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

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ClinicalTrials.gov Identifier: NCT02449031
Recruitment Status : Active, not recruiting
First Posted : May 20, 2015
Last Update Posted : September 9, 2020
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Mylan Inc.

Tracking Information
First Submitted Date May 4, 2015
First Posted Date May 20, 2015
Last Update Posted Date September 9, 2020
Actual Study Start Date May 5, 2015
Estimated Primary Completion Date July 31, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 15, 2015)
  • 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 ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: May 15, 2015)
Relative change in FEV1 % predicted from baseline. [ Time Frame: 1 year ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
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
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.
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 Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 5 Years
Biospecimen Not Provided
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.

Condition Pseudomonas Aeruginosa in Cystic Fibrosis
Intervention
  • Drug: TOBI Podhaler
  • Drug: TOBI
    tobramycin inhalation solution, USP
  • Drug: Bethkis
    tobramycin inhalation solution
  • Drug: Cayston
    aztreonam for inhalation solution
Study Groups/Cohorts
  • TOBI Podhaler cohort
    Intervention: Drug: TOBI Podhaler
  • non-TOBI Podhaler cohort
    Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment
    Interventions:
    • Drug: TOBI
    • Drug: Bethkis
    • Drug: Cayston
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: May 17, 2018)
260
Original Estimated Enrollment
 (submitted: May 15, 2015)
500
Estimated Study Completion Date July 31, 2023
Estimated Primary Completion Date July 31, 2023   (Final data collection date for primary outcome measure)
Eligibility 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).
Sex/Gender
Sexes Eligible for Study: All
Ages 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02449031
Other Study ID Numbers CTBM100C2407
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Mylan Inc.
Study Sponsor Mylan Inc.
Collaborators Cystic Fibrosis Foundation
Investigators Not Provided
PRS Account Mylan Inc.
Verification Date September 2020