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

A Multicenter, Human Factors Validation Study in Cystic Fibrosis Patients Aged 6 Years and Older to Evaluate the User Interface of TOBI® Podhaler™ (Tobramycin Inhalation Powder) Using Placebo Capsules

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

Study Overview

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

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.

• Study Type: Interventional
• Enrollment (Actual): 47
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami Hospital
    • Miami, Florida, United States, 33136
      • Batchelor Childrens Research Institute
    • Orlando, Florida, United States, 32827
      • Nemours Childrens Hospital
    • Pensacola, Florida, United States, 32504
      • Nemour's Children's Clinic - Pensacola
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • Tulane Lakeside Hospital for Women and Children
  • Ohio
    • Toledo, Ohio, United States, 43606
      • Toledo Childrens Hospital
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • University of Oklahoma Health Science Center
    • Oklahoma City, Oklahoma, United States, 73112
      • Respiratory Diseases of Children and Adolescents
  • Virginia
    • Richmond, Virginia, United States, 23298
      • VCU Children's Pavilion

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Open-Label Placebo; One dose (4 capsules) of placebo	Drug: Placebo; One dose (4 capsules) of placebo; Device: 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	1 Day

Collaborators and Investigators

• Sponsor: Mylan Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 26, 2018
• Primary Completion (Actual): March 19, 2019
• Study Completion (Actual): June 17, 2019

Study Registration Dates

• First Submitted: March 29, 2018
• First Submitted That Met QC Criteria: April 10, 2018
• First Posted (Actual): April 18, 2018

Study Record Updates

• Last Update Posted (Actual): March 3, 2022
• Last Update Submitted That Met QC Criteria: March 1, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Keywords: CF, Human Factors, TOBI® Podhaler™, user interface, IFU
• 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: CTBM100C2419

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes