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A Methodology Trial Using the VR647 Inhalation System in Pediatric Subjects Who Have Previously Received Inhaled Therapy

Primary Purpose

Cough, Wheezing

Status

Completed

Phase

Early Phase 1

Locations

United States

Study Type

Interventional

Intervention

VR647 Inhalation System + VR647 Smart Card 3 Secs

VR647 Inhalation System + VR647 Smart Card 2 Secs

VR647 Inhalation System + VR647 Smart Card 4 Secs

About this trial

This is an interventional other trial for Cough focused on measuring Nebulizer, Pediatric, Methodological study

Eligibility Criteria

1 Year - 5 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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

Sites / Locations

Allergy & Asthma Associates of Southern California
Clinical Research Institute, Inc
St. Louis Children's Hospital
North Carolina Clinical Research
National Allergy and Asthma Research, LLC

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Arm Label

Visit 1 and Visit 2, Test Arm 2A

Visit 1 and Visit 2, Test Arm 2B

Arm Description

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

Subjects who carried out 3 attempts at Visit 1, achieved nebulization success and were subsequently included in Test Arm 2B for Visit 2

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

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.

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.

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.

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).

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).

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).

Full Information

NCT ID

NCT04011709

First Posted

June 17, 2019

Last Updated

July 4, 2019

Sponsor

Vectura Limited

1. Study Identification

Unique Protocol Identification Number

NCT04011709

Brief Title

A Methodology Trial Using the VR647 Inhalation System in Pediatric Subjects Who Have Previously Received Inhaled Therapy

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2019

Overall Recruitment Status

Completed

Study Start Date

November 3, 2017 (Actual)

Primary Completion Date

February 20, 2018 (Actual)

Study Completion Date

February 20, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vectura Limited

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief 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. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cough, Wheezing

Keywords

Nebulizer, Pediatric, Methodological study

7. Study Design

Primary Purpose

Other

Study Phase

Early Phase 1

Interventional Study Model

Parallel Assignment

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)

Allocation

Non-Randomized

Enrollment

41 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Visit 1 and Visit 2, Test Arm 2A

Arm Type

Other

Arm Description

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

Arm Title

Visit 1 and Visit 2, Test Arm 2B

Arm Type

Other

Arm Description

Subjects who carried out 3 attempts at Visit 1, achieved nebulization success and were subsequently included in Test Arm 2B for Visit 2

Intervention Type

Combination Product

Intervention Name(s)

VR647 Inhalation System + VR647 Smart Card 3 Secs

Intervention Description

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)

Intervention Type

Combination Product

Intervention Name(s)

VR647 Inhalation System + VR647 Smart Card 2 Secs

Intervention Description

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)

Intervention Type

Combination Product

Intervention Name(s)

VR647 Inhalation System + VR647 Smart Card 4 Secs

Intervention Description

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)

Primary Outcome Measure Information:

Title

Number of subjects achieving nebulization success using a mouthpiece overall and by age (years) at each visit.

Description

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.

Time Frame

4 to 8 days

Secondary Outcome Measure Information:

Title

Number of breaths completed by subjects using a mouthpiece overall and by age (years) at each visit.

Description

Number of breaths completed by subjects using a mouthpiece recorded at each visit.

Time Frame

4 to 8 days

Title

Number of subjects aged 3 to <5 years who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 at each visit.

Description

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.

Time Frame

4 to 8 days

Title

Number of subjects who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 overall and by age (years) at each visit.

Description

Number of subjects who require parent(s)/legal guardian(s) hands-on assistance as assessed (Yes/No) by the Investigator on the inhalation checklist.

Time Frame

4 to 8 days

Title

The quality of nebulization at Attempt 3 as recorded in the inhalation checklist overall and by age (years) at each visit.

Description

Time Frame

4 to 8 days

Title

Mean modified Patient Satisfaction and Preference Questionnaire (PASAPQ) total score at Attempt 3 at Visit 2 overall and by age (years).

Description

Time Frame

4 to 8 days

Title

Mean modified PASAPQ satisfaction score at Attempt 3 at Visit 2 overall and by age (years).

Description

Time Frame

4 to 8 days

Title

Mean modified PASAPQ score indicating willingness to continue with the device at Attempt 3 at Visit 2 overall and by age (years).

Description

Time Frame

4 to 8 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Gary Burgess, MD

Organizational Affiliation

Vectura Ltd

Official's Role

Study Director

Facility Information:

Facility Name

Allergy & Asthma Associates of Southern California

City

Mission Viejo

State/Province

California

ZIP/Postal Code

92691

Country

United States

Facility Name

Clinical Research Institute, Inc

City

Plymouth

State/Province

Minnesota

ZIP/Postal Code

55441

Country

United States

Facility Name

St. Louis Children's Hospital

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Facility Name

North Carolina Clinical Research

City

Raleigh

State/Province

North Carolina

ZIP/Postal Code

27607

Country

United States

Facility Name

National Allergy and Asthma Research, LLC

City

Charleston

State/Province

South Carolina

ZIP/Postal Code

29407

Country

United States

12. IPD Sharing Statement

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A Methodology Trial Using the VR647 Inhalation System in Pediatric Subjects Who Have Previously Received Inhaled Therapy

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