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

Phase 3

Completed

• Conditions: Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
• Interventions: Drug: TOBI; Drug: Placebo

• Registration Number: NCT01082367
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-03-05
• Study First Submitted QC: 2010-03-05
• Study First Posted: 2010-03-08
• Study Start: 2010-04
• Primary Completion: 2015-06
• Study Completion: 2015-06
• Results First Submitted: 2015-12-22
• Results First Submitted QC: 2016-03-24
• Results First Posted: 2016-04-27
• Status Verified: 2016-06
• Last Update Submitted: 2016-06-06
• Last Update Posted: 2016-07-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 50

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
TOBI (tobramycin inhaled solution)/Placebo	Placebo	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).
Placebo/TOBI	Placebo	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).
TOBI (tobramycin inhaled solution)/Placebo	TOBI	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).
Placebo/TOBI	TOBI	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).

Primary Outcome Measures

Name	Time	Method
Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle	Day 29	Sputum/throat swab cultures were assessed.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇨🇭 Zürich, Switzerland