Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02449031
Recruitment Status : Active, not recruiting
First Posted : May 20, 2015
Last Update Posted : January 4, 2019
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Mylan Inc.

Brief 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.

Condition or disease Intervention/treatment
Pseudomonas Aeruginosa in Cystic Fibrosis Drug: TOBI Podhaler Drug: TOBI Drug: Bethkis Drug: Cayston

Detailed 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.

Layout table for study information
Study Type : Observational [Patient Registry]
Actual Enrollment : 260 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
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
Actual Study Start Date : May 5, 2015
Estimated Primary Completion Date : July 31, 2023
Estimated Study Completion Date : July 31, 2023


Group/Cohort Intervention/treatment
TOBI Podhaler cohort Drug: TOBI Podhaler
non-TOBI Podhaler cohort
Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment
Drug: TOBI
tobramycin inhalation solution, USP

Drug: Bethkis
tobramycin inhalation solution

Drug: Cayston
aztreonam for inhalation solution




Primary Outcome Measures :
  1. Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline. [ Time Frame: 1 year ]
  2. Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum. [ Time Frame: 1 year ]
  3. 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. [ Time Frame: Up to 5 years ]
  4. 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. [ Time Frame: Up to 5 years ]
  5. Number of pulmonary exacerbations and those leading to hospitalization. [ Time Frame: 1 year ]
  6. Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization. [ Time Frame: 1 year ]
  7. Incidence rate of patients with one or more pulmonary exacerbations. [ Time Frame: 1 year ]
  8. Incidence rate of pulmonary exacerbations. [ Time Frame: 1 year ]
  9. Time to first pulmonary exacerbation. [ Time Frame: 1 year ]
  10. Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations. [ Time Frame: 1 year ]
  11. Mortality rate [ Time Frame: 1 year ]
  12. Pharmacokinetic properties of TOBI Podhaler as measured by sputum specimens collected during the on-treatment cycles. [ Time Frame: 1 year ]
  13. Number of respiratory related hospitalizations. [ Time Frame: 1 year ]
  14. Duration of stay for respiratory related hospitalizations. [ Time Frame: 1 year ]
  15. Number of non-respiratory related hospitalizations. [ Time Frame: 1 year ]
  16. Duration of stay for non-respiratory related hospitalizations. [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Relative change in FEV1 % predicted from baseline. [ Time Frame: 1 year ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

CF patients chronically colonized with P. aeruginosa enrolled in the Cystic Fibrosis Foundation (CFF) PortCF registry and using TOBI Podhaler (TOBI Podhaler-treated cohort) or another FDA-approved inhaled antipseudomonal antibiotic (non-TOBI Podhaler-treated cohort).

It is anticipated that this patient population will include a subset of patients with increased P. aeruginosa MICs to tobramycin at baseline.

Criteria

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02449031


Locations
Layout table for location information
United States, Alaska
Novartis Investigative Site
Anchorage, Alaska, United States, 99508
United States, Arkansas
Novartis Investigative Site
Little Rock, Arkansas, United States, 72202
United States, California
Novartis Investigative Site
Bellflower, California, United States, 90706
Novartis Investigative Site
Fullerton, California, United States, 92831
Novartis Investigative Site
Los Angeles, California, United States, 90027
Novartis Investigative Site
Madera, California, United States, 93636
United States, Connecticut
Novartis Investigative Site
Hartford, Connecticut, United States, 06102
Novartis Investigative Site
New Haven, Connecticut, United States, 06519
Novartis Investigative Site
Stamford, Connecticut, United States, 06902
United States, Florida
Novartis Investigative Site
Gainesville, Florida, United States, 32610
Novartis Investigative Site
Miami, Florida, United States, 33136
Novartis Investigative Site
Orlando, Florida, United States, 32803
Novartis Investigative Site
Tampa, Florida, United States, 33606
United States, Georgia
Novartis Investigative Site
Atlanta, Georgia, United States, 30322
United States, Idaho
Novartis Investigative Site
Boise, Idaho, United States, 83712
United States, Illinois
Novartis Investigative Site
Chicago, Illinois, United States, 60611
United States, Indiana
Novartis Investigative Site
Indianapolis, Indiana, United States, 46202-5225
United States, Iowa
Novartis Investigative Site
Iowa City, Iowa, United States, 52242
United States, Louisiana
Novartis Investigative Site
New Orleans, Louisiana, United States, 70112
United States, Massachusetts
Novartis Investigative Site
Boston, Massachusetts, United States, 02115
United States, Michigan
Novartis Investigative Site
Detroit, Michigan, United States, 97205
Novartis Investigative Site
Grand Rapids, Michigan, United States, 49503
Novartis Investigative Site
Ypsilanti, Michigan, United States, 48197
United States, Mississippi
Novartis Investigative Site
Jackson, Mississippi, United States, 39216
United States, Missouri
Novartis Investigative Site
Kansas City, Missouri, United States, 64108
United States, Montana
Novartis Investigative Site
Billings, Montana, United States, 59101
United States, Nebraska
Novartis Investigative Site
Omaha, Nebraska, United States, 68198
United States, New Hampshire
Novartis Investigative Site
Lebanon, New Hampshire, United States, 03756
United States, New Jersey
Novartis Investigative Site
New Brunswick, New Jersey, United States, 8901
United States, New York
Novartis Investigative Site
New Hyde Park, New York, United States, 11040
United States, North Carolina
Novartis Investigative Site
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Novartis Investigative Site
Akron, Ohio, United States, 44308-1062
United States, Oklahoma
Novartis Investigative Site
Oklahoma City, Oklahoma, United States, 73104
United States, Pennsylvania
Novartis Investigative Site
Hershey, Pennsylvania, United States, 17033-085
Novartis Investigative Site
Philadelphia, Pennsylvania, United States, 19104
Novartis Investigative Site
Philadelphia, Pennsylvania, United States, 19107
United States, South Carolina
Novartis Investigative Site
Charleston, South Carolina, United States, 29425
United States, South Dakota
Novartis Investigative Site
Sioux Falls, South Dakota, United States, 57104
United States, Tennessee
Novartis Investigative Site
Nashville, Tennessee, United States, 37232
United States, Texas
Novartis Investigative Site
Austin, Texas, United States, 78723
Novartis Investigative Site
Dallas, Texas, United States, 75390
Novartis Investigative Site
Tyler, Texas, United States, 75708
United States, Utah
Novartis Investigative Site
Salt Lake City, Utah, United States, 84132
United States, Vermont
Novartis Investigative Site
Burlington, Vermont, United States, 5405
United States, Virginia
Novartis Investigative Site
Norfolk, Virginia, United States, 23507
Novartis Investigative Site
Richmond, Virginia, United States, 23298
United States, Washington
Novartis Investigative Site
Spokane, Washington, United States, 99204
Sponsors and Collaborators
Mylan Inc.
Cystic Fibrosis Foundation
Additional Information:

Layout table for additonal information
Responsible Party: Mylan Inc.
ClinicalTrials.gov Identifier: NCT02449031    
Other Study ID Numbers: CTBM100C2407
First Posted: May 20, 2015    Key Record Dates
Last Update Posted: January 4, 2019
Last Verified: January 2019
Additional relevant MeSH terms:
Layout table for MeSH terms
Pseudomonas Infections
Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Gram-Negative Bacterial Infections
Bacterial Infections
Tobramycin
Anti-Bacterial Agents
Anti-Infective Agents