Human Factors Study to Validate the User Interface of TOBI Podhaler Using Placebo Capsules

The objective is to conduct a human factors validation study to demonstrate that the user interface of TOBI® Podhaler™ can support safe and effective use for the intended users. The human factors validation study will be conducted in patients with cystic fibrosis aged 6 years and older under simulated yet representative of realistic use conditions where patients with no prior training or use of TOBI® Podhaler™ will be asked to inhale the contents of placebo capsules through the Podhaler device. Due to the use of placebo capsules, the study is considered an interventional clinical study and will be conducted accordingly. The study is an open label, un-blinded, non-randomized study which consists of one visit.

Inclusion criteria

• Written informed consent or, parent/guardian consent and where applicable pediatric assent, must be obtained before any assessment is performed
• Male and female subjects aged 6 years and older
• Confirmed diagnosis of CF by one or more of the following tests for CF as documented in the patient's medical history : (1) quantitative pilocarpine iontophoresis sweat chloride test of > 60 mmol/L or 60 mEq/L, (2) genotype with identifiable CF-causing mutations on both chromosomes, (3) an abnormal nasal transepithelial potential difference characteristic of CF
• FEV1 value must be at least 25% of normal predicted values for age, sex, and height as documented in the patient's medical history (historical values within 3 months can be used for this criterion).
• Able to comply with all protocol requirements
• Clinically stable in the opinion of the investigator

Exclusion criteria

• Subjects currently enrolled in studies that are not considered as observational non-investigational studies
• Subjects or caregivers who have used the Podhaler device previously
• Hemoptysis more than approximately 60 mL at any time within 30 days prior to enrollment
• History of hypersensitivity to the inhaled placebo dry powder (DSPC and/or calcium chloride powder)
• Signs and symptoms of acute pulmonary disease, e.g. pneumonia, pneumothorax, bronchospasm, acute respiratory infection
• Clinically significant conditions or findings at enrollment that might interfere with the accurate and valid assessment of this study
• Subjects or caregivers who are considered potentially unreliable or considered unlikely to be compliant within the trial
• Pregnant women