Trial Outcomes & Findings for An Open-label Study to Evaluate Correct Use and Ease of Use of the ELLIPTA DPI in Pediatric Subjects With Asthma (NCT NCT03478657)
NCT ID: NCT03478657
Last Updated: 2021-03-26
Results Overview
Participants were trained in the correct use of ELLIPTA DPI by a healthcare professional (HCP) on Day 1 (Visit 1) following which eligible participants were required to take one inhalation of placebo from the ELLIPTA DPI once daily for 28 days. At Day 28 (Visit 2), participants were administered a questionnaire on ease of use of ELLIPTA DPI. Participants and their parents/guardian were required to complete the questionnaire, following which participants were assessed for their ability to demonstrate correct use of ELLIPTA DPI (1 attempt with no training or instruction and then 1 attempt after instruction from parent/guardian). Percentage of participants who demonstrated correct use of ELLIPTA was calculated as "number of participants who demonstrated correct use at Visit 2" divided by "number of participants whose use of the ELLIPTA DPI was evaluated at Visit 2" multiplied by 100. The 95% confidence interval (CI) was calculated using the exact binomial distribution.
COMPLETED
PHASE4
222 participants
At Day 28
2021-03-26
Participant Flow
This was a multi-center, single arm, stratified, open-label, randomized placebo-only study conducted in pediatric participants. Participants were stratified based on age at Visit 0 (Stratum 1: 5 to 7 years old inclusive and Stratum 2: 8 to 11 years old inclusive).
A total of 232 participants were screened, of which 10 failed screening and 222 participants were enrolled in the study. The study was conducted in the United States and Canada.
Participant milestones
| Measure |
Stratum 1: 5-7 Years
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Overall Study
STARTED
|
88
|
134
|
|
Overall Study
COMPLETED
|
88
|
133
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Stratum 1: 5-7 Years
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
An Open-label Study to Evaluate Correct Use and Ease of Use of the ELLIPTA DPI in Pediatric Subjects With Asthma
Baseline characteristics by cohort
| Measure |
Stratum 1: 5-7 Years
n=88 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
n=134 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
Total
n=222 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
6.1 Years
STANDARD_DEVIATION 0.80 • n=5 Participants
|
9.7 Years
STANDARD_DEVIATION 1.10 • n=7 Participants
|
8.3 Years
STANDARD_DEVIATION 2.02 • n=5 Participants
|
|
Sex: Female, Male
Female
|
41 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
89 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
47 Participants
n=5 Participants
|
86 Participants
n=7 Participants
|
133 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian-Central/South Asian Heritage
|
5 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian-Japanese Heritage
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian-South East Asian Heritage
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black Or African American
|
24 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
61 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White-Arabic/North African Heritage
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White-White/Caucasian/European Heritage
|
49 Participants
n=5 Participants
|
75 Participants
n=7 Participants
|
124 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Multiple
|
4 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At Day 28Population: Modified intent to treat (MITT) Population comprised of all participants who were screened, received at least one dose of the study medication (placebo) and were randomized to a version of the ease of use questionnaire at Visit 2.
Participants were trained in the correct use of ELLIPTA DPI by a healthcare professional (HCP) on Day 1 (Visit 1) following which eligible participants were required to take one inhalation of placebo from the ELLIPTA DPI once daily for 28 days. At Day 28 (Visit 2), participants were administered a questionnaire on ease of use of ELLIPTA DPI. Participants and their parents/guardian were required to complete the questionnaire, following which participants were assessed for their ability to demonstrate correct use of ELLIPTA DPI (1 attempt with no training or instruction and then 1 attempt after instruction from parent/guardian). Percentage of participants who demonstrated correct use of ELLIPTA was calculated as "number of participants who demonstrated correct use at Visit 2" divided by "number of participants whose use of the ELLIPTA DPI was evaluated at Visit 2" multiplied by 100. The 95% confidence interval (CI) was calculated using the exact binomial distribution.
Outcome measures
| Measure |
Stratum 1: 5-7 Years
n=88 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
n=133 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Percentage of Participants From Each Stratum Who Demonstrated Correct Use of the ELLIPTA DPI at Visit 2, Attempt 1 (Day 28)
|
92 Percentage of participants
Interval 84.0 to 97.0
|
93 Percentage of participants
Interval 88.0 to 97.0
|
PRIMARY outcome
Timeframe: At Day 28Population: MITT Population. Only those participants who demostrated correct use at Visit 2, attempt 1 has been analyzed.
Participants were trained in the correct use of ELLIPTA DPI at Visit 1 by the HCP and eligible participants were required to take one inhalation of placebo from the ELLIPTA DPI once daily for 28 days. At Day 28 (Visit 2), participants were administered a questionnaire on the ease of use of ELLIPTA DPI. For participants in Stratum 1, questionnaire was provided to the interviewer and responses were recorded. For Stratum 2, the questionnaire was self-administered (or interviewer administered, if participant was unable to self-administer). Percentage of participants who rated the ELLIPTA DPI as easy to use was calculated as "number of participants who rate the ELLIPTA as easy" divided by "number of participants who demonstrated correct use at Visit 2 (Attempt #1)" multiplied by 100. The 95% CI for the percentages are calculated using the exact binomial distribution.
Outcome measures
| Measure |
Stratum 1: 5-7 Years
n=81 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
n=124 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Percentage of Participants Who Rated the ELLIPTA DPI as Easy to Use Among Those Who Could Demonstrate Correct Use at Visit 2, Attempt 1 (Day 28)
|
98 Percentage of participants
Interval 91.0 to 99.0
|
98 Percentage of participants
Interval 93.0 to 99.0
|
SECONDARY outcome
Timeframe: Day 1Population: ITT Population.
Participants were trained by HCP in correct use of ELLIPTA DPI at Visit 1 following which participants were assessed on their ability to use the ELLIPTA DPI without guidance from the HCP or parent/guardian. The participants were given a maximum of five attempts. Participants who were unable to use the ELLIPTA DPI correctly were trained by the HCP for the first three attempts. For the fourth and fifth attempts, the participants' parent/guardian assisted in the training of the ELLIPTA DPI. Percentage number of participants who demonstrated correct use of the ELLIPTA DPI along with 95% CI at Visit 1 (attempts 1 to 5) has been presented. The 95% CI for the percentages are calculated using the exact binomial distribution.
Outcome measures
| Measure |
Stratum 1: 5-7 Years
n=88 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
n=134 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Percentage of Participants From Each Stratum Who Demonstrated Correct Use of the ELLIPTA DPI at Visit 1, Attempt 1 (Day 1)
|
24 Percentage of participants
Interval 15.0 to 34.0
|
50 Percentage of participants
Interval 41.0 to 59.0
|
SECONDARY outcome
Timeframe: At Day 28Population: MITT Population.
Participants were trained in the correct use of ELLIPTA DPI by a HCP on Day 1 (Visit 1) following which eligible participants were required to take one inhalation of placebo from the ELLIPTA DPI once daily for 28 days. At Day 28 (Visit 2), participants were administered a questionnaire on ease of use of ELLIPTA DPI. Participants and their parents/guardian were required to complete the questionnaire, following which participants were assessed for their ability to demonstrate correct use of ELLIPTA DPI (1 attempt with no training or instruction and then 1 attempt after instruction from parent/guardian). Percentage of participants who demonstrated correct use of ELLIPTA was calculated as "number of participants who demonstrated correct use at Visit 2" divided by "number of participants whose use of the ELLIPTA DPI was evaluated at Visit 2" multiplied by 100. The 95% CI was calculated using the exact binomial distribution.
Outcome measures
| Measure |
Stratum 1: 5-7 Years
n=221 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Percentage of Participants Who Demonstrated Correct Use of the ELLIPTA DPI at Visit 2, Attempt 1 in All Participants (Stratum 1+Stratum 2)
|
93 Percentage of participants
Interval 89.0 to 96.0
|
—
|
SECONDARY outcome
Timeframe: At Day 28Population: MITT Population. Only those participants who demonstrated correct use at Visit 2, attempt 1 were analyzed.
Participants were trained in the correct use of ELLIPTA DPI at Visit 1 by the HCP and eligible participants were required to take one inhalation of placebo from the ELLIPTA DPI once daily for 28 days. At Day 28 (Visit 2), participants were administered a questionnaire on the ease of use of ELLIPTA DPI. For participants in Stratum 1, questionnaire was provided to the interviewer and responses were recorded. For Stratum 2, the questionnaire was self-administered (or interviewer administered, if participant was unable to self-administer). Percentage of participants who rated the ELLIPTA DPI as easy to use was calculated as "number of participants who rate the ELLIPTA as easy" divided by "number of participants who demonstrated correct use at Visit 2 (Attempt #1)" multiplied by 100. The 95% CI for the percentages are calculated using the exact binomial distribution.
Outcome measures
| Measure |
Stratum 1: 5-7 Years
n=205 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Percentage of Participants Who Rated Easy Use of ELLIPTA DPI at Visit 2 in All Participants (Stratum 1+Stratum 2)
|
98 Percentage of participants
Interval 94.0 to 99.0
|
—
|
SECONDARY outcome
Timeframe: Day 1Population: ITT Population.
Participants were trained by HCP in correct use of ELLIPTA DPI at Visit 1 following which participants were assessed on their ability to use the ELLIPTA DPI without guidance from the HCP or parent/guardian. The participants were given a maximum of five attempts. Participants who were unable to use the ELLIPTA DPI correctly were trained by the HCP for the first three attempts. For the fourth and fifth attempts, the participants' parent/guardian assisted in the training of the ELLIPTA DPI. Percentage of participants who has withdrawn at any point throughout the correct use demonstration and performed a critical error, was considered as one who performed at least one critical error. Participant who has withdrawn at any point throughout correct use demonstration and did not perform all the critical error items were also considered as one who performed at least one critical error. The 95% CI for the percentages are calculated using the exact binomial distribution.
Outcome measures
| Measure |
Stratum 1: 5-7 Years
n=88 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA dry powder inhaler (DPI) for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI.
|
Stratum 2: 8-11 Years
n=134 Participants
Participants received an oral inhalation of placebo once daily via ELLIPTA DPI for 28 days. At Day 28 (Visit 2), participants received a questionnaire on ease of use of ELLIPTA DPI. Participants were asked questions on ease of use and asked to perform correct use of the ELLIPTA DPI
|
|---|---|---|
|
Percentage of Participants Who Reported at Least One Critical Error From Each Stratum With the Use of ELLIPTA DPI at Visit 1, Attempt 1
|
51 Percentage of participants
Interval 40.0 to 62.0
|
25 Percentage of participants
Interval 18.0 to 34.0
|
Adverse Events
Stratum 1 + Stratum 2
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Stratum 1 + Stratum 2
n=222 participants at risk
All participants from Stratum 1 and Stratum 2 (5 to 11 years) were included.
|
|---|---|
|
Infections and infestations
Viral upper respiratory tract infection
|
2.3%
5/222 • Number of events 5 • Serious adverse events (SAEs) and non-SAEs were collected up to Day 28
SAEs and non-SAEs were summarized for the ITT Population. Since all participants received placebo as treatment, data has been combined for all the arms.
|
|
Infections and infestations
Upper respiratory tract infection
|
1.4%
3/222 • Number of events 3 • Serious adverse events (SAEs) and non-SAEs were collected up to Day 28
SAEs and non-SAEs were summarized for the ITT Population. Since all participants received placebo as treatment, data has been combined for all the arms.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.4%
3/222 • Number of events 3 • Serious adverse events (SAEs) and non-SAEs were collected up to Day 28
SAEs and non-SAEs were summarized for the ITT Population. Since all participants received placebo as treatment, data has been combined for all the arms.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee GSK agreements may vary with individual investigators, but will not prohibit any investigator from publishing. GSK supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial.
- Publication restrictions are in place
Restriction type: OTHER