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
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 50
- Locations
- 1
- Primary Endpoint
- Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle
Study Overview
Brief Summary
This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Crossover
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 3 Months to 6 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
TOBI (tobramycin inhaled solution)/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).
Intervention: TOBI (Drug)
TOBI (tobramycin inhaled solution)/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).
Intervention: Placebo (Drug)
Placebo/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).
Intervention: TOBI (Drug)
Placebo/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).
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle
Time Frame: Day 29
Sputum/throat swab cultures were assessed.
Secondary Outcomes
- Percentage of Participants Free From P. Aeruginosa 28 Days After Termination of the Second Treatment Cycle(Day 91)
- Percentage of Participants P Aeruginosa-free at Termination of the Double Blind Period(Day 91)