Hartford, Connecticut 06102

  • Pseudomonas Aeruginosa in Cystic Fibrosis

Purpose:

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.


Study summary:

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.


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).


Study is Available At:


Original ID:

CTBM100C2407


NCT ID:

NCT02449031


Secondary ID:


Study Acronym:


Brief Title:

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


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


Overall Status:

Active, not recruiting


Study Phase:

N/A


Genders:

N/A


Minimum Age:

6 Years


Maximum Age:

N/A


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Mylan Inc.


Oversight Authority:

United States: Food and Drug Administration


Reasons Why Stopped:


Study Type:

Observational [Patient Registry]


Study Design:


Number of Arms:

0


Number of Groups:

2


Total Enrollment:

260


Enrollment Type:

Actual


Study Dates

Start Date:May 5, 2015
Completion Date:July 31, 2023
Completion Type:Anticipated
Primary Completion Date:July 31, 2023
Primary Completion Type:Anticipated
Verification Date:January 2019
Last Changed Date:January 3, 2019
First Received Date:May 4, 2015

Study Outcomes

Outcome Type:Secondary Outcome
Measure:Relative change in FEV1 % predicted from baseline.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Duration of stay for non-respiratory related hospitalizations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Number of non-respiratory related hospitalizations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Duration of stay for respiratory related hospitalizations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Number of respiratory related hospitalizations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Pharmacokinetic properties of TOBI Podhaler as measured by sputum specimens collected during the on-treatment cycles.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Mortality rate
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Time to first pulmonary exacerbation.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Incidence rate of pulmonary exacerbations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Incidence rate of patients with one or more pulmonary exacerbations.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Number of pulmonary exacerbations and those leading to hospitalization.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure: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
Safety Issues:False
Outcome Type:Primary Outcome
Measure: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
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum.
Time Frame:1 year
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline.
Time Frame:1 year
Safety Issues:False

Study Interventions

Intervention Type:Drug
Name:TOBI Podhaler
Arm Name:TOBI Podhaler cohort
Intervention Type:Drug
Name:TOBI
Description:tobramycin inhalation solution, USP
Arm Name:non-TOBI Podhaler cohort
Intervention Type:Drug
Name:Bethkis
Description:tobramycin inhalation solution
Arm Name:non-TOBI Podhaler cohort
Intervention Type:Drug
Name:Cayston
Description:aztreonam for inhalation solution
Arm Name:non-TOBI Podhaler cohort

Study Arms

Study Arm Type:Other
Arm Name:TOBI Podhaler cohort
Study Arm Type:Other
Arm Name:non-TOBI Podhaler cohort
Description:Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment

Study Agencies

Agency Class:Industry
Agency Type:Lead Sponsor
Agency Name:Mylan Inc.
Agency Class:Other
Agency Type:Collaborator
Agency Name:Cystic Fibrosis Foundation

Samples and Retentions

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.
Sample Method:Non-Probability Sample

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: April 07, 2020

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