Cystic FibrosisPhase 4NCT03502070

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

A Phase 4 study testing Tobi Podhaler in people with Cystic Fibrosis, run by Mylan Inc. at 9 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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 Read more about this study

Who is it for?: Ages 6 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: Tobi Podhaler
Type of treatment: Pill or oral medicine, Medical device
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 47 participants
Where: 9 sitesUnited States
Study dates: 26 Jun 2018to 17 Jun 2019
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 6 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

United States9 sites

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

Other trials in United States

About this study

Official title: 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

Read the 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.

Conditions: Cystic Fibrosis
Being tested: Placebo, Tobi Podhaler
Keywords: CF, Human Factors, TOBI® Podhaler™, user interface, IFU
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PlaceboDrug
One dose (4 capsules) of placebo
Tobi PodhalerDevice
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

Study groups in NCT03502070
Study group	Role	What they receive
Open-Label Placebo	Other	One dose (4 capsules) of placebo Receives: Drug: Placebo; Device: Tobi Podhaler

What is being measured

The primary outcome is the main question the study is designed to answer.

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
Measured: 1 Day

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

26 Jun 201817 Jun 2019

First posted18 Apr 2018
Study start26 Jun 2018
Primary completion19 Mar 2019
Study completion17 Jun 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Other
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 47 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03502070
Sponsor's protocol ID: CTBM100C2419
Status last verified: Mar 2022
First submitted: 29 Mar 2018
Last updated: 3 Mar 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 3 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT03502070
Acronym / sponsor ID: CTBM100C2419
Conditions: Cystic Fibrosis
Keywords: CF, Human Factors, TOBI® Podhaler™, user interface, IFU
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 47 participants (actual)
Lead sponsor: Mylan Inc.Industry (pharma, biotech, devices)
Study start: 26 Jun 2018 (actual)
Primary completion: 19 Mar 2019 (actual)
Study completion: 17 Jun 2019 (actual)
First posted: 18 Apr 2018 (actual)
Results: Posted 30 Jun 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Other
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Open-Label Placebo	Other	Drug: Placebo, Device: Tobi PodhalerOne dose (4 capsules) of placebo

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

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

Key exclusion criteria (8 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PlaceboOne dose (4 capsules) of placebo	Drug	Open-Label Placebo
Tobi PodhalerThe 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	Device	Open-Label Placebo

Primary outcome

Number of Participants With Critical Use Errors and Close Calls Associated With the Simulated Inhalation of One Dose Using TOBI Podhaler
Time frame: 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

Results and safety

Results posted to the registry on 30 Jun 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Open-Label Placebo	47	45

Primary outcome: Number of Participants With Critical Use Errors and Close Calls Associated With the Simulated Inhalation of One Dose Using TOBI Podhaler

Time frame: 1 Day. Count of participants. Unit: Participants

Measure	Open-Label Placebo n = 45
Value	4

Adverse events

Time frame: Adverse events were collected from time of signing Informed Consent through completion of their study visit, 1 day. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Open-Label Placebo	0 of 45 (0%)	1 of 45 (2.2%)	0 of 45

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Cystic Fibrosis

RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 3
Compare Oral Itraconazole and Standard Care Versus Standard Care Alone in Patients With Non-cystic Fibrosis Related Bronchiectasis With Chronic Aspergillus Infection in Reducing Bronchiectasis Exacerbations
Bronchiectasis, Bronchiectasis Adult
Not yet recruitingObservational study
Type 2 Inflammation in Adults With Cystic Fibrosis
Cystic Fibrosis (CF), Type 2 Inflammatory Disorders
Active, not recruitingObservational study
Feasibility and Acceptance of SonoHeal for Airway Clearance and Preliminary Comparison With the Standard of Care
Cystic Fibrosis (CF), Non Cystic Fibrosis Bronchiectasis

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

9 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Tobi Podhaler in people with Cystic Fibrosis, run by Mylan Inc. at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Jun 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Critical Use Errors and Close Calls Associated With the Simulated Inhalation of One Dose Using TOBI Podhaler (time frame: 1 Day). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03502070.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2022.