Page Title
Clinical Trial Finder
Anti-Infective Completed with Results
OPTIMIZE: Phase 3 study of tobramycin solution for inhalation with and without azithromycin (OPTIMIZE-IP-12)
This study evaluated the effect of treatment with tobramycin inhalation solution (TIS) with and without azithromycin in people with CF who had their first isolation of Pseudomonas aeruginosa (Pa) from a respiratory culture (or their first culture after being negative for Pa for at least 2 years).
Participants were randomized to receive oral azithromycin (10 mg/kg up to a maximum of 500 mg per dose) or placebo three times a week for 18 months. All participants received TIS therapy (TOBI® 300mg BID delivered by inhalation using the PARI LC PLUS™ nebulizer) during the first treatment quarter, consisting of a 28-day course of TIS therapy with a second 28-day course for those participants who remained Pa positive at 21 days. Subsequently, participants received TIS therapy at the beginning of each quarter if their quarterly cultures were Pa positive.
Eligibility
-
Age:
6 Months to 18 Years -
Mutation(s):
No Mutation Requirement -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Study Results
-
What We Learned:
This study found that participants receiving azithromycin in addition to TIS therapy experienced a significant reduction in risk of exacerbation, as measured by time to first exacerbation requiring antibiotics and increased weight when compared to placebo. Azithromycin was well-tolerated.
-
Primary Findings:
Effectiveness:
This study was conducted between June 2014 and March 2017. A total of 221 participants were randomized and enrolled (azithromycin: N=110 and placebo: N=111). Following recommendation by the Data Safety Monitoring Board (DSMB) overseeing the trial, enrollment was stopped early by the National Heart, Lung, and Blood Institute (NHLBI) because the trial data showed early efficacy based on primary endpoint parameters determined prior to the study starting. The trial was amended to continue as an open-label study for the remainder of the planned study follow-up.
The primary efficacy endpoint was time to pulmonary exacerbation requiring antibiotics. The study met its primary endpoint. Risk of pulmonary exacerbation was reduced by 44% in participants receiving oral azithromycin on top of their TIS therapy when compared with placebo (p=0.046).
The study also found that weight increased by 1.27 kg in the azithromycin group compared to the placebo group (p=0.046). No significant difference was observed in time to Pa reoccurrence between the two groups.
Safety:
Azithromycin was well-tolerated and no significant differences in serious adverse events were observed between the two groups.
-
Citation:
Am J Respir Crit Care Med ;DOI 10.1164/rccm.201802-0215OC [Epub ahead of print]
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
18 months -
Number of Study Visits:
8
Additional Information
-
Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Ramsey, Bonnie -
Study Drugs:
Eligibility
-
Age:
6 Months to 18 Years -
Mutation(s):
No Mutation Requirement -
FEV1% Predicted:
No FEV1 Limit -
Pseudomonas Status:
Positive Respiratory Cultures Required
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Related Topics
Sign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Check the Drug Development Pipeline
We’re attacking CF from every angle. Learn about the status of CF drugs in development.
Learn More