Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 Years

Study Overview
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Brief Summary
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This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.

Official Title
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A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years

Conditions
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Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients

Intervention / Treatment
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* Drug: TOBI
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of cystic fibrosis Early lower respiratory tract infection with P. aeruginosa, Exclusion Criteria: Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics. Administration of loop diuretics within 7 days prior to study drug administration. Other protocol-defined inclusion/exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 3 Months
Maximum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: TOBI (tobramycin inhaled solution)/Placebo<br>Participants randomized to TOBI received the investigational treatment for 28 days twice daily (bi)d in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the open label (OL) phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received placebo for 28 days bid (second treatment cycle). | Drug: TOBI<br>* TOBI (tobramycin inhaled solution)<br>Drug: Placebo<br>* Placebo<br>|
| Placebo Comparator: Placebo/TOBI<br>Participants randomized to placebo group received 0.9 % saline (NaCl) for 28 days bid in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the OL phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received TOBI for 28 days bid (second treatment cycle). | Drug: TOBI<br>* TOBI (tobramycin inhaled solution)<br>Drug: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle | Sputum/throat swab cultures were assessed. | Day 29 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Free From P. Aeruginosa 28 Days After Termination of the Second Treatment Cycle | Sputum/throat swab cultures were assessed. | Day 91 | 
| Percentage of Participants P Aeruginosa-free at Termination of the Double Blind Period | Sputum/throat swab cultures were assessed. | Day 91 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Tobramycin Inhalation solution, Cystic fibrosis, Lung disease, anti-bacterial agents