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

Early Phase 1CompletedNCT04011709

Vectura Limited41 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

OTHER

Masking

NONE

Enrollment

Eligibility

Sex: ALLMin age: 1 YearMax age: 5 Years

Medical Language ↔ Plain English

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

Locations (5)

Allergy & Asthma Associates of Southern California

Mission Viejo, California, United States

Clinical Research Institute, Inc

Plymouth, Minnesota, United States

St. Louis Children's Hospital

St Louis, Missouri, United States

North Carolina Clinical Research

Raleigh, North Carolina, United States

National Allergy and Asthma Research, LLC

Charleston, South Carolina, United States

Outcomes

Primary Outcomes

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.

Time frame: 4 to 8 days

Secondary Outcomes

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.

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.

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

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.

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.

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.

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).

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).

Time frame: 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).

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).

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).

Time frame: 4 to 8 days

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

Overview

Conditions

Interventions

Eligibility

Locations (5)

Outcomes

Primary Outcomes

Secondary Outcomes