Cystic Fibrosis
Conditions
Keywords
Human factors, TOBI® Podhaler™, Instructions For use, IFU, CF patients
Brief summary
The purpose of this actual use human factors (HF) study is to validate the approved US TOBI Podhaler Instructions for Use (IFU), by establishing that the IFU effectively communicates the information necessary to achieve safe and effective use of the Podhaler device.
Detailed description
The objective of the HF study is to determine whether cystic fibrosis patients in the US, representative of potential TOBI Podhaler users, can understand and follow the approved IFU and the extent to which the approved IFU supports safe and effective use of the Podhaler device. This study is an 'actual use' study, in that patients will inhale the contents of placebo capsules through the Podhaler device. Due to the use of placebo capsules, the study is considered a clinical study and will be conducted accordingly. The study is therefore a human factors observational use study conducted within a clinical study. It is an open label, unblinded, non-randomized study and consists of two visits. At visit 2 patients who are eligible will participate in a human factor assessment to determine whether or not a patient understands the IFU content and can demonstrate safe and effective use of the Podhaler device.
Interventions
DRUGPlacebo
one dose (4 capsules) of placebo
DEVICETobi Podhaler
The capsule containing the study medication has to be released from the blister card, be inserted into the Podhaler device, actuated and the study drug be inhaled according to instructions for use
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male and female subjects aged 6 years and older at screening * Confirmed diagnosis of CF * Pulmonary function FEV1 value at least 25% of normal predicted values * Must be physically and cognitively able to read, alone or with the assistance of their caregiver
Exclusion criteria
* Subjects currently enrolled in studies that are not considered observational noninvestigational studies. * Subjects who have used the Podhaler device previously * Hemoptysis more than 60mL at any time within 30 days prior to study drug administration * History of hypersensitivity to inhaled dry powder * Signs and symptoms of acute pulmonary disease, e.g., pneumonia, pneumothorax
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | 1 Day | Recording all use errors and close calls associated with inhalation of one dose of TOBI Podhaler (i.e., inhaling the contents of four placebo capsules via the Podhaler device) by subjects (CF patients, and caregivers, if applicable). The study population consisted of CF patients (and caregivers) naïve to the Podhaler device and untrained in the use of the device. Assessing the root cause of use errors and close calls for 6 defined critical errors (agreed with the FDA) and establishing those which can be attributed to a lack of clarity or presence of ambiguities in the IFU content, i.e., errors attributable to a failure to understand the IFU. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Open Label one dose (4 capsules) of matching placebo to Tobramycin inhalation powder hard capsule | 45 |
| Total | 45 |
Baseline characteristics
| Characteristic | Open Label |
|---|---|
| Age, Continuous | 14.9 Years STANDARD_DEVIATION 7.53 |
| Sex: Female, Male Female | 19 Participants |
| Sex: Female, Male Male | 26 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 0 / 15 | 0 / 45 | 0 / 15 | 0 / 15 |
| serious Total, serious adverse events | 1 / 15 | 1 / 45 | 0 / 15 | 0 / 15 |
Outcome results
Primary
The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device
Recording all use errors and close calls associated with inhalation of one dose of TOBI Podhaler (i.e., inhaling the contents of four placebo capsules via the Podhaler device) by subjects (CF patients, and caregivers, if applicable). The study population consisted of CF patients (and caregivers) naïve to the Podhaler device and untrained in the use of the device. Assessing the root cause of use errors and close calls for 6 defined critical errors (agreed with the FDA) and establishing those which can be attributed to a lack of clarity or presence of ambiguities in the IFU content, i.e., errors attributable to a failure to understand the IFU.
Time frame: 1 Day
Population: The Full Analysis Set (FAS) included all enrolled patients who completed an HF assessment as defined by completion of inhalation of the contents of at least one capsule and related HF assessment.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Open Label | The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | Failure to open blisters | 0 Patients failing to understand IFU |
| Open Label | The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | Attempting to swallow the capsule | 0 Patients failing to understand IFU |
| Open Label | The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | Placing capsule into the mouthpiece | 0 Patients failing to understand IFU |
| Open Label | The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | Failure to pierce any of the 4 capsules (caps)* | 3 Patients failing to understand IFU |
| Open Label | The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | Failure to inhale from all 4 capsules | 0 Patients failing to understand IFU |
| Open Label | The Use of the Approved US TOBI Podhaler Instructions for Use (IFU) to Communicates the Information Necessary to Achieve Safe and Effective Use of the Podhaler Device | Failure to inhale all powder from any of 4 caps† | 6 Patients failing to understand IFU |