Trial Outcomes & Findings for Azithromycin for Child Survival in Niger: Mortality and Resistance Trial (NCT NCT04224987)
NCT ID: NCT04224987
Last Updated: 2025-05-14
Results Overview
Mortality rate (deaths per 1,000 person-years at risk) among children 1-59 months of age, comparing the azithro 1-59 and placebo arms.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
864493 participants
Primary outcome timeframe
from 6 months up to 2.5 years
Results posted on
2025-05-14
Participant Flow
Please note number of participants in participant flow, baseline characteristics, and analyzed population will differ. A participant is included in the participant flow if they participated in the study in at least one round. However participants are only included in baseline collections if they participated in the first data collection for that community. Participants are only included in analyzed populations if they participated in at least two rounds of data collection.
Unit of analysis: Communities
Participant milestones
| Measure |
Azithro 1-11
Biannual weight- or height-based dose of oral azithromycin suspension to children 1-11 months old and oral placebo or no intervention to children 12-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Azithro 1-59
Biannual age, weight- or height-based dose of oral azithromycin suspension to children 1-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Placebo
Biannual weight- or height-based dose of oral placebo to children 1-59 months old
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
|---|---|---|---|
|
Overall Study
STARTED
|
245227 751
|
334446 1229
|
284820 929
|
|
Overall Study
COMPLETED
|
245227 751
|
334446 1229
|
284820 929
|
|
Overall Study
NOT COMPLETED
|
0 0
|
0 0
|
0 0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Azithromycin for Child Survival in Niger: Mortality and Resistance Trial
Baseline characteristics by cohort
| Measure |
Azithro 1-11
n=431 communities
Biannual weight- or height-based dose of oral azithromycin suspension to children 1-11 months old and oral placebo or no intervention to children 12-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Azithro 1-59
n=463 communities
Biannual age, weight- or height-based dose of oral azithromycin suspension to children 1-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Placebo
n=463 communities
Biannual weight- or height-based dose of oral placebo to children 1-59 months old
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Total
n=1357 communities
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
Age in months categorical · 1-11 months
|
7569 Participants
n=93 Participants
|
8220 Participants
n=4 Participants
|
7981 Participants
n=27 Participants
|
23770 Participants
n=483 Participants
|
|
Age, Customized
Age in months categorical · 12-23 months
|
8452 Participants
n=93 Participants
|
8922 Participants
n=4 Participants
|
8773 Participants
n=27 Participants
|
26147 Participants
n=483 Participants
|
|
Age, Customized
Age in months categorical · 24-59 months
|
25043 Participants
n=93 Participants
|
27270 Participants
n=4 Participants
|
26948 Participants
n=27 Participants
|
79261 Participants
n=483 Participants
|
|
Sex: Female, Male
Female
|
20605 Participants
n=93 Participants
|
22078 Participants
n=4 Participants
|
21582 Participants
n=27 Participants
|
64265 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
20459 Participants
n=93 Participants
|
22334 Participants
n=4 Participants
|
22120 Participants
n=27 Participants
|
64913 Participants
n=483 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Black or African American
|
41064 Participants
n=93 Participants
|
44412 Participants
n=4 Participants
|
43702 Participants
n=27 Participants
|
129178 Participants
n=483 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Region of Enrollment
Niger
|
41064 participants
n=93 Participants
|
44412 participants
n=4 Participants
|
43702 participants
n=27 Participants
|
129178 participants
n=483 Participants
|
|
Children per community
|
95.28 children per community
STANDARD_DEVIATION 71.36 • n=93 Participants
|
95.92 children per community
STANDARD_DEVIATION 67.48 • n=4 Participants
|
94.39 children per community
STANDARD_DEVIATION 61.65 • n=27 Participants
|
95.19 children per community
STANDARD_DEVIATION 66.79 • n=483 Participants
|
PRIMARY outcome
Timeframe: from 6 months up to 2.5 yearsMortality rate (deaths per 1,000 person-years at risk) among children 1-59 months of age, comparing the azithro 1-59 and placebo arms.
Outcome measures
| Measure |
Azithro 1-11
n=104 Participants
Biannual weight- or height-based dose of oral azithromycin suspension to children 1-11 months old and oral placebo or no intervention to children 12-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Azithro 1-59
n=153 Participants
Biannual age, weight- or height-based dose of oral azithromycin suspension to children 1-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Placebo
n=124 Participants
Biannual weight- or height-based dose of oral placebo to children 1-59 months old
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
|---|---|---|---|
|
All-cause Mortality (1-59 Months Old)
|
13.8 deaths per 1,000 person-years
Interval 12.9 to 14.8
|
11.9 deaths per 1,000 person-years
Interval 11.3 to 12.6
|
13.9 deaths per 1,000 person-years
Interval 13.0 to 14.8
|
PRIMARY outcome
Timeframe: from 6 months up to 2.5 yearsMortality rate (deaths per 1,000 person-years at risk) among children 1-11 months of age, comparing the azithro 1-11 and placebo arms.
Outcome measures
| Measure |
Azithro 1-11
n=38 Participants
Biannual weight- or height-based dose of oral azithromycin suspension to children 1-11 months old and oral placebo or no intervention to children 12-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Azithro 1-59
n=38 Participants
Biannual age, weight- or height-based dose of oral azithromycin suspension to children 1-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Placebo
n=32 Participants
Biannual weight- or height-based dose of oral placebo to children 1-59 months old
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
|---|---|---|---|
|
All-cause Mortality (1-11 Months Old)
|
22.3 deaths per 1,000 person years
Interval 10.0 to 24.7
|
18.5 deaths per 1,000 person years
Interval 16.7 to 20.4
|
23.9 deaths per 1,000 person years
Interval 21.6 to 26.2
|
PRIMARY outcome
Timeframe: from 6 months up to 2.5 yearsMortality rate (deaths per 1,000 person-years at risk) among children 12-59 months of age with rates compared between azithro 1-11 and azithro 1-59 communities.
Outcome measures
| Measure |
Azithro 1-11
n=65 Participants
Biannual weight- or height-based dose of oral azithromycin suspension to children 1-11 months old and oral placebo or no intervention to children 12-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Azithro 1-59
n=115 Participants
Biannual age, weight- or height-based dose of oral azithromycin suspension to children 1-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Placebo
n=92 Participants
Biannual weight- or height-based dose of oral placebo to children 1-59 months old
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
|---|---|---|---|
|
All-cause Mortality (12-59 Months Old)
|
12.2 deaths per 1,000 person-years
Interval 11.3 to 13.1
|
10.7 deaths per 1,000 person-years
Interval 10.0 to 11.4
|
12.0 deaths per 1,000 person-years
Interval 11.2 to 13.0
|
PRIMARY outcome
Timeframe: After 4 distributions (approximately 24 months)Prevalence of resistance to macrolides including those determinants known to be found in Streptococcus pneumoniae, Streptococcus pyogenes, and Staphylococcus aureus from nasopharyngeal swabs in children 1-59 months old.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: After 4 distributions (approximately 24 months)Load of genetic determinants of resistance to macrolides including those determinants known to be found in Campylobacter spp, Salmonella spp, Shigella spp, and Escherichia coli from rectal swabs in children 1-59 months old, defined as read number per million base pairs, using DNA-seq (metagenomic deep sequencing)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: After 4 distributions (approximatively 24 month after first distribution)Mortality rate compared by arm in subgroups based on weight in children 1-11 months over 2.5 years
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: After 4 distributions (approximatively 24 month after first distribution)Prevalence of resistance to macrolides from nasopharyngeal swabs and load of genetic determinants of resistance to macrolides from rectal swabs after 4 distributions in: * Children 7-12 years old at 24 months from baseline * Caregivers/guardians of eligible children at 24 months from baseline
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearProgram costs as captured by routine administrative data collection during the substudy and by micro-costing activities, per doses delivered
Outcome measures
Outcome data not reported
Adverse Events
Azithro 1-11
Serious events: 1 serious events
Other events: 0 other events
Deaths: 1666 deaths
Azithro 1-59
Serious events: 1 serious events
Other events: 0 other events
Deaths: 1914 deaths
Placebo
Serious events: 3 serious events
Other events: 0 other events
Deaths: 1923 deaths
Serious adverse events
| Measure |
Azithro 1-11
n=104374 participants at risk
Biannual weight- or height-based dose of oral azithromycin suspension to children 1-11 months old and oral placebo or no intervention to children 12-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Azithro 1-59
n=153626 participants at risk
Biannual age, weight- or height-based dose of oral azithromycin suspension to children 1-59 months old
Azithromycin: Azithromycin will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight or age-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
Placebo
n=124617 participants at risk
Biannual weight- or height-based dose of oral placebo to children 1-59 months old
Placebo: Placebo will be administered as a directly observed dose in oral suspension form for children:
1. Single-dose of 20mg/kg in children (up to the maximum adult dose of 1g)
2. For children 1-11 months of age, weight-based dosing will be used
3. For children 12-59 months of age, height-based dosing will be used via height-stick approximation as currently performed by Niger's trachoma program
|
|---|---|---|---|
|
General disorders
hospitalization for malaria
|
0.00%
0/104374 • 28 days
|
0.00%
0/153626 • 28 days
|
0.00%
2/124617 • Number of events 2 • 28 days
|
|
General disorders
Malaria, fever, diarrhea, vomiting
|
0.00%
1/104374 • Number of events 1 • 28 days
|
0.00%
0/153626 • 28 days
|
0.00%
0/124617 • 28 days
|
|
Gastrointestinal disorders
hospitalization for vomiting, diarrhea, and fever
|
0.00%
0/104374 • 28 days
|
0.00%
0/153626 • 28 days
|
0.00%
1/124617 • Number of events 1 • 28 days
|
|
Gastrointestinal disorders
diarrhea, fever and vomiting
|
0.00%
0/104374 • 28 days
|
0.00%
1/153626 • Number of events 1 • 28 days
|
0.00%
0/124617 • 28 days
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place