Pseudomonas Aeruginosa in Cystic FibrosisNCT02449031
Observational Study in Cystic Fibrosis Patients Using TOBI® PODHALER® or Other FDA Approved Inhaled Antipseudomonal Antibacterial Drugs
An observational study of Pseudomonas Aeruginosa in Cystic Fibrosis, run by Mylan Inc. at 47 sites in United States.
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… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Pseudomonas Aeruginosa in Cystic Fibrosis
- What is being tested?
- TOBI® PODHALER®, TOBI® and Bethkis®
- How many people
- 409 participants
- Where
- 47 sitesUnited States
- Study dates
- 5 May 2015to 31 Dec 2021
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- ≥ 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.
Who cannot join
- 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).
Trial locations and contacts
47 sites in 1 country.
47 sites in 1 country
United States47 sites
Novartis Investigative Site
Akron, Ohio, 44308-1062
Novartis Investigative Site
Anchorage, Alaska, 99508
Novartis Investigative Site
Atlanta, Georgia, 30322
Novartis Investigative Site
Austin, Texas, 78723
Novartis Investigative Site
Bellflower, California, 90706
Novartis Investigative Site
Billings, Montana, 59101
Novartis Investigative Site
Boise, Idaho, 83712
Novartis Investigative Site
Boston, Massachusetts, 02115
Novartis Investigative Site
Burlington, Vermont, 5405
Novartis Investigative Site
Charleston, South Carolina, 29425
Novartis Investigative Site
Chicago, Illinois, 60611
Novartis Investigative Site
Dallas, Texas, 75390
Novartis Investigative Site
Detroit, Michigan, 97205
Novartis Investigative Site
Fullerton, California, 92831
Novartis Investigative Site
Gainesville, Florida, 32610
Novartis Investigative Site
Grand Rapids, Michigan, 49503
Novartis Investigative Site
Hartford, Connecticut, 06102
Novartis Investigative Site
Hershey, Pennsylvania, 17033-085
Novartis Investigative Site
Indianapolis, Indiana, 46202-5225
Novartis Investigative Site
Iowa City, Iowa, 52242
Novartis Investigative Site
Jackson, Mississippi, 39216
Novartis Investigative Site
Kansas City, Missouri, 64108
Novartis Investigative Site
Lebanon, New Hampshire, 03756
Novartis Investigative Site
Little Rock, Arkansas, 72202
Novartis Investigative Site
Los Angeles, California, 90027
Novartis Investigative Site
Madera, California, 93636
Novartis Investigative Site
Miami, Florida, 33136
Novartis Investigative Site
Nashville, Tennessee, 37232
Novartis Investigative Site
New Brunswick, New Jersey, 8901
Novartis Investigative Site
New Haven, Connecticut, 06519
Novartis Investigative Site
New Hyde Park, New York, 11040
Novartis Investigative Site
New Orleans, Louisiana, 70112
Novartis Investigative Site
Norfolk, Virginia, 23507
Novartis Investigative Site
Oklahoma City, Oklahoma, 73104
Novartis Investigative Site
Omaha, Nebraska, 68198
Novartis Investigative Site
Orlando, Florida, 32803
Novartis Investigative Site
Philadelphia, Pennsylvania, 19104
Novartis Investigative Site
Philadelphia, Pennsylvania, 19107
Novartis Investigative Site
Richmond, Virginia, 23298
Novartis Investigative Site
Salt Lake City, Utah, 84132
Novartis Investigative Site
Sioux Falls, South Dakota, 57104
Novartis Investigative Site
Spokane, Washington, 99204
Novartis Investigative Site
Stamford, Connecticut, 06902
Novartis Investigative Site
Tampa, Florida, 33606
Novartis Investigative Site
Tyler, Texas, 75708
Novartis Investigative Site
Winston-Salem, North Carolina, 27157
Novartis Investigative Site
Ypsilanti, Michigan, 48197
About this study
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
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.
Read the 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.
- Being tested
- TOBI® PODHALER®, TOBI®, Bethkis®, Cayston®
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TOBI® PODHALER®Drug
TOBI®Drug
tobramycin inhalation solution, USP
Bethkis®Drug
tobramycin inhalation solution
Cayston®Drug
aztreonam for inhalation solution
| Study group | Role | What they receive |
|---|---|---|
| TOBI® PODHALER® cohort | Not described Receives: Drug: TOBI® PODHALER® | |
| non-TOBI® PODHALER® cohort | Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment Receives: Drug: TOBI®; Drug: Bethkis®; Drug: Cayston® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline.
Measured: 1 year
Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum.
Measured: 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.
Measured: 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.
Measured: Up to 5 years
Number of pulmonary exacerbations and those leading to hospitalization.
Measured: 1 year
Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization.
Measured: 1 year
Incidence rate of patients with one or more pulmonary exacerbations.
Measured: 1 year
Incidence rate of pulmonary exacerbations.
Measured: 1 year
Time to first pulmonary exacerbation.
Measured: 1 year
Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations.
Measured: 1 year
Mortality rate
Measured: 1 year
Pharmacokinetic properties of TOBI® PODHALER® as measured by sputum specimens collected during the on-treatment cycles.
Measured: 1 year
Number of respiratory related hospitalizations.
Measured: 1 year
Duration of stay for respiratory related hospitalizations.
Measured: 1 year
Number of non-respiratory related hospitalizations.
Measured: 1 year
Duration of stay for non-respiratory related hospitalizations.
Measured: 1 year
Secondary outcomes (1)
Relative change in FEV1 % predicted from baseline.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2015 (estimated)
- Study start5 May 2015
- Primary completion31 Dec 2021
- Study completion31 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pseudomonas Aeruginosa in Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 409 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Mylan Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02449031
- Sponsor's protocol ID
- CTBM100C2407
- Status last verified
- Mar 2022
- First submitted
- 4 May 2015
- Last updated
- 22 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02449031
- Acronym / sponsor ID
- CTBM100C2407
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 409 participants (actual)
- Lead sponsor
- Mylan Inc.Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 5 May 2015 (actual)
- Primary completion
- 31 Dec 2021 (actual)
- Study completion
- 31 Dec 2021 (actual)
- First posted
- 20 May 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| TOBI® PODHALER® cohort | – | Drug: TOBI® PODHALER® |
| non-TOBI® PODHALER® cohort | – | Drug: TOBI®, Drug: Bethkis®, Drug: Cayston®Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (8 listed)
- ≥ 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®).
Key exclusion criteria (5 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TOBI® PODHALER® | Drug | TOBI® PODHALER® cohort |
| TOBI®tobramycin inhalation solution, USP | Drug | non-TOBI® PODHALER® cohort |
| Bethkis®tobramycin inhalation solution | Drug | non-TOBI® PODHALER® cohort |
| Cayston®aztreonam for inhalation solution | Drug | non-TOBI® PODHALER® cohort |
Primary outcome
Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline.
Time frame: 1 year
Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum.
Time frame: 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.
Time frame: 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.
Time frame: Up to 5 years
Number of pulmonary exacerbations and those leading to hospitalization.
Time frame: 1 year
Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization.
Time frame: 1 year
Incidence rate of patients with one or more pulmonary exacerbations.
Time frame: 1 year
Incidence rate of pulmonary exacerbations.
Time frame: 1 year
Time to first pulmonary exacerbation.
Time frame: 1 year
Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations.
Time frame: 1 year
Mortality rate
Time frame: 1 year
Pharmacokinetic properties of TOBI® PODHALER® as measured by sputum specimens collected during the on-treatment cycles.
Time frame: 1 year
Number of respiratory related hospitalizations.
Time frame: 1 year
Duration of stay for respiratory related hospitalizations.
Time frame: 1 year
Number of non-respiratory related hospitalizations.
Time frame: 1 year
Duration of stay for non-respiratory related hospitalizations.
Time frame: 1 year
1 secondary outcome
Relative change in FEV1 % predicted from baseline.
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
47 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pseudomonas Aeruginosa in Cystic Fibrosis, run by Mylan Inc. at 47 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 47 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline. (time frame: 1 year). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Mar 2022.