Trial Outcomes & Findings for Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa (NCT NCT00431964)
NCT ID: NCT00431964
Last Updated: 2015-08-19
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
263 participants
Primary outcome timeframe
change from baseline to day 168
Results posted on
2015-08-19
Participant Flow
Participant milestones
| Measure |
Active
azithromycin 250 mg tablets
azithromycin 250 mg tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Placebo
placebo tablets (matched to active drug in appearance)
placebo tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
|---|---|---|
|
Overall Study
STARTED
|
131
|
132
|
|
Overall Study
Randomized/Received Drug
|
131
|
129
|
|
Overall Study
Did Not Receive Drug
|
0
|
3
|
|
Overall Study
Withdrew From Study
|
6
|
5
|
|
Overall Study
COMPLETED
|
125
|
124
|
|
Overall Study
NOT COMPLETED
|
6
|
8
|
Reasons for withdrawal
| Measure |
Active
azithromycin 250 mg tablets
azithromycin 250 mg tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Placebo
placebo tablets (matched to active drug in appearance)
placebo tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
1
|
2
|
|
Overall Study
Physician Decision
|
2
|
2
|
|
Overall Study
Withdrawal by Subject
|
2
|
1
|
|
Overall Study
Parental Consent Withdrawn
|
0
|
2
|
|
Overall Study
Due to Unstable Health
|
0
|
1
|
Baseline Characteristics
Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa
Baseline characteristics by cohort
| Measure |
Active
n=131 Participants
azithromycin 250 mg tablets
azithromycin 250 mg tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Placebo
n=129 Participants
placebo tablets (matched to active drug in appearance)
placebo tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Total
n=260 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
131 Participants
n=5 Participants
|
129 Participants
n=7 Participants
|
260 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
10.7 years
STANDARD_DEVIATION 3.25 • n=5 Participants
|
10.6 years
STANDARD_DEVIATION 3.10 • n=7 Participants
|
10.7 years
STANDARD_DEVIATION 3.17 • n=5 Participants
|
|
Sex: Female, Male
Female
|
54 Participants
n=5 Participants
|
59 Participants
n=7 Participants
|
113 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
77 Participants
n=5 Participants
|
70 Participants
n=7 Participants
|
147 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: change from baseline to day 168Population: Modified intent to treat (randomized and at least one dose of study drug).
Outcome measures
| Measure |
Active
n=131 Participants
azithromycin 250 mg tablets
azithromycin 250 mg tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Placebo
n=129 Participants
placebo tablets (matched to active drug in appearance)
placebo tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
|---|---|---|
|
Change in FEV1 From Baseline to End of Treatment at Day 168
|
0.08 liters
Standard Deviation 0.25
|
0.06 liters
Standard Deviation 0.28
|
Adverse Events
Active
Serious events: 12 serious events
Other events: 121 other events
Deaths: 0 deaths
Placebo
Serious events: 14 serious events
Other events: 128 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Active
n=131 participants at risk
azithromycin 250 mg tablets
azithromycin 250 mg tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Placebo
n=129 participants at risk
placebo tablets (matched to active drug in appearance)
placebo tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Gastrointestinal disorders
Constipation
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Gastrointestinal disorders
Vomiting
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
General disorders
Asthenia
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
General disorders
Exercise tolerance decreased
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
General disorders
Implant site pain
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
General disorders
Pyrexia
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Infections and infestations
Burkholderia cepacia infection
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Infections and infestations
Pseudomonas infection
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Infections and infestations
Sinusitis
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Investigations
Pulmonary function test decreased
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
1.6%
2/129 • Number of events 2 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial secretion retention
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
3.1%
4/129 • Number of events 4 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
1.6%
2/129 • Number of events 2 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Surgical and medical procedures
Gastrostomy tube insertion
|
0.76%
1/131 • Number of events 1 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Surgical and medical procedures
Oesophagogastric fundoplasty
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
|
Surgical and medical procedures
Sinus operation
|
0.00%
0/131 • Randomization through Day 168
|
0.78%
1/129 • Number of events 1 • Randomization through Day 168
|
Other adverse events
| Measure |
Active
n=131 participants at risk
azithromycin 250 mg tablets
azithromycin 250 mg tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
Placebo
n=129 participants at risk
placebo tablets (matched to active drug in appearance)
placebo tablets: \*One (1) tablet three times weekly for patients who weigh 40-79 lbs
\*Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
|
|---|---|---|
|
Ear and labyrinth disorders
Ear pain
|
0.00%
0/131 • Randomization through Day 168
|
7.0%
9/129 • Number of events 11 • Randomization through Day 168
|
|
Gastrointestinal disorders
Abdominal pain
|
8.4%
11/131 • Number of events 11 • Randomization through Day 168
|
8.5%
11/129 • Number of events 11 • Randomization through Day 168
|
|
Gastrointestinal disorders
Abdominal pain upper
|
12.2%
16/131 • Number of events 23 • Randomization through Day 168
|
15.5%
20/129 • Number of events 37 • Randomization through Day 168
|
|
Gastrointestinal disorders
Constipation
|
6.1%
8/131 • Number of events 10 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/131 • Randomization through Day 168
|
8.5%
11/129 • Number of events 12 • Randomization through Day 168
|
|
Gastrointestinal disorders
Nausea
|
8.4%
11/131 • Number of events 15 • Randomization through Day 168
|
9.3%
12/129 • Number of events 15 • Randomization through Day 168
|
|
Gastrointestinal disorders
Vomiting
|
16.0%
21/131 • Number of events 29 • Randomization through Day 168
|
24.0%
31/129 • Number of events 40 • Randomization through Day 168
|
|
General disorders
Fatigue
|
6.9%
9/131 • Number of events 9 • Randomization through Day 168
|
10.1%
13/129 • Number of events 14 • Randomization through Day 168
|
|
General disorders
Pain
|
0.00%
0/131 • Randomization through Day 168
|
5.4%
7/129 • Number of events 7 • Randomization through Day 168
|
|
General disorders
Pyrexia
|
22.1%
29/131 • Number of events 31 • Randomization through Day 168
|
31.8%
41/129 • Number of events 54 • Randomization through Day 168
|
|
Infections and infestations
Pseudomonas infection
|
6.9%
9/131 • Number of events 9 • Randomization through Day 168
|
11.6%
15/129 • Number of events 15 • Randomization through Day 168
|
|
Investigations
Pulmonary function test decreased
|
6.1%
8/131 • Number of events 9 • Randomization through Day 168
|
11.6%
15/129 • Number of events 16 • Randomization through Day 168
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/131 • Randomization through Day 168
|
7.0%
9/129 • Number of events 9 • Randomization through Day 168
|
|
Nervous system disorders
Headache
|
22.9%
30/131 • Number of events 53 • Randomization through Day 168
|
31.0%
40/129 • Number of events 84 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
48.1%
63/131 • Number of events 118 • Randomization through Day 168
|
70.5%
91/129 • Number of events 172 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.9%
9/131 • Number of events 12 • Randomization through Day 168
|
7.0%
9/129 • Number of events 10 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
34.4%
45/131 • Number of events 63 • Randomization through Day 168
|
32.6%
42/129 • Number of events 64 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
22.1%
29/131 • Number of events 40 • Randomization through Day 168
|
33.3%
43/129 • Number of events 64 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
7.6%
10/131 • Number of events 11 • Randomization through Day 168
|
18.6%
24/129 • Number of events 25 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
6.9%
9/131 • Number of events 10 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
19.1%
25/131 • Number of events 37 • Randomization through Day 168
|
27.9%
36/129 • Number of events 62 • Randomization through Day 168
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
7.6%
10/131 • Number of events 10 • Randomization through Day 168
|
7.0%
9/129 • Number of events 10 • Randomization through Day 168
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.9%
9/131 • Number of events 12 • Randomization through Day 168
|
0.00%
0/129 • Randomization through Day 168
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place