Clinical trial NCT04011709: 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
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Cough
Wheezing
Keywords: Nebulizer
Pediatric
Methodological study
Lead sponsor: Vectura Limited
Sponsor class: INDUSTRY
First posted: 2019-07-08
Enrollment: 41
Sex: ALL
Minimum age (years): 1
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention 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.
Primary purpose: OTHER

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.

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

Primary outcomes:
measure: Number of subjects achieving nebulization success using a mouthpiece overall and by age (years) at each visit.
timeFrame: 4 to 8 days
Secondary outcomes:
measure: Number of breaths completed by subjects using a mouthpiece overall and by age (years) at each visit.
timeFrame: 4 to 8 days
measure: Number of subjects aged 3 to <5 years who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 at each visit.
timeFrame: 4 to 8 days
measure: Number of subjects who require parent(s)/legal guardian(s) hands-on assistance at Attempt 3 overall and by age (years) at each visit.
timeFrame: 4 to 8 days
measure: The quality of nebulization at Attempt 3 as recorded in the inhalation checklist overall and by age (years) at each visit.
timeFrame: 4 to 8 days
measure: Mean modified Patient Satisfaction and Preference Questionnaire (PASAPQ) total score at Attempt 3 at Visit 2 overall and by age (years).
timeFrame: 4 to 8 days
measure: Mean modified PASAPQ satisfaction score at Attempt 3 at Visit 2 overall and by age (years).
timeFrame: 4 to 8 days
measure: Mean modified PASAPQ score indicating willingness to continue with the device at Attempt 3 at Visit 2 overall and by age (years).
timeFrame: 4 to 8 days
Other outcomes:
Locations:
city: Mission Viejo
country: United States
facility: Allergy & Asthma Associates of Southern California
state: California
city: Plymouth
country: United States
facility: Clinical Research Institute, Inc
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Raleigh
country: United States
facility: North Carolina Clinical Research
state: North Carolina
city: Charleston
country: United States
facility: National Allergy and Asthma Research, LLC
state: South Carolina
Study officials:
affiliation: Vectura Ltd
name: Gary Burgess, MD
role: STUDY_DIRECTOR
References: