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

A Multi-center, Human Factors Engineering (HFE) Usability Study in Cystic Fibrosis Patients to Validate the Approved Instructions for Use (IFU) of TOBI® Podhaler™ (Tobramycin Inhalation Powder) Using Placebo Capsules

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.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: Placebo; Device: Tobi Podhaler

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.

• Study Type: Interventional
• Enrollment (Actual): 45
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Novartis Investigative Site
  • California
    • Long Beach, California, United States, 90806
      • Novartis Investigative Site
    • Los Angeles, California, United States, 90027
      • Novartis Investigative Site
    • Ventura, California, United States, 93003
      • Novartis Investigative Site
  • District of Columbia
    • Washington, District of Columbia, United States, 20010-2120
      • Novartis Investigative Site
  • Florida
    • Jacksonville, Florida, United States, 32207
      • Novartis Investigative Site
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Novartis Investigative Site
  • New York
    • New York, New York, United States, 10595
      • Novartis Investigative Site
  • Ohio
    • Toledo, Ohio, United States, 43606
      • Novartis Investigative Site
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Novartis Investigative Site
    • Oklahoma City, Oklahoma, United States, 73112
      • Novartis Investigative Site
  • Oregon
    • Portland, Oregon, United States, 97239
      • Novartis Investigative Site
  • Texas
    • Houston, Texas, United States, 77030
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Open label; one dose (4 capsules) of placebo	Drug: Placebo; one dose (4 capsules) of placebo; Device: Tobi 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	1 Day

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start: August 1, 2014
• Primary Completion (Actual): February 1, 2015
• Study Completion (Actual): February 1, 2015

Study Registration Dates

• First Submitted: June 24, 2014
• First Submitted That Met QC Criteria: June 27, 2014
• First Posted (Estimate): June 30, 2014

Study Record Updates

• Last Update Posted (Estimate): May 23, 2016
• Last Update Submitted That Met QC Criteria: April 14, 2016
• Last Verified: April 1, 2016

More Information

Terms related to this study

• Keywords: Human factors, TOBI® Podhaler™, Instructions For use, IFU, CF patients
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Anti-Infective Agents; Anti-Bacterial Agents; Tobramycin
• Other Study ID Numbers: CTBM100C2412