Clinical trial NCT00982930: Open Label Extension to Bridging Study CTBM100C2303
Official title: A Phase III Open-Label Extension Study to Assess the Safety and Efficacy of Tobramycin Inhalation Powder After Manufacturing Process Modifications (TIPnew) in Cystic Fibrosis (CF) Subjects Who Completed Participation in Study CTBM100C2303.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pseudomonas Aeruginosa
Cystic Fibrosis
Keywords: Tobramycin Inhalation Powder
Cystic fibrosis
Lung diseases
Anti-Bacterial Agents
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2009-09-23
Enrollment: 55
Sex: ALL
Minimum age (years): 6
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who had completed their study participation in CTBM100C2303 (all visits) and who were proven infected with Pseudomonas aeruginosa (P. aeruginosa) at enrollment into CTBM100C2303.


Eligibility criteria: Inclusion Criteria:

* Completed all visits in study CTBM100C2303, and visit 4 took place not more than 5 days before enrollment into this study.
* Confirmed diagnosis of cystic fibrosis participants with P. aeruginosa infection.
* Forced Expiratory Volume in One Second (FEV1) at screening (study CTBM100C2303) must be between 25% and 80% of normal predicted values.

Exclusion Criteria:

* Any use of inhaled anti-pseudomonal antibiotics between the termination of the core trial CTMB100C2303 and the enrollment into this study.
* Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.

Primary outcomes:
measure: Percentage of Participants With Adverse Events (AEs)
timeFrame: From first administration of study drug to study completion (up to approximately 25 weeks)
measure: Percentage of Participants With Serious Adverse Events (SAEs)
timeFrame: From time of consent to 4 weeks after study completion (up to approximately 29 weeks)
measure: Percentage of Death Cases
timeFrame: From time of consent to 4 weeks after study completion (up to approximately 29 weeks)
measure: Percentage of Participants With Adverse Events and Serious Adverse Events Leading to Permanent Study Discontinuation
timeFrame: From first administration of study drug to study completion (up to approximately 25 weeks)
measure: Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
timeFrame: From baseline to study completion (up to approximately 25 weeks)
measure: Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
timeFrame: Pre-dose and 30 minutes Post-dose on Day 1 and Day 29 of every Cycle (2, 3, 4)
measure: Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
timeFrame: From first dose of study drug to study completion (up to approximately 25 weeks)
measure: Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
timeFrame: From first administration of study drug to study completion (up to approximately 25 weeks)
Secondary outcomes:
measure: Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing at Each Cycle and Study Completion
timeFrame: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)
measure: Change From Baseline in Forced Vital Capacity (FVC) Percent (%) Predicted to End of Dosing at Each Cycle and Study Completion
timeFrame: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)
measure: Change From Baseline in Forced Expiratory Flow Rate Over 25 Percent and 75 Percent (FEF25-75%) Predicted to End of Dosing at Each Cycle and Study Completion
timeFrame: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)
measure: Absolute Change From Baseline in Sputum Pseudomonas Aeruginosa Density [log10 Colony Forming Units (CFU) Per Gram Sputum] to End of Dosing at Each Cycle and Study Completion
timeFrame: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)
measure: Change From Baseline in Pseudomonas Aeruginosa Minimum Inhibitory Concentration (MIC) to End of Dosing at Each Cycle and Study Completion
timeFrame: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)
measure: Percentage of Participants With Anti-Pseudomonal Antibiotic Use During The Treatment Period
timeFrame: From first administration of study drug to study completion (up to approximately 25 weeks)
measure: Number of Days of Hospitalization Due to Respiratory Serious Adverse Events
timeFrame: From first administration of study drug to study completion (up to approximately 25 weeks)
Other outcomes:
Locations:
city: Tallin
country: Estonia
facility: Novartis Investigative Site
city: Yaroslavl
country: Russian Federation
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Konstan MW, Flume PA, Galeva I, Wan R, Debonnett LM, Maykut RJ, Angyalosi G. One-year safety and efficacy of tobramycin powder for inhalation in patients with cystic fibrosis. Pediatr Pulmonol. 2016 Apr;51(4):372-8. doi: 10.1002/ppul.23358. Epub 2015 Dec 27.