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

Phase 4

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: Placebo; Device: Tobi Podhaler

• Registration Number: NCT03502070
• Lead Sponsor: Mylan Inc.

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.

Study Timeline

• Study First Submitted: 2018-03-29
• Study First Submitted QC: 2018-04-10
• Study First Posted: 2018-04-18
• Study Start: 2018-06-26
• Primary Completion: 2019-03-19
• Study Completion: 2019-06-17
• Results First Submitted: 2020-06-12
• Results First Submitted QC: 2020-06-12
• Results First Posted: 2020-06-30
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-01
• Last Update Posted: 2022-03-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 47

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-Label Placebo	Tobi Podhaler	One dose (4 capsules) of placebo
Open-Label Placebo	Placebo	One dose (4 capsules) of placebo

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (9)

Batchelor Childrens Research Institute; 🇺🇸 Miami, Florida, United States

University of Miami Hospital; 🇺🇸 Miami, Florida, United States

Nemours Childrens Hospital; 🇺🇸 Orlando, Florida, United States

Nemour's Children's Clinic - Pensacola; 🇺🇸 Pensacola, Florida, United States

Tulane Lakeside Hospital for Women and Children; 🇺🇸 New Orleans, Louisiana, United States

Toledo Childrens Hospital; 🇺🇸 Toledo, Ohio, United States

Respiratory Diseases of Children and Adolescents; 🇺🇸 Oklahoma City, Oklahoma, United States

University of Oklahoma Health Science Center; 🇺🇸 Oklahoma City, Oklahoma, United States

VCU Children's Pavilion; 🇺🇸 Richmond, Virginia, United States