Observational Study in Cystic Fibrosis Patients Using TOBI® PODHALER® or Other FDA Approved Inhaled Antipseudomonal Antibacterial Drugs

Pseudomonas Aeruginosa in Cystic Fibrosis Clinical Trial

Official title:

A Prospective Observational Study in Cystic Fibrosis Patients With Chronic Respiratory Pseudomonas Aeruginosa Infection Treated With TOBI® PODHALER® (Tobramycin Inhalation Powder) or Other FDA Approved Inhaled Antipseudomonal Antibacterial Drugs

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02449031
• Other study ID #: CTBM100C2407
• Status: Completed
• Start date: May 5, 2015
• Est. completion date: December 31, 2021

Study information

• Verified date: March 2022
• Source: Viatris Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

This is a multicenter, prospective, two cohort, observational study over a 5-year period in Cystic Fibrosis (CF) patients with chronic Pseudomonas aeruginosa infection.The study will collect data over 1 year on respiratory function, antibacterial effectiveness, and clinical outcomes of treatment with inhaled antipseudomonal antibiotics and data over 5 years on microbiological and safety assessments.

Description:

This study will include CF patients chronically colonized with P. aeruginosa enrolled in the Cystic Fibrosis Foundation (CFF) PortCF registry and using TOBI® PODHALER® or another FDA-approved inhaled antipseudomonal antibiotic. No therapeutic intervention will be assigned and physicians will use their discretion in choosing a treatment regimen for their patients.

Sputum samples (primarily collected during routine clinical follow-up) from patients able to spontaneously produce sputum will be sent to a central laboratory for analysis.

In addition, this study will include two optional sub-studies for qualifying patients in the first study year - Sputum microbiology sub-study and TOBI® PODHALER® sputum pharmacokinetics (PK) sub-study.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 409
• Est. completion date: December 31, 2021
• Est. primary completion date: December 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

• = 6 years of age.

• Documented FEV1 = 25% predicted in the previous year.

• Diagnosis of cystic fibrosis.

• Established diagnosis of chronic P. aeruginosa infection of the lungs defined as two or more positive P. aeruginosa cultures in the previous year as documented in the subject's medical history (this may include a history of one positive culture in the year prior to enrollment and one positive culture from the specimen collected at the baseline visit).

• Prescribed and initiated chronic treatment with FDA-approved inhaled antipseudomonal antibiotic for chronic P. aeruginosa infection (e.g. TOBI® PODHALER®, TOBI®, Cayston® and Bethkis®).

• Actively enrolled or willingness to enroll in PortCF registry.

• Willing and able to provide written informed consent or, parent/guardian consent and where applicable pediatric assent, for participation and use of relevant clinical data previously captured in PortCF.

• Anticipated to have good adherence to routine visits, defined as the investigator having good knowledge that the patient has been to at least 2-3 routine visits in the previous year.

Exclusion Criteria:

• Documented FEV1 < 25% predicted in the previous year.

• Current participation in an interventional clinical study with an inhaled antibiotic treatment.

• Treatment with compounded tobramycin (e.g. the use of tobramycin IV solution adapted for use by inhalation).

• Treatment with inhaled antipseudomonal antibacterial drug(s) that are not FDA approved.

• Patients undergoing an early eradication regimen for CF (first line therapy).

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Pseudomonas Aeruginosa in Cystic Fibrosis
• Pseudomonas Infections

Intervention

Drug:
• TOBI® PODHALER®

• TOBI®
tobramycin inhalation solution, USP
• Bethkis®
tobramycin inhalation solution
• Cayston®
aztreonam for inhalation solution

Locations

Country	Name	City	State
United States	Novartis Investigative Site	Akron	Ohio
United States	Novartis Investigative Site	Anchorage	Alaska
United States	Novartis Investigative Site	Atlanta	Georgia
United States	Novartis Investigative Site	Austin	Texas
United States	Novartis Investigative Site	Bellflower	California
United States	Novartis Investigative Site	Billings	Montana
United States	Novartis Investigative Site	Boise	Idaho
United States	Novartis Investigative Site	Boston	Massachusetts
United States	Novartis Investigative Site	Burlington	Vermont
United States	Novartis Investigative Site	Charleston	South Carolina
United States	Novartis Investigative Site	Chicago	Illinois
United States	Novartis Investigative Site	Dallas	Texas
United States	Novartis Investigative Site	Detroit	Michigan
United States	Novartis Investigative Site	Fullerton	California
United States	Novartis Investigative Site	Gainesville	Florida
United States	Novartis Investigative Site	Grand Rapids	Michigan
United States	Novartis Investigative Site	Hartford	Connecticut
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	Jackson	Mississippi
United States	Novartis Investigative Site	Kansas City	Missouri
United States	Novartis Investigative Site	Lebanon	New Hampshire
United States	Novartis Investigative Site	Little Rock	Arkansas
United States	Novartis Investigative Site	Los Angeles	California
United States	Novartis Investigative Site	Madera	California
United States	Novartis Investigative Site	Miami	Florida
United States	Novartis Investigative Site	Nashville	Tennessee
United States	Novartis Investigative Site	New Brunswick	New Jersey
United States	Novartis Investigative Site	New Haven	Connecticut
United States	Novartis Investigative Site	New Hyde Park	New York
United States	Novartis Investigative Site	New Orleans	Louisiana
United States	Novartis Investigative Site	Norfolk	Virginia
United States	Novartis Investigative Site	Oklahoma City	Oklahoma
United States	Novartis Investigative Site	Omaha	Nebraska
United States	Novartis Investigative Site	Orlando	Florida
United States	Novartis Investigative Site	Philadelphia	Pennsylvania
United States	Novartis Investigative Site	Philadelphia	Pennsylvania
United States	Novartis Investigative Site	Richmond	Virginia
United States	Novartis Investigative Site	Salt Lake City	Utah
United States	Novartis Investigative Site	Sioux Falls	South Dakota
United States	Novartis Investigative Site	Spokane	Washington
United States	Novartis Investigative Site	Stamford	Connecticut
United States	Novartis Investigative Site	Tampa	Florida
United States	Novartis Investigative Site	Tyler	Texas
United States	Novartis Investigative Site	Winston-Salem	North Carolina
United States	Novartis Investigative Site	Ypsilanti	Michigan

Sponsors (2)

Lead Sponsor	Collaborator
Mylan Inc.	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline.		1 year
Primary	Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum.		1 year
Primary	Minimum inhibitory concentration (MIC) of tobramycin and the following antipseudomonal antibacterial drugs (meropenem, imipenem, ceftazidime, aztreonam and ciprofloxacin) for P. aeruginosa sputum isolates in both treatment cohorts.		Up to 5 years
Primary	Frequency of the following treatment emergent pathogens in sputum: S. aureus (MRSA and MSSA), S. maltophilia, A. xylosoxidans, and Burkholderia spp.in both treatment cohorts.		Up to 5 years
Primary	Number of pulmonary exacerbations and those leading to hospitalization.		1 year
Primary	Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization.		1 year
Primary	Incidence rate of patients with one or more pulmonary exacerbations.		1 year
Primary	Incidence rate of pulmonary exacerbations.		1 year
Primary	Time to first pulmonary exacerbation.		1 year
Primary	Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations.		1 year
Primary	Mortality rate		1 year
Primary	Pharmacokinetic properties of TOBI® PODHALER® as measured by sputum specimens collected during the on-treatment cycles.		1 year
Primary	Number of respiratory related hospitalizations.		1 year
Primary	Duration of stay for respiratory related hospitalizations.		1 year
Primary	Number of non-respiratory related hospitalizations.		1 year
Primary	Duration of stay for non-respiratory related hospitalizations.		1 year
Secondary	Relative change in FEV1 % predicted from baseline.		1 year