Trial Outcomes & Findings for Study to Validate the Instructions for Use of TOBI® Podhaler™ in Cystic Fibrosis Patients (NCT NCT02178540)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

45 participants

Primary outcome timeframe

1 Day

Results posted on

2016-05-23

Participant Flow

Participant milestones

Participant milestones
Measure	Open Label one dose (4 capsules) of matching placebo to Tobramycin inhalation powder hard capsule
Overall Study STARTED	45
Overall Study COMPLETED	45
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Validate the Instructions for Use of TOBI® Podhaler™ in Cystic Fibrosis Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open Label n=45 Participants one dose (4 capsules) of matching placebo to Tobramycin inhalation powder hard capsule
Age, Continuous	14.9 Years STANDARD_DEVIATION 7.53 • n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	26 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Open Label n=45 Participants one dose (4 capsules) of matching placebo to Tobramycin inhalation powder hard capsule
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
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
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
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
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
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

Adverse Events

Open Label (6-10) Years Old

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Total - All Participants

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Open Label (11-17)Years Old

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Open Label (>=18) Years Old

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Open Label (6-10) Years Old n=15 participants at risk one dose (4 capsules) of matching placebo to Tobramycin inhalation powder hard capsule	Total - All Participants n=45 participants at risk	Open Label (11-17)Years Old n=15 participants at risk	Open Label (>=18) Years Old n=15 participants at risk
Infections and infestations Bronchopneumonia	6.7% 1/15	2.2% 1/45	0.00% 0/15	0.00% 0/15
Investigations Pulmonary function test decreased	6.7% 1/15	2.2% 1/45	0.00% 0/15	0.00% 0/15

Other adverse events

Adverse event data not reported

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial
Publication restrictions are in place

Restriction type: OTHER