Trial Outcomes & Findings for The Anti-inflammatory Effect of Prophylactic Macrolides on Children With Chronic Lung Disease (NCT NCT02544984)

NCT ID: NCT02544984

Last Updated: 2018-06-06

Results Overview

Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time at the end of the winter season, which accounts for the variability in the amount of time participants were observed.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

60 participants

Primary outcome timeframe

5 to 8 months

Results posted on

2018-06-06

Participant Flow

Participant milestones

Participant milestones
Measure
Placebo
The control group will be provided with a placebo medication of similar taste, color, texture, and consistency as the study medication. Patients will receive placebo at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Azithromycin
The azithromycin group will receive azithromycin at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Overall Study
STARTED
30
30
Overall Study
COMPLETED
28
28
Overall Study
NOT COMPLETED
2
2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

The Anti-inflammatory Effect of Prophylactic Macrolides on Children With Chronic Lung Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo
n=30 Participants
The control group will be provided with a placebo medication of similar taste, color, texture, and consistency as the study medication. Patients will receive placebo at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Azithromycin
n=30 Participants
The azithromycin group will receive azithromycin at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Total
n=60 Participants
Total of all reporting groups
Age, Customized
Age · 6 -24 months
19 Participants
n=39 Participants
19 Participants
n=41 Participants
38 Participants
n=35 Participants
Age, Customized
Age · 24 months-72 months
11 Participants
n=39 Participants
11 Participants
n=41 Participants
22 Participants
n=35 Participants
Sex: Female, Male
Female
16 Participants
n=39 Participants
11 Participants
n=41 Participants
27 Participants
n=35 Participants
Sex: Female, Male
Male
14 Participants
n=39 Participants
19 Participants
n=41 Participants
33 Participants
n=35 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
17 Participants
n=39 Participants
14 Participants
n=41 Participants
31 Participants
n=35 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
13 Participants
n=39 Participants
16 Participants
n=41 Participants
29 Participants
n=35 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Asian
1 Participants
n=39 Participants
1 Participants
n=41 Participants
2 Participants
n=35 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Black or African American
9 Participants
n=39 Participants
10 Participants
n=41 Participants
19 Participants
n=35 Participants
Race (NIH/OMB)
White
1 Participants
n=39 Participants
2 Participants
n=41 Participants
3 Participants
n=35 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Unknown or Not Reported
19 Participants
n=39 Participants
17 Participants
n=41 Participants
36 Participants
n=35 Participants
Region of Enrollment
United States
30 Participants
n=39 Participants
30 Participants
n=41 Participants
60 Participants
n=35 Participants
Gestational Age
<28 weeks
22 Participants
n=39 Participants
20 Participants
n=41 Participants
42 Participants
n=35 Participants
Gestational Age
28-31 weeks
6 Participants
n=39 Participants
4 Participants
n=41 Participants
10 Participants
n=35 Participants
Gestational Age
32-36 weeks
2 Participants
n=39 Participants
6 Participants
n=41 Participants
8 Participants
n=35 Participants
Number of Participants who Received Synagis Injection
14 Participants
n=39 Participants
16 Participants
n=41 Participants
30 Participants
n=35 Participants
Number of Patients with Tracheostomy
5 Participants
n=39 Participants
5 Participants
n=41 Participants
10 Participants
n=35 Participants

PRIMARY outcome

Timeframe: 5 to 8 months

Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time at the end of the winter season, which accounts for the variability in the amount of time participants were observed.

Outcome measures

Outcome measures
Measure
Placebo
n=30 Participants
The control group will be provided with a placebo medication of similar taste, color, texture, and consistency as the study medication. Patients will receive placebo at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Azithromycin
n=30 Participants
The azithromycin group will receive azithromycin at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Number of Unscheduled Face-to-face Physician Visits (Clinic Visits, ER Visits, and Hospitalizations)
Unscheduled clinic visits for respiratory symptoms
16 visits
21 visits
Number of Unscheduled Face-to-face Physician Visits (Clinic Visits, ER Visits, and Hospitalizations)
Unscheduled clinic visits for other symptoms
3 visits
5 visits
Number of Unscheduled Face-to-face Physician Visits (Clinic Visits, ER Visits, and Hospitalizations)
Emergency room visits
15 visits
6 visits
Number of Unscheduled Face-to-face Physician Visits (Clinic Visits, ER Visits, and Hospitalizations)
Hospital admissions
6 visits
5 visits

SECONDARY outcome

Timeframe: 5 to 8 months

Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time at the end of the winter season, which accounts for the variability in the amount of time participants were observed.

Outcome measures

Outcome measures
Measure
Placebo
n=30 Participants
The control group will be provided with a placebo medication of similar taste, color, texture, and consistency as the study medication. Patients will receive placebo at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Azithromycin
n=30 Participants
The azithromycin group will receive azithromycin at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Number of Adverse Events
4 adverse events
2 adverse events

SECONDARY outcome

Timeframe: 5 to 8 months

Population: Cost-effective analyses are only justified for interventions that are shown to be effective. Because the intervention at study (the macrolide azithromycin) showed no benefit and led to worse outcomes than those with usual care, an economic evaluation is not warranted and claims data was not collected.

Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 12 months

To determine if prophylactic use of azithromycin will reduce the total number of unscheduled face-to-face physician visit for respiratory related illness in a clinic, urgent care, emergency room or hospital setting during the following 12 months after the intervention.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

To determine if prophylactic use of azithromycin will reduce level of airway resistance as measured by an Airwave Oscillometry System in subjects above 2 years of age, at the time of respiratory illnesses, during the 3-6 months intervention.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 8 months

Using a standardize nasal wash procedure, respiratory samples will be collected at enrollment, at end of study, and during acute respiratory illness requiring face-to-face provider interaction. Children with a tracheostomy will have both a nasal wash sample and a tracheal aspirate sample collected. The respiratory samples will be stabilized with a universal transport media, processed and stored at -80 C for future testing. Testing will be performed for cytokines/chemokines; other biomarkers of disease such as LDH, MPO, and caspase; viral and bacterial respiratory pathogens; and microbiome.

Outcome measures

Outcome data not reported

Adverse Events

Placebo

Serious events: 0 serious events
Other events: 3 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=28 participants at risk
The control group will be provided with a placebo medication of similar taste, color, texture, and consistency as the study medication. Patients will receive placebo at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Azithromycin
n=28 participants at risk
The azithromycin group will receive azithromycin at a dose of 5 mg/kg to be given once a day on Monday, Wednesday, and Friday. The dosage will not be adjusted if a new weight is obtained during the trial period.
Gastrointestinal disorders
Diarrhea
7.1%
2/28 • Number of events 2 • 5 to 8 months - Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.
Parents were contacted biweekly, either in clinic or by phone, to monitor for any adverse reactions, including rash, nausea, vomiting, diarrhea, or abdominal cramping.
0.00%
0/28 • 5 to 8 months - Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.
Parents were contacted biweekly, either in clinic or by phone, to monitor for any adverse reactions, including rash, nausea, vomiting, diarrhea, or abdominal cramping.
Gastrointestinal disorders
Vomiting
7.1%
2/28 • Number of events 2 • 5 to 8 months - Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.
Parents were contacted biweekly, either in clinic or by phone, to monitor for any adverse reactions, including rash, nausea, vomiting, diarrhea, or abdominal cramping.
3.6%
1/28 • Number of events 1 • 5 to 8 months - Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.
Parents were contacted biweekly, either in clinic or by phone, to monitor for any adverse reactions, including rash, nausea, vomiting, diarrhea, or abdominal cramping.
Skin and subcutaneous tissue disorders
Rash
0.00%
0/28 • 5 to 8 months - Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.
Parents were contacted biweekly, either in clinic or by phone, to monitor for any adverse reactions, including rash, nausea, vomiting, diarrhea, or abdominal cramping.
3.6%
1/28 • Number of events 1 • 5 to 8 months - Participants were observed for a minimum of 5 months and a maximum of 8 months, depending on when study enrollment occurred. Patients were recruited for this study on a rolling basis during the start of the winter season and then completed the intervention at the same time during the end of the winter season, which accounts for the variability in the amount of time participants were observed.
Parents were contacted biweekly, either in clinic or by phone, to monitor for any adverse reactions, including rash, nausea, vomiting, diarrhea, or abdominal cramping.

Additional Information

Ricardo A. Mosquera, MD

The University of Texas Health Science Center at Houston

Phone: (713) 500-5650

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place