Trial Outcomes & Findings for Azithromycin Treatment for the Airway Microbiome in Asthma (NCT NCT03736629)

Results Overview

Score ranging from 0 to 40 indicating the degree of asthma symptoms. Lower score indicating worse asthma symptoms.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

Baseline and 8 weeks

Results posted on

2023-08-23

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.
Overall Study STARTED	3	2	12
Overall Study COMPLETED	3	2	12
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Azithromycin Treatment for the Airway Microbiome in Asthma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=3 Participants 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=2 Participants 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls n=12 Participants Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.	Total n=17 Participants Total of all reporting groups
Age, Continuous	50.5 years n=5 Participants	47.4 years n=7 Participants	35.5 years n=5 Participants	39.5 years n=4 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	2 Participants n=7 Participants	9 Participants n=5 Participants	14 Participants n=4 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	3 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants	8 Participants n=4 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Region of Enrollment United States	3 participants n=5 Participants	2 participants n=7 Participants	12 participants n=5 Participants	17 participants n=4 Participants

PRIMARY outcome

Timeframe: Baseline and 8 weeks

Population: Outcome variable is not applicable to non-asthmatic controls, as they were not assessed for asthma symptoms.

Score ranging from 0 to 40 indicating the degree of asthma symptoms. Lower score indicating worse asthma symptoms.

Outcome measures

Outcome measures
Measure	Placebo n=3 Participants 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=2 Participants 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.
Asthma Control Test (ACT) Score Change From Baseline Over 8 Weeks	0.67 score on a scale Standard Error 0.67	0.00 score on a scale Standard Error 2.00	—

SECONDARY outcome

Timeframe: 8 weeks

Volume of air exhaled in 1 second on a forced expiration, expressed as percent of predicted, indicating the degree of airflow obstruction in asthma

Outcome measures

Outcome measures
Measure	Placebo n=3 Participants 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=2 Participants 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls n=12 Participants Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.
Forced Expiratory Volume (FEV1) Change From Baseline Over 8 Weeks	1.67 percentage of predicted Standard Error 3.76	-4.00 percentage of predicted Standard Error 11.00	-2.92 percentage of predicted Standard Error 1.37

SECONDARY outcome

Timeframe: Baseline and 8 weeks

Population: Some patients had missing data.

Percentage of 'eosinophils', an inflammatory cell seen in the airways of people with asthma, indicating the degree of inflammation

Outcome measures

Outcome measures
Measure	Placebo n=1 Participants 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=1 Participants 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls n=5 Participants Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.
Sputum Eosinophils Change From Baseline Over 8 Weeks	0.00 percentage of eosinophils Interval 0.0 to 0.0	0.00 percentage of eosinophils Interval 0.0 to 0.0	0.06 percentage of eosinophils Interval -0.33 to 0.64

SECONDARY outcome

Timeframe: Baseline and 8 weeks

Population: Some patients had missing data.

Percentage of 'neutrophils', an inflammatory cell seen in the airways of people with asthma, indicating the degree of inflammation

Outcome measures

Outcome measures
Measure	Placebo n=1 Participants 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=1 Participants 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls n=5 Participants Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.
Sputum Neutrophils Change From Baseline Over 8 Weeks	-0.00 percentage of neutrophils Interval 0.0 to 0.0	1.59 percentage of neutrophils Interval 1.59 to 1.59	-10.03 percentage of neutrophils Interval -46.88 to 8.03

SECONDARY outcome

Timeframe: 8 weeks

Population: Outcome measure is not applicable to non-asthmatic controls, as they were not assessed for asthma severity.

The outcome is derived from a weighted scoring system called CompEx that describes asthma symptoms recorded on a daily basis. Final result is a dichotomous outcome indicating whether the patient had a "diary event" based on changes in peak expiratory flow, reliever use, and asthma symptoms or a serious exacerbation (deterioration of asthma leading to oral corticosteroid use, emergency room admission, or hospital admission).

Outcome measures

Outcome measures
Measure	Placebo n=2 Participants 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=2 Participants 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth	Non-asthmatic Controls Non-asthmatic controls to assess variability of microbiome composition and diversity over time in a normal population. No intervention given.
Number of Participants With Diary Event or Serious Asthma Exacerbation Over 8 Weeks	1 Participants	0 Participants	—

SECONDARY outcome

Timeframe: 8 weeks

Population: Insufficient sample size for meaningful results in intervention arms due to low accrual. Microbiome assays were not performed.

A measure of the diversity of the bacteria in the sputum or oral sample; that is, how many different types of bacteria are present. This value decreases in severe asthma

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 8 weeks

Population: Insufficient sample size for meaningful results in intervention arms. Microbiome assays were not performed.

A second measure of the diversity of the bacteria in the sputum or oral sample; that is, how many different types of bacteria are present. This value decreases in severe asthma

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 8 weeks

Population: Insufficient sample size for meaningful results in intervention arms. Microbiome assays were not performed.

A measure of how many of the top types of bacteria are present in the sputum or oral sample. Value changes with asthma

Outcome measures

Outcome data not reported

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Azithromycin

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Placebo n=3 participants at risk 8 weeks of placebo capsule once daily by mouth Placebo: Placebo	Azithromycin n=2 participants at risk 8 weeks of Azithromycin (250 mg) capsule once daily by mouth Azithromycin: 8 weeks of Azithromycin (250 mg) once daily by mouth
Respiratory, thoracic and mediastinal disorders Nasal congestion	33.3% 1/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	0.00% 0/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Respiratory, thoracic and mediastinal disorders Wheezing	33.3% 1/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	0.00% 0/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Gastrointestinal disorders Diarrhea	0.00% 0/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	50.0% 1/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Injury, poisoning and procedural complications Bruising	0.00% 0/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	50.0% 1/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	50.0% 1/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Gastrointestinal disorders Nausea	0.00% 0/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	50.0% 1/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Gastrointestinal disorders Vomiting	0.00% 0/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	50.0% 1/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.
Nervous system disorders Headache	0.00% 0/3 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.	50.0% 1/2 • 14 weeks Note: Adverse events were not collected in the Non-asthmatic controls.

Additional Information

Theodore Karrison (Research Professor)

University of Chicago

Phone: 773-702-9326

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place