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

NCT02449031 | Completed

• 1 other identifier: CTBM100C2407
• Study Type: observational
• Target: 409
• Locations: 1 country
• Sites: 47

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.

Conditions

Pseudomonas Aeruginosa in Cystic Fibrosis

Outcome Measures

Primary Outcomes (16)

• Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline.

  1 year

• Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum.

  1 year

• 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

• 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

• Number of pulmonary exacerbations and those leading to hospitalization.

  1 year

• Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization.

  1 year

• Incidence rate of patients with one or more pulmonary exacerbations.

  1 year

• Incidence rate of pulmonary exacerbations.

  1 year

• Time to first pulmonary exacerbation.

  1 year

• Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations.

  1 year

• Mortality rate

  1 year

• Pharmacokinetic properties of TOBI® PODHALER® as measured by sputum specimens collected during the on-treatment cycles.

  1 year

• Number of respiratory related hospitalizations.

  1 year

• Duration of stay for respiratory related hospitalizations.

  1 year

• Number of non-respiratory related hospitalizations.

  1 year

• Duration of stay for non-respiratory related hospitalizations.

  1 year

Secondary Outcomes (1)

• Relative change in FEV1 % predicted from baseline.

  1 year

Study Arms (2)

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®; Drug: Bethkis®; Drug: Cayston®

Interventions

TOBI® PODHALER® DRUG

TOBI® PODHALER® cohort

TOBI® DRUG

tobramycin inhalation solution, USP

non-TOBI® PODHALER® cohort

Bethkis® DRUG

tobramycin inhalation solution

non-TOBI® PODHALER® cohort

Cayston® DRUG

aztreonam for inhalation solution

non-TOBI® PODHALER® cohort

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• ≥ 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Mylan Inc.: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (47)

Novartis Investigative Site

Anchorage, Alaska, 99508, United States

Location

Novartis Investigative Site

Little Rock, Arkansas, 72202, United States

Location

Novartis Investigative Site

Bellflower, California, 90706, United States

Location

Novartis Investigative Site

Fullerton, California, 92831, United States

Location

Novartis Investigative Site

Los Angeles, California, 90027, United States

Location

Novartis Investigative Site

Madera, California, 93636, United States

Location

Novartis Investigative Site

Hartford, Connecticut, 06102, United States

Location

Novartis Investigative Site

New Haven, Connecticut, 06519, United States

Location

Novartis Investigative Site

Stamford, Connecticut, 06902, United States

Location

Novartis Investigative Site

Gainesville, Florida, 32610, United States

Location

Novartis Investigative Site

Miami, Florida, 33136, United States

Location

Novartis Investigative Site

Orlando, Florida, 32803, United States

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Novartis Investigative Site

Tampa, Florida, 33606, United States

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Novartis Investigative Site

Atlanta, Georgia, 30322, United States

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Novartis Investigative Site

Boise, Idaho, 83712, United States

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Novartis Investigative Site

Chicago, Illinois, 60611, United States

Location

Novartis Investigative Site

Indianapolis, Indiana, 46202-5225, United States

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Novartis Investigative Site

Iowa City, Iowa, 52242, United States

Location

Novartis Investigative Site

New Orleans, Louisiana, 70112, United States

Location

Novartis Investigative Site

Boston, Massachusetts, 02115, United States

Location

Novartis Investigative Site

Detroit, Michigan, 97205, United States

Location

Novartis Investigative Site

Grand Rapids, Michigan, 49503, United States

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Novartis Investigative Site

Ypsilanti, Michigan, 48197, United States

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Novartis Investigative Site

Jackson, Mississippi, 39216, United States

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Novartis Investigative Site

Kansas City, Missouri, 64108, United States

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Novartis Investigative Site

Billings, Montana, 59101, United States

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Novartis Investigative Site

Omaha, Nebraska, 68198, United States

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Novartis Investigative Site

Lebanon, New Hampshire, 03756, United States

Location

Novartis Investigative Site

New Brunswick, New Jersey, 8901, United States

Location

Novartis Investigative Site

New Hyde Park, New York, 11040, United States

Location

Novartis Investigative Site

Winston-Salem, North Carolina, 27157, United States

Location

Novartis Investigative Site

Akron, Ohio, 44308-1062, United States

Location

Novartis Investigative Site

Oklahoma City, Oklahoma, 73104, United States

Location

Novartis Investigative Site

Hershey, Pennsylvania, 17033-085, United States

Location

Novartis Investigative Site

Philadelphia, Pennsylvania, 19104, United States

Location

Novartis Investigative Site

Philadelphia, Pennsylvania, 19107, United States

Location

Novartis Investigative Site

Charleston, South Carolina, 29425, United States

Location

Novartis Investigative Site

Sioux Falls, South Dakota, 57104, United States

Location

Novartis Investigative Site

Nashville, Tennessee, 37232, United States

Location

Novartis Investigative Site

Austin, Texas, 78723, United States

Location

Novartis Investigative Site

Dallas, Texas, 75390, United States

Location

Novartis Investigative Site

Tyler, Texas, 75708, United States

Location

Novartis Investigative Site

Salt Lake City, Utah, 84132, United States

Location

Novartis Investigative Site

Burlington, Vermont, 5405, United States

Location

Novartis Investigative Site

Norfolk, Virginia, 23507, United States

Location

Novartis Investigative Site

Richmond, Virginia, 23298, United States

Location

Novartis Investigative Site

Spokane, Washington, 99204, United States

Location

MeSH Terms

Interventions

Tobramycin; Aztreonam

Intervention Hierarchy (Ancestors)

Nebramycin; Kanamycin; Aminoglycosides; Glycosides; Carbohydrates; Monobactams; beta-Lactams; Lactams; Amides; Organic Chemicals; Sulfur Compounds; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2015
• First Posted: May 20, 2015
• Study Start: May 5, 2015
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2021
• Last Updated: March 22, 2022

Record last verified: 2022-03

Locations

US (47)