CoughEarly Phase 1NCT04011709
A Methodology Trial Using the VR647 Inhalation System in Pediatric Subjects Who Have Previously Received Inhaled Therapy
A Early Phase 1 study testing VR647 Inhalation System + VR647 Smart Card 3 Secs, VR647 Inhalation System + VR647 Smart Card 2 Secs and VR647 Inhalation System + VR647 Smart Card 4 Secs in people with Cough and Wheezing, run by Vectura Limited at 5 sites in United States.
Trial summary
This is an open-label, non-drug methodology trial in which subjects undergo a stepwise series of tests to evaluate their ability to use a mouthpiece to deliver nebulized therapy. The trial consists of a screening assessment and familiarization session, and two nebulization assessments, conducted over two site visits. Read more about this study
- Who is it for?
- Ages 1 year to 5 years
- What is being tested?
- VR647 Inhalation System + VR647 Smart Card 3 Secs, VR647 Inhalation System + VR647 Smart Card 2 Secs and VR647 Inhalation System + VR647 Smart Card 4 Secs
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 5 sitesUnited States
- Study dates
- 3 Nov 2017to 20 Feb 2018
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 1 year to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female subjects aged 1 to <5 years
- History of coughing and/or recurrent wheezing within the last year, otherwise healthy
- Use of an inhaler with a spacer or nebulizer for at least 30 days (consecutive or otherwise) within the last year. Examples of inhaled or nebulized therapy include the use of a regular controller (inhaled corticosteroids (ICS) or ICS/long-acting beta-agonists), or a reliever therapy (e.g., albuterol) on an as needed basis for the relief of symptoms
- Written informed consent from the subjects's parent(s)/legal guardian(s), including privacy authorization, prior to trial participation
- Subject's parent(s)/legal guardian(s) must be willing and able to comply with the trial procedures and visit schedules
Who cannot join
- Screening (Visit 1)
- Any significant medical conditions (apart from a history of coughing and/or recurrent wheezing) that in the opinion of the investigator would interfere with the subject's ability to use the mouthpiece and/or follow the protocol procedures
- Subjects with current respiratory symptoms or breathing difficulties at the time of screening
- History of paradoxical bronchospasm or hyper-responsiveness following inhaled therapy that in the opinion of investigator may put the subject at risk when inhaling isotonic saline
- Parent(s)/legal guardian(s) who is an employee of the investigational site or the Sponsor, who is directly involved in the trial, or a family member of such a person
- Nebulization Assessment 2 (Visit 2)
- Development of any new illness or condition between the Screening (Visit 1) and prior to Nebulization Assessment 2 (Visit 2) that in the opinion of the investigator would interfere with the subject's ability to use the mouthpiece and/or follow the protocol procedures
- Any safety concerns that in the opinion of the investigator would jeopardize the subject by his/her continued participation in the trial
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
National Allergy and Asthma Research, LLC
Charleston, South Carolina, 29407
Allergy & Asthma Associates of Southern California
Mission Viejo, California, 92691
Clinical Research Institute, Inc
Plymouth, Minnesota, 55441
North Carolina Clinical Research
Raleigh, North Carolina, 27607
St. Louis Children's Hospital
St Louis, Missouri, 63110
About this study
Official title: An Open-label, Non-drug Methodology Trial to Evaluate the Use of a Mouthpiece With the VR647 Inhalation System During Delivery of Isotonic Saline (0.9% NaCl) in Subjects Less Than Five Years of Age Who Have Previously Received an Inhaled Therapy Delivered Either by a Nebulizer or by a Pressurized Metered-dose Inhaler Plus Spacer
This is an open-label, non-drug methodology trial in which subjects undergo a stepwise series of tests to evaluate their ability to use a mouthpiece to deliver nebulized therapy.
The trial consists of a screening assessment and familiarization session, and two nebulization assessments, conducted over two site visits. The screening assessment, familiarization session and first nebulization assessment will take place at Visit 1, while the second nebulization assessment will take place at Visit 2. The total duration between Visits 1 and 2 will be 4 - 8 days. Each visit is expected to last up to 2 hours.
Read the detailed description
At the initial visit (Visit 1), eligible subjects (i.e., children) and their parent(s)/legal guardian(s) will be approached to ascertain their interest in trial participation. Interested subjects and their parent(s)/legal guardian(s) will proceed to a nebulizer familiarization session. After informed consent has been obtained from the subject's parent(s)/legal guardian(s), the subject will proceed to Nebulization Assessment 1.
At Nebulization Assessment 1 (Visit 1), all subjects will perform 3 attempts of 19 inhalations, each inhalation of 3 seconds duration. Nebulization Assessment 2 (Visit 2) will occur 4-8 days after Visit 1. Subjects, who failed to achieve nebulization success at Attempt 3 during Visit 1, will perform 3 attempts of 28 inhalations, each inhalation of 2 seconds duration at Visit 2. Subjects, who achieved nebulization success at Attempt 3 during Visit 1, will perform 3 attempts of 14 inhalations, each inhalation of 4 seconds duration at Visit 2.
At each visit, Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl). Nebulization success will be assessed at Attempt 3.
- Being tested
- VR647 Inhalation System + VR647 Smart Card 3 Secs, VR647 Inhalation System + VR647 Smart Card 2 Secs, VR647 Inhalation System + VR647 Smart Card 4 Secs
- Keywords
- Nebulizer, Pediatric, Methodological study
- Medical subject headings
- Cough, Respiratory Sounds
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VR647 Inhalation System + VR647 Smart Card 3 SecsCombination product
3 attempts of 19 inhalations, 3 second per breath. Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl)
VR647 Inhalation System + VR647 Smart Card 2 SecsCombination product
3 attempts of 28 inhalations, 2 second per breath. Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl)
VR647 Inhalation System + VR647 Smart Card 4 SecsCombination product
3 attempts of 14 inhalations, 4 second per breath. Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl)
| Study group | Role | What they receive |
|---|---|---|
| Visit 1 and Visit 2, Test Arm 2A | Other | Subjects who carried out 3 attempts at Visit 1, failed to achieve nebulization success and subsequently were included in Test Arm 2A for Visit 2 Receives: Combination Product: VR647 Inhalation System + VR647 Smart Card 3 Secs; Combination Product: VR647 Inhalation System + VR647 Smart Card 2 Secs |
| Visit 1 and Visit 2, Test Arm 2B | Other | Subjects who carried out 3 attempts at Visit 1, achieved nebulization success and were subsequently included in Test Arm 2B for Visit 2 Receives: Combination Product: VR647 Inhalation System + VR647 Smart Card 3 Secs; Combination Product: VR647 Inhalation System + VR647 Smart Card 4 Secs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects achieving nebulization success using a mouthpiece overall and by age (years) at each visit.
Nebulization success using a mouthpiece defined as actuating the nebulizer on at least 70% of planned inhalations and inhaling at least 50% of the total intended inhalation volume.
Measured: 4 to 8 days
Secondary outcomes (7)
Number of breaths completed by subjects using a mouthpiece overall and by age (years) at each visit.
Number of breaths completed by subjects using a mouthpiece recorded at each visit.
Measured: 4 to 8 days
Number of subjects aged 3 to <5 years who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 at each visit.
Number of subjects aged 3 to <5 years who require parent(s)/legal guardian(s) hands-on assistance as assessed (Yes/No) by the Investigator on the inhalation checklist.
Measured: 4 to 8 days
Number of subjects who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 overall and by age (years) at each visit.
Number of subjects who require parent(s)/legal guardian(s) hands-on assistance as assessed (Yes/No) by the Investigator on the inhalation checklist.
Measured: 4 to 8 days
The quality of nebulization at Attempt 3 as recorded in the inhalation checklist overall and by age (years) at each visit.
The inhalation checklist consists of 5 questions regarding inhalation quality answered Yes/No (Q1, Q2, Q3 and Q5) or by percentage (Q4), with Yes answers for Q1-3 and >50% for Q4 (or Yes answers for Q1-3 and Q5 in the successful subgroup analysis) being scored as perfect.
Measured: 4 to 8 days
Mean modified Patient Satisfaction and Preference Questionnaire (PASAPQ) total score at Attempt 3 at Visit 2 overall and by age (years).
The PASAPQ is a validated multi-item measure of satisfaction and preference with inhaler devices. The modified PASAPQ consists of 10 questions. The first 8 questions generate the total score domain (scale: 1 (i.e., very dissatisfied) to 7 (i.e., very satisfied); range: 14.3 to 100, calculated as ((Q1+Q2+Q3+Q4+Q5+Q6+Q7+Q8)/56)*100).
Measured: 4 to 8 days
Mean modified PASAPQ satisfaction score at Attempt 3 at Visit 2 overall and by age (years).
The PASAPQ is a validated multi-item measure of satisfaction and preference with inhaler devices. The modified PASAPQ consists of 10 questions. Question 9 asks for overall satisfaction with the device(s) used in the trial (scale: 1 (i.e., very dissatisfied) to 7 (i.e., very satisfied); range: 1-7).
Measured: 4 to 8 days
Mean modified PASAPQ score indicating willingness to continue with the device at Attempt 3 at Visit 2 overall and by age (years).
The PASAPQ is a validated multi-item measure of satisfaction and preference with inhaler devices. The modified PASAPQ consists of 10 questions. Question 10 asks about willingness to continue with the device(s) used in the trial (scale: 0 (i.e, not willing) to 100 (i.e., definitely willing; range: 0-100).
Measured: 4 to 8 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2019
- Study start3 Nov 2017
- Primary completion20 Feb 2018
- Study completion20 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CoughStudies 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
- Early Phase 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- This open-label, non-drug methodology trial consists of a screening assessment, familiarization session and a nebulization assessment at one visit and a second nebulization assessment at a second visit.
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vectura Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gary Burgess, MD, Study director, Vectura Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04011709
- Sponsor's protocol ID
- VR647/0/003
- Status last verified
- Jul 2019
- First submitted
- 17 Jun 2019
- Last updated
- 8 Jul 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-drug Methodology Trial to Evaluate the Use of a Mouthpiece With the VR647 Inhalation System During Delivery of Isotonic Saline (0.9% NaCl) in Subjects Less Than Five Years of Age Who Have Previously Received an Inhaled Therapy Delivered Either by a Nebulizer or by a Pressurized Metered-dose Inhaler Plus Spacer
- ClinicalTrials.gov ID
- NCT04011709
- Acronym / sponsor ID
- VR647/0/003
- Keywords
- Nebulizer, Pediatric, Methodological study
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Vectura LimitedIndustry (pharma, biotech, devices)
- Study start
- 3 Nov 2017 (actual)
- Primary completion
- 20 Feb 2018 (actual)
- Study completion
- 20 Feb 2018 (actual)
- First posted
- 8 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, non-drug methodology trial in which subjects undergo a stepwise series of tests to evaluate their ability to use a mouthpiece to deliver nebulized therapy.
The trial consists of a screening assessment and familiarization session, and two nebulization assessments, conducted over two site visits. The screening assessment, familiarization session and first nebulization assessment will take place at Visit 1, while the second nebulization assessment will take place at Visit 2. The total duration between Visits 1 and 2 will be 4 - 8 days. Each visit is expected to last up to 2 hours.
Detailed description
At the initial visit (Visit 1), eligible subjects (i.e., children) and their parent(s)/legal guardian(s) will be approached to ascertain their interest in trial participation. Interested subjects and their parent(s)/legal guardian(s) will proceed to a nebulizer familiarization session. After informed consent has been obtained from the subject's parent(s)/legal guardian(s), the subject will proceed to Nebulization Assessment 1.
At Nebulization Assessment 1 (Visit 1), all subjects will perform 3 attempts of 19 inhalations, each inhalation of 3 seconds duration. Nebulization Assessment 2 (Visit 2) will occur 4-8 days after Visit 1. Subjects, who failed to achieve nebulization success at Attempt 3 during Visit 1, will perform 3 attempts of 28 inhalations, each inhalation of 2 seconds duration at Visit 2. Subjects, who achieved nebulization success at Attempt 3 during Visit 1, will perform 3 attempts of 14 inhalations, each inhalation of 4 seconds duration at Visit 2.
At each visit, Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl). Nebulization success will be assessed at Attempt 3.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This open-label, non-drug methodology trial consists of a screening assessment, familiarization session and a nebulization assessment at one visit and a second nebulization assessment at a second visit.
| Arm | Type | Receives |
|---|---|---|
| Visit 1 and Visit 2, Test Arm 2A | Other | Combination Product: VR647 Inhalation System + VR647 Smart Card 3 Secs, Combination Product: VR647 Inhalation System + VR647 Smart Card 2 SecsSubjects who carried out 3 attempts at Visit 1, failed to achieve nebulization success and subsequently were included in Test Arm 2A for Visit 2 |
| Visit 1 and Visit 2, Test Arm 2B | Other | Combination Product: VR647 Inhalation System + VR647 Smart Card 3 Secs, Combination Product: VR647 Inhalation System + VR647 Smart Card 4 SecsSubjects who carried out 3 attempts at Visit 1, achieved nebulization success and were subsequently included in Test Arm 2B for Visit 2 |
Who may qualify
- Ages
- 1 year to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Male or female subjects aged 1 to <5 years
- History of coughing and/or recurrent wheezing within the last year, otherwise healthy
- Use of an inhaler with a spacer or nebulizer for at least 30 days (consecutive or otherwise) within the last year. Examples of inhaled or nebulized therapy include the use of a regular controller (inhaled corticosteroids (ICS) or ICS/long-acting beta-agonists), or a reliever therapy (e.g., albuterol) on an as needed basis for the relief of symptoms
- Written informed consent from the subjects's parent(s)/legal guardian(s), including privacy authorization, prior to trial participation
- Subject's parent(s)/legal guardian(s) must be willing and able to comply with the trial procedures and visit schedules
Key exclusion criteria (8 listed)
- Screening (Visit 1)
- Any significant medical conditions (apart from a history of coughing and/or recurrent wheezing) that in the opinion of the investigator would interfere with the subject's ability to use the mouthpiece and/or follow the protocol procedures
- Subjects with current respiratory symptoms or breathing difficulties at the time of screening
- History of paradoxical bronchospasm or hyper-responsiveness following inhaled therapy that in the opinion of investigator may put the subject at risk when inhaling isotonic saline
- Parent(s)/legal guardian(s) who is an employee of the investigational site or the Sponsor, who is directly involved in the trial, or a family member of such a person
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 |
|---|---|---|
| VR647 Inhalation System + VR647 Smart Card 3 Secs3 attempts of 19 inhalations, 3 second per breath. Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl) | Combination product | Visit 1 and Visit 2, Test Arm 2A, Visit 1 and Visit 2, Test Arm 2B |
| VR647 Inhalation System + VR647 Smart Card 2 Secs3 attempts of 28 inhalations, 2 second per breath. Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl) | Combination product | Visit 1 and Visit 2, Test Arm 2A |
| VR647 Inhalation System + VR647 Smart Card 4 Secs3 attempts of 14 inhalations, 4 second per breath. Attempts 1 and 2 will be made with air only. Attempt 3 will be made with isotonic saline (0.9% NaCl) | Combination product | Visit 1 and Visit 2, Test Arm 2B |
Primary outcome
Number of subjects achieving nebulization success using a mouthpiece overall and by age (years) at each visit.
Time frame: 4 to 8 days
Nebulization success using a mouthpiece defined as actuating the nebulizer on at least 70% of planned inhalations and inhaling at least 50% of the total intended inhalation volume.
7 secondary outcomes
Number of breaths completed by subjects using a mouthpiece overall and by age (years) at each visit.
Time frame: 4 to 8 days
Number of subjects aged 3 to <5 years who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 at each visit.
Time frame: 4 to 8 days
Number of subjects who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 overall and by age (years) at each visit.
Time frame: 4 to 8 days
The quality of nebulization at Attempt 3 as recorded in the inhalation checklist overall and by age (years) at each visit.
Time frame: 4 to 8 days
Mean modified Patient Satisfaction and Preference Questionnaire (PASAPQ) total score at Attempt 3 at Visit 2 overall and by age (years).
Time frame: 4 to 8 days
Mean modified PASAPQ satisfaction score at Attempt 3 at Visit 2 overall and by age (years).
Time frame: 4 to 8 days
Mean modified PASAPQ score indicating willingness to continue with the device at Attempt 3 at Visit 2 overall and by age (years).
Time frame: 4 to 8 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2019.
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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.
Gary Burgess, MD study director
Vectura Ltd
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing VR647 Inhalation System + VR647 Smart Card 3 Secs, VR647 Inhalation System + VR647 Smart Card 2 Secs and VR647 Inhalation System + VR647 Smart Card 4 Secs in people with Cough and Wheezing, run by Vectura Limited at 5 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 5 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 8 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 subjects achieving nebulization success using a mouthpiece overall and by age (years) at each visit. (time frame: 4 to 8 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Jul 2019.