831 in IPF Patients With Idiopathic Pulmonary Fibrosis (NCT NCT03865927)
NCT ID: NCT03865927
Last Updated: 2026-05-11
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
58 participants
Primary outcome timeframe
From baseline thru week 24
Results posted on
2026-05-11
Participant Flow
Participant milestones
| Measure |
GKT137831
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
Placebo Oral Tablet
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
|---|---|---|
|
Overall Study
STARTED
|
30
|
28
|
|
Overall Study
COMPLETED
|
22
|
22
|
|
Overall Study
NOT COMPLETED
|
8
|
6
|
Reasons for withdrawal
| Measure |
GKT137831
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
Placebo Oral Tablet
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
4
|
4
|
|
Overall Study
Unacceptable side effects
|
4
|
1
|
|
Overall Study
Subject did not want to take study medicine
|
0
|
1
|
Baseline Characteristics
GKT137831 in IPF Patients With Idiopathic Pulmonary Fibrosis
Baseline characteristics by cohort
| Measure |
GKT137831
n=30 Participants
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
Placebo Oral Tablet
n=28 Participants
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=44 Participants
|
6 Participants
n=10 Participants
|
12 Participants
n=30 Participants
|
|
Age, Categorical
>=65 years
|
24 Participants
n=44 Participants
|
22 Participants
n=10 Participants
|
46 Participants
n=30 Participants
|
|
Age, Continuous
|
71 years
STANDARD_DEVIATION 6.5 • n=44 Participants
|
70 years
STANDARD_DEVIATION 6.3 • n=10 Participants
|
70 years
STANDARD_DEVIATION 6.4 • n=30 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=44 Participants
|
7 Participants
n=10 Participants
|
14 Participants
n=30 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=44 Participants
|
21 Participants
n=10 Participants
|
44 Participants
n=30 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
30 Participants
n=44 Participants
|
28 Participants
n=10 Participants
|
58 Participants
n=30 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=44 Participants
|
1 Participants
n=10 Participants
|
2 Participants
n=30 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=44 Participants
|
1 Participants
n=10 Participants
|
2 Participants
n=30 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=44 Participants
|
26 Participants
n=10 Participants
|
54 Participants
n=30 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=44 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=30 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=44 Participants
|
28 participants
n=10 Participants
|
58 participants
n=30 Participants
|
PRIMARY outcome
Timeframe: From baseline thru week 24Changes 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.
Outcome measures
| Measure |
Placebo Oral Tablet
n=22 plasma
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
GKT137831
n=21 plasma
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
|---|---|---|
|
Surrogate Biomarker of Oxidative Stress by Mass Spectroscopy
|
13.3 micromole/M tyrosine
Standard Deviation 39.3
|
-79.9 micromole/M tyrosine
Standard Deviation 255.5
|
SECONDARY outcome
Timeframe: Baseline to week 24Population: There are no remaining funds to perform these assays and these results will not be available.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to week 24Population: Drop out of subjects (discontinued) by participant or PI
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.
Outcome measures
| Measure |
Placebo Oral Tablet
n=27 Participants
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
GKT137831
n=29 Participants
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
|---|---|---|
|
Pulmonary Function by Spirometry
Baseline
|
2.7 Liters
Standard Deviation 0.8
|
2.8 Liters
Standard Deviation 0.9
|
|
Pulmonary Function by Spirometry
24 week
|
2.6 Liters
Standard Deviation 0.9
|
2.8 Liters
Standard Deviation 1.0
|
SECONDARY outcome
Timeframe: Baseline to week 24Population: Drop out due to discontinuations.
Six-minute walk distance (6MWD) will be compared at baseline and as changes from baseline among experimental arm participants and control subjects
Outcome measures
| Measure |
Placebo Oral Tablet
n=25 Participants
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
GKT137831
n=29 Participants
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
|---|---|---|
|
Ambulatory Ability by Measuring Walk Distance in Six Minutes
Baseline
|
1191 feet
Standard Deviation 425
|
1190 feet
Standard Deviation 440
|
|
Ambulatory Ability by Measuring Walk Distance in Six Minutes
24 week
|
1249 feet
Standard Deviation 355
|
1168 feet
Standard Deviation 440
|
SECONDARY outcome
Timeframe: Up to week 24The number of participants who had adverse events will be compared between experimental arm participants and those in the control arm.
Outcome measures
| Measure |
Placebo Oral Tablet
n=28 Participants
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
GKT137831
n=30 Participants
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
|---|---|---|
|
Evaluation of Safety by Adverse Events
|
17 participants
|
19 participants
|
Adverse Events
GKT137831
Serious events: 7 serious events
Other events: 19 other events
Deaths: 0 deaths
Placebo Oral Tablet
Serious events: 3 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GKT137831
n=30 participants at risk
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
Placebo Oral Tablet
n=28 participants at risk
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
|---|---|---|
|
Nervous system disorders
Neurologic Complications (syncope, lighteadedness)
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
0.00%
0/28 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Dysfunction
|
16.7%
5/30 • Number of events 5 • 24 weeks
|
3.6%
1/28 • Number of events 1 • 24 weeks
|
|
Blood and lymphatic system disorders
Prolongation of Prothrombin Time
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
0.00%
0/28 • 24 weeks
|
|
Surgical and medical procedures
Hospitalization for elective surgery
|
0.00%
0/30 • 24 weeks
|
7.1%
2/28 • Number of events 2 • 24 weeks
|
Other adverse events
| Measure |
GKT137831
n=30 participants at risk
GKT137831 will be administered orally, at a dose of 400 mg twice daily, for a total of 24 weeks.
GKT137831: GKT137831 is a NOX enzyme inhibitor
|
Placebo Oral Tablet
n=28 participants at risk
Identically-appearing placebo oral tablets will be administered orally, twice daily, for a total of 24 weeks.
Placebo Oral Tablet: see Arm/Group description
|
|---|---|---|
|
Nervous system disorders
Agitation
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
7.1%
2/28 • Number of events 2 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
23.3%
7/30 • Number of events 10 • 24 weeks
|
25.0%
7/28 • Number of events 9 • 24 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
30.0%
9/30 • Number of events 14 • 24 weeks
|
28.6%
8/28 • Number of events 10 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
40.0%
12/30 • Number of events 18 • 24 weeks
|
21.4%
6/28 • Number of events 6 • 24 weeks
|
|
General disorders
fatigue
|
13.3%
4/30 • Number of events 4 • 24 weeks
|
10.7%
3/28 • Number of events 3 • 24 weeks
|
|
Nervous system disorders
Headache
|
13.3%
4/30 • Number of events 6 • 24 weeks
|
7.1%
2/28 • Number of events 3 • 24 weeks
|
|
Metabolism and nutrition disorders
Hypokalemia
|
6.7%
2/30 • Number of events 4 • 24 weeks
|
7.1%
2/28 • Number of events 2 • 24 weeks
|
|
Hepatobiliary disorders
Liver enzyme elevation
|
10.0%
3/30 • Number of events 5 • 24 weeks
|
0.00%
0/28 • 24 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
0.00%
0/28 • 24 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
7.1%
2/28 • Number of events 2 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Tract Infection
|
13.3%
4/30 • Number of events 4 • 24 weeks
|
3.6%
1/28 • Number of events 1 • 24 weeks
|
|
Gastrointestinal disorders
Vomiting
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
7.1%
2/28 • Number of events 2 • 24 weeks
|
|
Gastrointestinal disorders
Anorexia
|
10.0%
3/30 • Number of events 3 • 24 weeks
|
0.00%
0/28 • 24 weeks
|
|
Nervous system disorders
Dizziness
|
16.7%
5/30 • Number of events 5 • 24 weeks
|
3.6%
1/28 • Number of events 1 • 24 weeks
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/30 • 24 weeks
|
7.1%
2/28 • Number of events 3 • 24 weeks
|
|
Gastrointestinal disorders
Nausea
|
20.0%
6/30 • Number of events 6 • 24 weeks
|
21.4%
6/28 • Number of events 6 • 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
6.7%
2/30 • Number of events 3 • 24 weeks
|
7.1%
2/28 • Number of events 2 • 24 weeks
|
|
Gastrointestinal disorders
Abdominal Pain
|
3.3%
1/30 • Number of events 1 • 24 weeks
|
17.9%
5/28 • Number of events 5 • 24 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place