Cystic Fibrosis
Conditions
Keywords
CF, Human Factors, TOBI® Podhaler™, user interface, IFU
Brief summary
The purpose of this study is to validate the user interface of TOBI Podhaler by establishing that the product can support safe and effective use for the intended users
Detailed description
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.
Interventions
DRUGPlacebo
One dose (4 capsules) of placebo
DEVICETobi Podhaler
The placebo capsule has to be released from the blister card and inserted into the Podhaler device. The device is then actuated and the study drug is inhaled according to the instructions for use
Sponsors
Mylan Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Critical Use Errors and Close Calls Associated With the Simulated Inhalation of One Dose Using TOBI Podhaler | 1 Day | Critical use errors and close calls for the following: placing of capsule into the top of the mouthpiece, failure to replace mouthpiece, failure to adequately pierce capsule, failure to release before inhaling, multiple piercing of capsule, exhalation into mouthpiece, inadequate seal around mouthpiece, inadequate inhalation technique, failure to use reserve device when capsule not adequately pierced, failure to administer 4 capsules per dose, swallowing of capsule |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Open-Label Placebo One dose (4 capsules) of placebo
Placebo: One dose (4 capsules) of placebo
Tobi Podhaler: The placebo capsule has to be released from the blister card and inserted into the Podhaler device. The device is then actuated and the study drug is inhaled according to the instructions for use | 45 |
| Total | 45 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Screen Failure | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Open-Label Placebo |
|---|---|
| Age, Continuous | 17.5 years STANDARD_DEVIATION 12.55 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 9 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 36 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 41 Participants |
| Sex: Female, Male Female | 21 Participants |
| Sex: Female, Male Male | 24 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 45 |
| other Total, other adverse events | 1 / 45 |
| serious Total, serious adverse events | 0 / 45 |
Outcome results
Primary
Number of Participants With Critical Use Errors and Close Calls Associated With the Simulated Inhalation of One Dose Using TOBI Podhaler
Critical use errors and close calls for the following: placing of capsule into the top of the mouthpiece, failure to replace mouthpiece, failure to adequately pierce capsule, failure to release before inhaling, multiple piercing of capsule, exhalation into mouthpiece, inadequate seal around mouthpiece, inadequate inhalation technique, failure to use reserve device when capsule not adequately pierced, failure to administer 4 capsules per dose, swallowing of capsule
Time frame: 1 Day
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Open-Label Placebo | Number of Participants With Critical Use Errors and Close Calls Associated With the Simulated Inhalation of One Dose Using TOBI Podhaler | 4 Participants |