Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02015663
• Other study ID #: CTBM100CUS03
• Status: Terminated
• Phase: Phase 4
• First received: December 13, 2013
• Last updated: February 4, 2015
• Start date: January 2014
• Est. completion date: December 2015

Study information

• Verified date: February 2015
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 31
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Tobramycin Inhalation Powder
Tobramycin Inhalation Powder 112 mg (four 28-mg capsules) taken via inhaler once or twice a day, depending on study arm

Locations

Country	Name	City	State
United States	Novartis Investigative Site	Akron	Ohio
United States	Novartis Investigative Site	Austin	Texas
United States	Novartis Investigative Site	Glenview	Illinois
United States	Novartis Investigative Site	Hershey	Pennsylvania
United States	Novartis Investigative Site	Indianapolis	Indiana
United States	Novartis Investigative Site	Iowa City	Iowa
United States	Novartis Investigative Site	Jacksonville	Florida
United States	Novartis Investigative Site	Los Angeles	California
United States	Novartis Investigative Site	Miami	Florida
United States	Novartis Investigative Site	Mobile	Alabama
United States	Novartis Investigative Site	New Hyde Park	New York
United States	Novartis Investigative Site	New York	New York
United States	Novartis Investigative Site	Oklahoma City	Oklahoma
United States	Novartis Investigative Site	Orlando	Florida
United States	Novartis Investigative Site	Orlando	Florida
United States	Novartis Investigative Site	Pensacola	Florida
United States	Novartis Investigative Site	Portland	Oregon
United States	Novartis Investigative Site	Sacramento	California
United States	Novartis Investigative Site	Salt Lake City	Utah
United States	Novartis Investigative Site	St. Louis	Missouri
United States	Novartis Investigative Site	Ventura	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in Forced Expiratory Volume in 1 second ( FEV1) percent predicted	The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.	Baseline and Day 168	No
Secondary	Percent change from baseline in Forced Expiratory Volume in 1 second (FEV1) percent predicted	The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.	Baseline and Day 168	No
Secondary	Percent change from baseline in Forced Vital Capacity (FVC) percent predicted	Forced Vital Capacity (FVC) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. FVC will be assessed via spirometry. A positive change from baseline in FVC indicates improvement in lung function.	Baseline and Day 168	No
Secondary	Percent change from baseline in forced expiratory flow (FEF) 25%-75% predicted	The Forced Expiratory Flow (FEF) 25%-75% measurement describes the amount of air expelled from the lungs during the middle half (25% - 75%) of the forced vital capacity test and is measured using spirometry. A positive change from baseline in FEF indicates improvement in lung function. The predicted percent will be assessed.	Baseline and day 168	No
Secondary	Change from baseline in Pseudomonas aeruginosa sputum density	Change from baseline in Pseudomonas aeruginosa sputum density will be measured by log10 colony forming units per gram of sputum.	Baseline and day 168	No
Secondary	Time to first hospitalization due to respiratory-related events	Time to the first hospitalization due to respiratory-related events (number of days) per patient.	Day 1 to day 168	Yes
Secondary	Percentage of patients with hospitalizations due to respiratory-related events	Percentage of patients with hospitalization due to respiratory-related events	Day 1 to day 168	Yes
Secondary	Length of hospital stay due to respiratory-related events	The number of days in length of hospital stay per patient due to respiratory-related events will be measured.	Day 1 to day 168	Yes
Secondary	Time to first usage of anti-pseudomonal antibiotic	Time to first usage of anti-pseudomonal antibiotic per patient will be assessed by number of days	Day 1 to day 168	No
Secondary	Percentage of patients who use anti-pseudomonal antibiotic	Percentage of patients who use anti-pseudomonal antibiotic will be assessed.	Day 1 to day 168	No
Secondary	Duration of use of anti-pseudomonal antibiotic	Number of days of use of anti-pseudomonal antibiotic per patient will be assessed.	Day 1 to day 168	No
Secondary	Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa	Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa will be measured by laboratory testing.	Baseline and day 168	Yes