Clinical trial NCT02015663: Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles
Official title: A 24-week, Open-label, Parallel-group, Interventional Phase IV Study Comparing Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles for the Treatment of Pulmonary Pseudomonas Aeruginosa in Patients With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
CF
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-12-19
Enrollment: 32
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.


Eligibility criteria: Inclusion Criteria:

1. Provide written informed consent, HIPPA (Health Insurance Portability and Accountability Act) authorization (where applicable), and assent (as appropriate) prior to the performance of any study-related procedure.
2. Confirmed diagnosis of CF
3. FEV1 at screening (Visit 1) ≥25% and ≤ 80% of normal predicted values for age, sex, and height
4. P. aeruginosa must be present within 6 months prior to screening and at screening
5. Able to comply with all protocol requirements
6. Clinically stable in the opinion of the investigator

Exclusion Criteria:

1. History of Burkholderia cenocepacia (Bcc) complex within 2 years prior to screening and/or Bcc complex at screening
2. Hemoptysis more than 60 cc at any time within 30 days prior to study drug administration
3. History of hearing loss or chronic tinnitus deemed clinically significant by the investigator
4. Serum creatinine 2 mg/dL or greater, BUN 40 mg/dL or greater, or an abnormal urinalysis defined as 2+ or greater proteinuria at screening
5. Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics
6. Patients who are unable to discontinue previously received inhaled antibiotic regimen(s) (inhaled antibiotics are not allowed other than study drug)
7. Use of inhaled aminoglycosides within 28 days prior to study drug administration (Visit 2)
8. Use of systemic anti-pseudomonal antibiotics within 28 days prior to study drug administration
9. Use of loop diuretics within 7 days prior to study drug administration
10. Administration of any investigational drug within 30 days prior to enrollment or 5 half-lives, whichever is longer
11. Signs and symptoms of acute pulmonary disease, e.g , pneumonia, pneumothorax
12. Hospitalization during the baseline visit
13. History of malignancy
14. Patients with clinically significant laboratory abnormalities (not associated with the study indication) at screening
15. Patients with other clinically significant conditions (not associated with the study indication) which might interfere with the assessment of this study
16. Patients or caregivers with a history of noncompliance to medical regimens and patients or caregivers who are considered potentially unreliable
17. Pregnant or nursing (lactating) women
18. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods.

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted
timeFrame: Baseline and Day 168
Secondary outcomes:
measure: Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted
timeFrame: Baseline and Day 168
measure: Percent Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted
timeFrame: Baseline and Day 168
measure: Percent Change From Baseline in Forced Expiratory Flow (FEF) 25%-75% Predicted
timeFrame: Baseline and day 168
measure: Change From Baseline in Pseudomonas Aeruginosa Sputum Density
timeFrame: Baseline and day 168
measure: Time to First Hospitalization Due to Respiratory-related Events
timeFrame: Day 1 to day 168
measure: Percentage of Patients With Hospitalizations Due to Respiratory-related Events
timeFrame: Day 1 to day 168
measure: Length of Hospital Stay Due to Respiratory-related Events
timeFrame: Day 1 to day 168
measure: Time to First Usage of Anti-pseudomonal Antibiotic
timeFrame: Day 1 to day 168
measure: Percentage of Patients Who Use Anti-pseudomonal Antibiotic
timeFrame: Day 1 to day 168
measure: Duration of Use of Anti-pseudomonal Antibiotic
timeFrame: Day 1 to day 168
measure: Change From Baseline in Tobramycin Minimal Inhibitory Concentration for Pseudomonas Aeruginosa
timeFrame: Baseline and day 168
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Novartis Investigative Site
state: Alabama
city: Los Angeles
country: United States
facility: Novartis Investigative Site
state: California
city: Sacramento
country: United States
facility: Novartis Investigative Site
state: California
city: Ventura
country: United States
facility: Novartis Investigative Site
state: California
city: Altamonte Springs
country: United States
facility: Novartis Investigative Site
state: Florida
city: Jacksonville
country: United States
facility: Novartis Investigative Site
state: Florida
city: Miami
country: United States
facility: Novartis Investigative Site
state: Florida
city: Orlando
country: United States
facility: Novartis Investigative Site
state: Florida
city: Pensacola
country: United States
facility: Novartis Investigative Site
state: Florida
city: Glenview
country: United States
facility: Novartis Investigative Site
state: Illinois
city: Indianapolis
country: United States
facility: Novartis Investigative Site
state: Indiana
city: Iowa City
country: United States
facility: Novartis Investigative Site
state: Iowa
city: St. Louis
country: United States
facility: Novartis Investigative Site
state: Missouri
city: New Hyde Park
country: United States
facility: Novartis Investigative Site
state: New York
city: New York
country: United States
facility: Novartis Investigative Site
state: New York
city: Akron
country: United States
facility: Novartis Investigative Site
state: Ohio
city: Oklahoma City
country: United States
facility: Novartis Investigative Site
state: Oklahoma
city: Portland
country: United States
facility: Novartis Investigative Site
state: Oregon
city: Hershey
country: United States
facility: Novartis Investigative Site
state: Pennsylvania
city: Austin
country: United States
facility: Novartis Investigative Site
state: Texas
city: Salt Lake City
country: United States
facility: Novartis Investigative Site
state: Utah
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: