Trial Outcomes & Findings for A Study of Standard Drugs for Mycobacterium Avium Complex (NCT NCT04287049)
NCT ID: NCT04287049
Last Updated: 2026-07-15
Results Overview
The early bactericidal activity of azithromycin for Mycobacterium avium will be determined as the change in Mycobacterium avium colony count (log10 colony forming unit (CFU) per mL) in sputum between baseline and day 15.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Day 0 and Day 15
Results posted on
2026-07-15
Participant Flow
Participants were recruited from the Johns Hopkins Center for Nontuberculous Mycobacteria and Bronchiectasis at Bayview Medical Center, Johns Hopkins Health System. Written informed consent was required for study participation.
Of 10 consented participants, 10 met inclusion criteria and were assigned treatment.
Participant milestones
| Measure |
14 Day Azithromycin Monotherapy
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
14 Day Azithromycin Monotherapy Completed
|
10
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
14 Day Azithromycin Monotherapy
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Overall Study
Death
|
1
|
Baseline Characteristics
A Study of Standard Drugs for Mycobacterium Avium Complex
Baseline characteristics by cohort
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Age, Continuous
|
71.5 years
STANDARD_DEVIATION 5.0 • n=20 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=20 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
|
Region of Enrollment
United States
|
10 Participants
n=20 Participants
|
|
Baseline Colony Forming Unit
|
2.66 log10 CFU/mL
STANDARD_DEVIATION 1.48 • n=20 Participants
|
|
Baseline Time to Positivity
|
129.3 hours
n=20 Participants
|
PRIMARY outcome
Timeframe: Day 0 and Day 15The early bactericidal activity of azithromycin for Mycobacterium avium will be determined as the change in Mycobacterium avium colony count (log10 colony forming unit (CFU) per mL) in sputum between baseline and day 15.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Mycobacterium Avium Colony Count in Sputum
|
-0.425 log10 CFU/mL
Standard Deviation 1.39
|
PRIMARY outcome
Timeframe: Day 0 and Day 15The time (hours) to positivity in MGIT of Mycobacterium avium will be compared between baseline and day 15.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Time to Positivity of Mycobacterium Avium Growth in the Mycobacterial Growth Indicator Tube (MGIT)
|
101 hours
Standard Deviation 283
|
SECONDARY outcome
Timeframe: Day 0 and Day 8The bactericidal activity of multidrug therapy for Mycobacterium avium will be determined as the change in Mycobacterium avium colony count (log10 CFU per mL) in sputum between baseline and day 8.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Mycobacterium Avium Colony Count in Sputum
|
0.261 log10 CFU/mL
Standard Deviation 1.02
|
SECONDARY outcome
Timeframe: Day 8 to Day 15The bactericidal activity of multidrug therapy for Mycobacterium avium will be determined as the change in Mycobacterium avium colony count (log10 CFU per mL) in sputum between day 8 and day 15.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Mycobacterium Avium Colony Count in Sputum
|
-0.686 log10 CFU/mL
Standard Deviation 1.39
|
SECONDARY outcome
Timeframe: Day 0 and 2 MonthsThe bactericidal activity of multidrug therapy for Mycobacterium avium will be determined as the change in Mycobacterium avium colony count (log10 CFU per mL) in sputum between baseline and 2 months.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=9 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Mycobacterium Avium Colony Count in Sputum
|
-1.56 log10 CFU/mL
Standard Deviation 0.955
|
SECONDARY outcome
Timeframe: Day 0 and Day 8The time (hours) to positivity in MGIT of Mycobacterium avium will be compared between baseline and day 8.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Time to Positivity of Mycobacterium Avium Growth in MGIT
|
72.6 hours
Standard Deviation 288
|
SECONDARY outcome
Timeframe: Day 7 and Day 14The time (hours) to positivity in MGIT of Mycobacterium avium will be compared between day 7 and day 14.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Time to Positivity of Mycobacterium Avium Growth in MGIT
|
28.1 hours
Standard Deviation 60.0
|
SECONDARY outcome
Timeframe: Baseline and 2 MonthsThe time (hours) to positivity in MGIT of Mycobacterium avium will be compared between baseline and 2 months.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=9 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Mean Change in Time to Positivity of Mycobacterium Avium Growth in MGIT
|
468 hours
Standard Deviation 412
|
SECONDARY outcome
Timeframe: Pre-dose, 2, 4 and 6 hours post-dose on day 15 (range: 11-19 days)Area-under-the-curve (ug/mL\*hr) will be predicted from plasma azithromycin levels using population pharmacokinetic modeling methods.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Estimation of Plasma Azithromycin Area-under-the-curve (AUC) Following Oral Dosing Azithromycin
|
2.62 μg/mL*h
Standard Deviation 0.964
|
SECONDARY outcome
Timeframe: 2 and 6 hours post-dose on day 29 (range: 25-31 days)Area-under-the-curve (ug/mL\*hr) will be predicted from plasma azithromycin levels using population pharmacokinetic modeling methods.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Estimation of Plasma Azithromycin Area-under-the-curve (AUC) Following Oral Dosing Azithromycin
|
1.50 μg/mL*h
Standard Deviation 0.413
|
SECONDARY outcome
Timeframe: Pre-dose, 2, 4 and 6 hours post-dose on day 15 (range: 11-19 days)Peak concentration (Cmax) will be predicted from plasma drug concentration in ug/mL following oral dosing of azithromycin.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Estimation of Maximum Plasma Concentration (Cmax) of Azithromycin
|
0.197 μg/mL
Standard Deviation 0.0772
|
SECONDARY outcome
Timeframe: 2 and 6 hours post-dose on day 29 (range: 25-31 days)Peak concentration (Cmax) will be predicted from plasma drug concentration in ug/mL following oral dosing of azithromycin.
Outcome measures
| Measure |
14 Day Azithromycin Monotherapy
n=10 Participants
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
Estimation of Maximum Plasma Concentration (Cmax) of Azithromycin
|
0.154 μg/mL
Standard Deviation 0.0552
|
Adverse Events
14 Day Azithromycin Monotherapy
Serious events: 0 serious events
Other events: 1 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
14 Day Azithromycin Monotherapy
n=10 participants at risk
For the first 14 days of therapy, participants will receive Azithromycin 250mg by mouth (PO) daily as monotherapy. Beyond day 14, all participants will receive guideline-based standard multi-drug therapy for Mycobacterium avium lung disease, as dictated by the physicians treating the participants.
Azithromycin: Azithromycin 250 mg PO daily
|
|---|---|
|
General disorders
Chills
|
10.0%
1/10 • Number of events 2 • Over the study period, at each visit, or if it is reported to the study team through hospitalization, for a total maximum duration up to 77 days.
|
|
General disorders
Fever
|
10.0%
1/10 • Number of events 2 • Over the study period, at each visit, or if it is reported to the study team through hospitalization, for a total maximum duration up to 77 days.
|
|
General disorders
Calcium, low
|
10.0%
1/10 • Number of events 1 • Over the study period, at each visit, or if it is reported to the study team through hospitalization, for a total maximum duration up to 77 days.
|
|
General disorders
Glucose, non-fasting, high
|
10.0%
1/10 • Number of events 1 • Over the study period, at each visit, or if it is reported to the study team through hospitalization, for a total maximum duration up to 77 days.
|
Additional Information
Elisa Ignatius, MD, MSc
Johns Hopkins University School of Medicine
Phone: +1-410-955-1288
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place