Trial Outcomes & Findings for Study of Epetraborole in Patients With Treatment-refractory MAC Lung Disease (NCT NCT05327803)
NCT ID: NCT05327803
Last Updated: 2026-06-10
Results Overview
Detection of within-patient changes in symptoms reported in a novel Patient-Reported Outcome (PRO) instrument at Month 6 (Clinical Response measured as improvement in at least one symptom and no worsening in any others from baseline)
TERMINATED
PHASE2/PHASE3
177 participants
Baseline to Month 6
2026-06-10
Participant Flow
This study included 2 phases of enrollment. 80 participants in Phase 2 were randomized 1:1 to Epetraborole or Placebo arms, both including Optimized Background Regimen (OBR) 97 participants in Phase 3 were randomized 2:1 to Epetraborole or Placebo arms, both including Optimized Background Regimen (OBR)
Phase 2 : 39 participants were randomized to Epetraborole + OBR arm and 41 participants were randomized to placebo + OBR arm. Phase 3 : 66 participants were randomized to Epetraborole + OBR arm and 31 participants were randomized to placebo + OBR arm.
Participant milestones
| Measure |
Epetraborole + OBR
epetraborole + Optimized Background Regimen
|
Placebo + OBR
Placebo + Optimized Background Regimen
|
|---|---|---|
|
Phase 2
STARTED
|
39
|
41
|
|
Phase 2
COMPLETED
|
35
|
34
|
|
Phase 2
NOT COMPLETED
|
4
|
7
|
|
Phase 3
STARTED
|
66
|
31
|
|
Phase 3
COMPLETED
|
52
|
22
|
|
Phase 3
NOT COMPLETED
|
14
|
9
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Epetraborole in Patients With Treatment-refractory MAC Lung Disease
Baseline characteristics by cohort
| Measure |
Phase 2 : Epetraborole + OBR
n=39 Participants
Phase 2 : Epetraborole + Optimized Background Regimen
|
Phase 2 : Placebo + OBR
n=41 Participants
Phase 2 : Placebo + Optimized Background Regimen
|
Phase 3 : Epetraborole + OBR
n=66 Participants
Phase 3 : Epetraborole + Optimized Background Regimen
|
Phase 3 : Placebo + OBR
n=31 Participants
Phase 2 : Placebo + Optimized Background Regimen
|
Total
n=177 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
>=65 years
|
21 Participants
n=9 Participants
|
23 Participants
n=27 Participants
|
45 Participants
n=267 Participants
|
18 Participants
n=265 Participants
|
107 Participants
n=568 Participants
|
|
Sex: Female, Male
Female
|
27 Participants
n=9 Participants
|
30 Participants
n=27 Participants
|
49 Participants
n=267 Participants
|
22 Participants
n=265 Participants
|
128 Participants
n=568 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
0 Participants
n=568 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
18 Participants
n=9 Participants
|
18 Participants
n=27 Participants
|
21 Participants
n=267 Participants
|
13 Participants
n=265 Participants
|
70 Participants
n=568 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=9 Participants
|
11 Participants
n=27 Participants
|
17 Participants
n=267 Participants
|
9 Participants
n=265 Participants
|
49 Participants
n=568 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
0 Participants
n=568 Participants
|
|
Race (NIH/OMB)
Asian
|
26 Participants
n=9 Participants
|
25 Participants
n=27 Participants
|
39 Participants
n=267 Participants
|
23 Participants
n=265 Participants
|
113 Participants
n=568 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
0 Participants
n=568 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=9 Participants
|
1 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
3 Participants
n=568 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=9 Participants
|
15 Participants
n=27 Participants
|
23 Participants
n=267 Participants
|
8 Participants
n=265 Participants
|
59 Participants
n=568 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
2 Participants
n=568 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
0 Participants
n=568 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
1 Participants
n=265 Participants
|
4 Participants
n=568 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
38 Participants
n=9 Participants
|
41 Participants
n=27 Participants
|
64 Participants
n=267 Participants
|
30 Participants
n=265 Participants
|
173 Participants
n=568 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
0 Participants
n=265 Participants
|
0 Participants
n=568 Participants
|
|
Region of Enrollment
United States
|
11 Participants
n=9 Participants
|
15 Participants
n=27 Participants
|
26 Participants
n=267 Participants
|
7 Participants
n=265 Participants
|
59 Participants
n=568 Participants
|
|
Region of Enrollment
Australia
|
2 Participants
n=9 Participants
|
1 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
1 Participants
n=265 Participants
|
6 Participants
n=568 Participants
|
|
Region of Enrollment
Japan
|
18 Participants
n=9 Participants
|
14 Participants
n=27 Participants
|
31 Participants
n=267 Participants
|
17 Participants
n=265 Participants
|
80 Participants
n=568 Participants
|
|
Region of Enrollment
South Korea
|
8 Participants
n=9 Participants
|
11 Participants
n=27 Participants
|
7 Participants
n=267 Participants
|
6 Participants
n=265 Participants
|
32 Participants
n=568 Participants
|
PRIMARY outcome
Timeframe: Baseline to Month 16Population: Safety Population
Percentage of Participants reporting treatment emergent adverse events
Outcome measures
| Measure |
Placebo + OBR
n=41 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=39 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Adverse Event Profile of 500 mg Once Daily Dose of Epetraborole
|
35 Participants
|
37 Participants
|
PRIMARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat
Detection of within-patient changes in symptoms reported in a novel Patient-Reported Outcome (PRO) instrument at Month 6 (Clinical Response measured as improvement in at least one symptom and no worsening in any others from baseline)
Outcome measures
| Measure |
Placebo + OBR
n=40 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=38 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Percentage of Participants Achieving Clinical Response
|
10 Participants
|
15 Participants
|
PRIMARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat
Detection of within-patient changes in symptoms reported in a novel Patient-Reported Outcome (PRO) instrument at Month 6 (Clinical Response measured as improvement in at least one symptom and no worsening in any others from baseline)
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=65 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Percentage of Participants Achieving Clinical Response
|
10 Participants
|
21 Participants
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat
Sputum conversion will be assessed using culture conversion based on 3 consecutive monthly negative sputum cultures for MAC by Month 6.
Outcome measures
| Measure |
Placebo + OBR
n=40 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=38 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Percentage of Participants Achieving Culture Conversion
|
4 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat (Limited to patients with evaluable baseline colony count categories)
Microbiological improvement will be assessed at Month 3 and Month 6 using decrease in MAC colony counts of ≥1 category.
Outcome measures
| Measure |
Placebo + OBR
n=38 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=34 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Percentage of Participants Achieving Microbiological Improvement
|
16 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: Measured at Baseline and months 1, 2, 3, 4, 5, and 6; Change from Baseline to Month 6 ReportedPopulation: Microbiological Intent-To-Treat (Limited to patients with evaluable Baseline and Month 6 results)
Mean change from baseline in Quality of Life - Bronchiectasis (QOL-B) respiratory domain score measured monthly through Month 6 (Change from Baseline to Month 6 Reported). QOL-B respiratory domain score is measured on a scale from 0 to 100 (with higher scores indicating better health-related quality of life).
Outcome measures
| Measure |
Placebo + OBR
n=35 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=34 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Change From Baseline in QOL-B Respiratory Domain PRO
|
0.30 Change from baseline score
Standard Error 2.569
|
7.20 Change from baseline score
Standard Error 2.447
|
SECONDARY outcome
Timeframe: Measured at Baseline and months 1, 2, 3, 4, 5, and 6; Change from Baseline to Month 6 ReportedPopulation: Microbiological Intent-To-Treat (Limited to patients with evaluable Baseline and Month 6 results)
Mean change from baseline in NTM Symptoms Module PRO measured monthly through Month 6 (Change from Baseline to Month 6 Reported). NTM Symptoms Module score is measured on a scale from 0 to 100 (with higher scores indicating better function).
Outcome measures
| Measure |
Placebo + OBR
n=35 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=34 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Change From Baseline in NTM Symptoms Module PRO
|
0.83 Change from baseline score
Standard Error 2.746
|
6.20 Change from baseline score
Standard Error 2.599
|
SECONDARY outcome
Timeframe: Measured at Baseline and months 1, 2, 3, 4, 5, and 6; Change from Baseline to Month 6 ReportedPopulation: Microbiological Intent-To-Treat (Limited to patients with evaluable Baseline and Month 6 results)
Mean change from baseline in Saint George's Respiratory Questionnaire for COPD Patients (SGRQ-C) total score measured monthly through Month 6 (Change from Baseline to Month 6 Reported). SGRQ-C total score is measured on a scale from 0 to 100 (with lower scores representing better quality of life).
Outcome measures
| Measure |
Placebo + OBR
n=34 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=34 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Change From Baseline in SGRQ-C PRO
|
0.33 Change from baseline score
Standard Error 2.464
|
-2.27 Change from baseline score
Standard Error 2.317
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat
Concordance between clinical response in a novel Patient-Reported Outcome (PRO) instrument and microbiological response will be assessed at Months 3 and 6. Microbiological response will be assessed using both sputum culture conversion and microbiological improvement.
Outcome measures
| Measure |
Placebo + OBR
n=40 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=38 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
PRO responder and culture conversion
|
1 Participants
|
2 Participants
|
|
Phase 2: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
PRO responder and no culture conversion
|
9 Participants
|
13 Participants
|
|
Phase 2: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
PRO failure and culture conversion
|
3 Participants
|
3 Participants
|
|
Phase 2: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
PRO failure and no culture conversion
|
27 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: Day 1 (2-3 hours post-dose) and Day 29 (within 1 hour pre-dose and 2-3 hours post-dose); Day 29 Result ReportedPopulation: PK Population
Cmax is the maximum plasma concentration of epetraborole estimated by population PK model.
Outcome measures
| Measure |
Placebo + OBR
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=39 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Maximum Plasma Concentration (Cmax) of Epetraborole
|
—
|
2.73 ug/mL
Geometric Coefficient of Variation 18.9
|
SECONDARY outcome
Timeframe: Day 1 (2-3 hours post-dose) and Day 29 (within 1 hour pre-dose and 2-3 hours post-dose); Day 1 Result ReportedPopulation: PK Population
AUC(0-24) is defined as area under the plasma concentration-time curve of epetraborole from timepoint 0 hours until 24 hours post dose estimated by population PK model.
Outcome measures
| Measure |
Placebo + OBR
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=39 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Area Under the Plasma Concentration-Time Curve From Time Point 0 Hours Until 24 Hours [AUC(0-24)] Post Dose
|
—
|
14.8 ug*hr/mL
Geometric Coefficient of Variation 19.8
|
SECONDARY outcome
Timeframe: Day 1 (2-3 hours post-dose) and Day 29 (within 1 hour pre-dose and 2-3 hours post-dose); Day 29 Result ReportedPopulation: PK Population
Vd is the apparent volume of distribution of epetraborole estimated by population PK model.
Outcome measures
| Measure |
Placebo + OBR
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=39 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 2: Volume of Distribution (Vd) of Epetraborole
|
—
|
174 L
Geometric Coefficient of Variation 16.2
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat
Sputum conversion will be assessed using culture conversion based on 3 consecutive monthly negative sputum cultures for MAC by Month 6.
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=65 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Percentage of Participants Achieving Culture Conversion
|
3 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat (Limited to patients with evaluable baseline colony count categories)
Microbiological improvement will be assessed at Month 3 and Month 6 using decrease in MAC colony counts of ≥1 category.
Outcome measures
| Measure |
Placebo + OBR
n=27 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=61 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Percentage of Participants Achieving Microbiological Improvement
|
7 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: Measured at Baseline and months 1, 2, 3, 4, 5, and 6; Change from Baseline to Month 6 ReportedPopulation: Microbiological Intent-To-Treat (Limited to patients with evaluable Baseline and Month 6 results)
Mean change from baseline in Quality of Life - Bronchiectasis (QOL-B) respiratory domain score measured monthly through Month 6 (Change from Baseline to Month 6 Reported). QOL-B respiratory domain score is measured on a scale from 0 to 100 (with higher scores indicating better health-related quality of life).
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=51 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Change From Baseline in QOL-B Respiratory Domain PRO
|
4.16 Change from baseline score
Standard Error 2.748
|
5.14 Change from baseline score
Standard Error 2.252
|
SECONDARY outcome
Timeframe: Measured at Baseline and months 1, 2, 3, 4, 5, and 6; Change from Baseline to Month 6 ReportedPopulation: Microbiological Intent-To-Treat (Limited to patients with evaluable Baseline and Month 6 results)
Mean change from baseline in NTM Symptoms Module PRO measured monthly through Month 6 (Change from Baseline to Month 6 Reported). NTM Symptoms Module score is measured on a scale from 0 to 100 (with higher scores indicating better function).
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=51 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Change From Baseline in NTM Symptoms Module PRO
|
1.66 Change from baseline score
Standard Error 3.032
|
3.55 Change from baseline score
Standard Error 2.477
|
SECONDARY outcome
Timeframe: Measured at Baseline and months 1, 2, 3, 4, 5, and 6; Change from Baseline to Month 6 ReportedPopulation: Microbiological Intent-To-Treat (Limited to patients with evaluable Baseline and Month 6 results)
Mean change from baseline in Saint George's Respiratory Questionnaire for COPD Patients (SGRQ-C) total score measured monthly through Month 6 (Change from Baseline to Month 6 Reported). SGRQ-C total score is measured on a scale from 0 to 100 (with lower scores representing better quality of life).
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=51 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Change From Baseline in SGRQ-C PRO
|
-3.36 Change from baseline total score
Standard Error 2.308
|
-2.49 Change from baseline total score
Standard Error 1.873
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Microbiological Intent-To-Treat
Concordance between clinical response in a novel Patient-Reported Outcome (PRO) instrument and microbiological response will be assessed at Months 3 and 6. Microbiological response will be assessed using both sputum culture conversion and microbiological improvement
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=65 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
Clinical Response and Culture Conversion
|
1 Participants
|
1 Participants
|
|
Phase 3: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
Clinical Response and No Culture Conversion
|
9 Participants
|
20 Participants
|
|
Phase 3: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
Clinical Failure and Culture Conversion
|
2 Participants
|
4 Participants
|
|
Phase 3: Concordance Analysis of PRO-based Clinical Response and Microbiological Response
Clinical Failure and No Culture Conversion
|
19 Participants
|
40 Participants
|
SECONDARY outcome
Timeframe: Baseline to Month 16Population: Safety Population
Percentage of Participants reporting treatment emergent adverse events.
Outcome measures
| Measure |
Placebo + OBR
n=31 Participants
Placebo + Optimized Background Regimen
|
Epetraborole + OBR
n=66 Participants
epetraborole + Optimized Background Regimen
|
|---|---|---|
|
Phase 3: Adverse Event Profile of 500 mg Once Daily Dose of Epetraborole
|
23 Participants
|
59 Participants
|
Adverse Events
Epetraborole + OBR
Placebo + OBR
Serious adverse events
| Measure |
Epetraborole + OBR
n=105 participants at risk
epetraborole + Optimized Background Regimen
|
Placebo + OBR
n=72 participants at risk
Placebo + Optimized Background Regimen
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
1.9%
2/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Vascular disorders
Aortic stenosis
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Cardiac disorders
Atrial fibrillation
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Infections and infestations
Atypical mycobacterial infection
|
2.9%
3/105 • Baseline to Month 16
|
4.2%
3/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Product Issues
Device breakage
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.95%
1/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Injury, poisoning and procedural complications
Hand fracture
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Nervous system disorders
Headache
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Infections and infestations
Infective exacerbation of bronchiectasis
|
1.9%
2/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Injury, poisoning and procedural complications
Ligament rupture
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Infections and infestations
Pneumonia
|
0.95%
1/105 • Baseline to Month 16
|
4.2%
3/72 • Baseline to Month 16
|
|
Infections and infestations
Pneumonia aspiration
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.95%
1/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Injury, poisoning and procedural complications
Respiratory fume inhalation disorder
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Nervous system disorders
Seizure
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Cardiac disorders
Sinus tachycardia
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Nervous system disorders
Stupor
|
0.95%
1/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Nervous system disorders
Syncope
|
0.00%
0/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
Other adverse events
| Measure |
Epetraborole + OBR
n=105 participants at risk
epetraborole + Optimized Background Regimen
|
Placebo + OBR
n=72 participants at risk
Placebo + Optimized Background Regimen
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
28.6%
30/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Investigations
Haemoglobin decreased
|
11.4%
12/105 • Baseline to Month 16
|
0.00%
0/72 • Baseline to Month 16
|
|
Nervous system disorders
Dizziness
|
10.5%
11/105 • Baseline to Month 16
|
1.4%
1/72 • Baseline to Month 16
|
|
Nervous system disorders
Headache
|
9.5%
10/105 • Baseline to Month 16
|
15.3%
11/72 • Baseline to Month 16
|
|
Gastrointestinal disorders
Nausea
|
9.5%
10/105 • Baseline to Month 16
|
4.2%
3/72 • Baseline to Month 16
|
|
Infections and infestations
COVID -19
|
8.6%
9/105 • Baseline to Month 16
|
9.7%
7/72 • Baseline to Month 16
|
|
Infections and infestations
Nasopharyngitis
|
6.7%
7/105 • Baseline to Month 16
|
11.1%
8/72 • Baseline to Month 16
|
|
Gastrointestinal disorders
Diarrhoea
|
5.7%
6/105 • Baseline to Month 16
|
4.2%
3/72 • Baseline to Month 16
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60