Trial Outcomes & Findings for A Study to Assess the Efficacy and Safety of Budesonide/Albuterol Metered-dose Inhaler (BDA MDI/PT027) in Adults and Children 4 Years of Age or Older With Asthma (NCT NCT03769090)

Last Updated: 2022-09-28

Results Overview

Time to first severe asthma exacerbation will be calculated as the time from randomization until the start date of the first severe asthma exacerbation. An asthma exacerbation will be considered severe if it results in at least one of the following: a temporary bolus/burst of systemic corticosteroids for at least 3 consecutive days to treat symptoms of asthma worsening (a single depo-injectable dose of corticosteroids will be considered equivalent), an emergency room or urgent care visit (\<24 hours in the facility for evaluation and treatment) due to asthma that required systemic corticosteroids, or an in-patient hospitalization (admission to an in-patient facility and/or ≥ 24 hours in a healthcare facility) due to asthma. The descriptive summary shows the number of participants with a severe exacerbation event, occurring between the date of randomization up to the date of randomized treatment discontinuation or a change in maintenance therapy.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

3132 participants

Primary outcome timeframe

From randomization up to a discontinuation of randomized treatment or a change in maintenance therapy. The mean and median reporting period for all participants was 44 and 48 weeks, respectively.

Results posted on

2022-09-28

Participant Flow

The first subject enrolled on 13 December 2018 and the last subject completed the study on 07 February 2022. Subjects were enrolled at 347 study centers worldwide (Argentina, Canada, Czechia, Germany, Serbia, Slovakia, South Africa, Spain, Ukraine, United Kingdom and the United States).

The randomized treatment phase started after a 2 to 4 week screening period during which participants used sponsor provided Ventolin HFA (albuterol sulfate) as needed. In addition to the 3,132 participants randomized, 2,488 participants were screened but did not participate, of which 2,456 were ineligible (98.7%). The next most frequent reason was withdrawal by the participant, with 18 meeting this criteria.

Participant milestones

Participant milestones
Measure	BDA MDI (PT027) 160/180 μg Budesonide/albuterol sulfate, BDA MDI, PT027 high dose Budesonide/albuterol sulfate metered-dose inhaler 160/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	BDA MDI (PT027) 80/180 μg Budesonide/albuterol sulfate, BDA MDI, PT027 low dose Budesonide/albuterol sulfate metered-dose inhaler 80/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	AS MDI (PT007) 180 µg Albuterol sulfate MDI, PT007 Albuterol sulfate metered-dose inhaler 180 μg: Albuterol sulfate inhalation aerosol
Overall Study STARTED	1016	1057	1059
Overall Study COMPLETED	915	933	916
Overall Study NOT COMPLETED	101	124	143

Reasons for withdrawal

Reasons for withdrawal
Measure	BDA MDI (PT027) 160/180 μg Budesonide/albuterol sulfate, BDA MDI, PT027 high dose Budesonide/albuterol sulfate metered-dose inhaler 160/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	BDA MDI (PT027) 80/180 μg Budesonide/albuterol sulfate, BDA MDI, PT027 low dose Budesonide/albuterol sulfate metered-dose inhaler 80/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	AS MDI (PT007) 180 µg Albuterol sulfate MDI, PT007 Albuterol sulfate metered-dose inhaler 180 μg: Albuterol sulfate inhalation aerosol
Overall Study Randomized and did not receive randomized study treatment	1	2	2
Overall Study Withdrawal by Subject	52	62	74
Overall Study Death	4	2	2
Overall Study Adverse Event	7	7	8
Overall Study A single severe exacerbation event lasting >20 days	0	1	2
Overall Study >= 3 severe exacerbation events within a 3 month period	1	4	4
Overall Study >= 5 total severe exacerbation events	0	1	1
Overall Study Protocol Violation	5	6	8
Overall Study Pregnancy	1	0	3
Overall Study Lost to Follow-up	19	25	21
Overall Study Condition under investigation worsened	2	0	1
Overall Study Lack of Efficacy	1	2	2
Overall Study Other reasons	8	12	15

Baseline Characteristics

A Study to Assess the Efficacy and Safety of Budesonide/Albuterol Metered-dose Inhaler (BDA MDI/PT027) in Adults and Children 4 Years of Age or Older With Asthma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BDA MDI (PT027) 160/180 μg n=1015 Participants Budesonide/albuterol sulfate, BDA MDI, PT027 high dose Budesonide/albuterol sulfate metered-dose inhaler 160/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	BDA MDI (PT027) 80/180 μg n=1055 Participants Budesonide/albuterol sulfate, BDA MDI, PT027 low dose Budesonide/albuterol sulfate metered-dose inhaler 80/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	AS MDI (PT007) 180 µg n=1057 Participants Albuterol sulfate MDI, PT007 Albuterol sulfate metered-dose inhaler 180 μg: Albuterol sulfate inhalation aerosol	Total n=3127 Participants Total of all reporting groups
Age, Continuous	50.6 years STANDARD_DEVIATION 15.05 • n=5 Participants	48.5 years STANDARD_DEVIATION 16.71 • n=7 Participants	49.1 years STANDARD_DEVIATION 17.22 • n=5 Participants	49.4 years STANDARD_DEVIATION 16.39 • n=4 Participants
Age, Customized Children (>=4 to <12 years)	0 Participants n=5 Participants	41 Participants n=7 Participants	42 Participants n=5 Participants	83 Participants n=4 Participants
Age, Customized Adolescents (>=12 to <18 years)	34 Participants n=5 Participants	32 Participants n=7 Participants	34 Participants n=5 Participants	100 Participants n=4 Participants
Age, Customized Adults (>=18 to <65 years)	789 Participants n=5 Participants	805 Participants n=7 Participants	784 Participants n=5 Participants	2378 Participants n=4 Participants
Age, Customized Elderly (>=65 years)	192 Participants n=5 Participants	177 Participants n=7 Participants	197 Participants n=5 Participants	566 Participants n=4 Participants
Sex: Female, Male Female	647 Participants n=5 Participants	686 Participants n=7 Participants	695 Participants n=5 Participants	2028 Participants n=4 Participants
Sex: Female, Male Male	368 Participants n=5 Participants	369 Participants n=7 Participants	362 Participants n=5 Participants	1099 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	235 Participants n=5 Participants	261 Participants n=7 Participants	316 Participants n=5 Participants	812 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	780 Participants n=5 Participants	794 Participants n=7 Participants	741 Participants n=5 Participants	2315 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Asian	29 Participants n=5 Participants	33 Participants n=7 Participants	23 Participants n=5 Participants	85 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	139 Participants n=5 Participants	141 Participants n=7 Participants	137 Participants n=5 Participants	417 Participants n=4 Participants
Race (NIH/OMB) White	820 Participants n=5 Participants	848 Participants n=7 Participants	869 Participants n=5 Participants	2537 Participants n=4 Participants
Race (NIH/OMB) More than one race	18 Participants n=5 Participants	20 Participants n=7 Participants	24 Participants n=5 Participants	62 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	8 Participants n=5 Participants	12 Participants n=7 Participants	4 Participants n=5 Participants	24 Participants n=4 Participants
Region of Enrollment Canada	19 Participants n=5 Participants	11 Participants n=7 Participants	12 Participants n=5 Participants	42 Participants n=4 Participants
Region of Enrollment Argentina	157 Participants n=5 Participants	200 Participants n=7 Participants	207 Participants n=5 Participants	564 Participants n=4 Participants
Region of Enrollment United States	274 Participants n=5 Participants	275 Participants n=7 Participants	290 Participants n=5 Participants	839 Participants n=4 Participants
Region of Enrollment Czechia	74 Participants n=5 Participants	64 Participants n=7 Participants	73 Participants n=5 Participants	211 Participants n=4 Participants
Region of Enrollment Ukraine	156 Participants n=5 Participants	150 Participants n=7 Participants	128 Participants n=5 Participants	434 Participants n=4 Participants
Region of Enrollment South Africa	106 Participants n=5 Participants	120 Participants n=7 Participants	115 Participants n=5 Participants	341 Participants n=4 Participants
Region of Enrollment United Kingdom	9 Participants n=5 Participants	15 Participants n=7 Participants	10 Participants n=5 Participants	34 Participants n=4 Participants
Region of Enrollment Slovakia	25 Participants n=5 Participants	30 Participants n=7 Participants	28 Participants n=5 Participants	83 Participants n=4 Participants
Region of Enrollment Serbia	65 Participants n=5 Participants	54 Participants n=7 Participants	57 Participants n=5 Participants	176 Participants n=4 Participants
Region of Enrollment Germany	109 Participants n=5 Participants	108 Participants n=7 Participants	112 Participants n=5 Participants	329 Participants n=4 Participants
Region of Enrollment Spain	21 Participants n=5 Participants	28 Participants n=7 Participants	25 Participants n=5 Participants	74 Participants n=4 Participants
Height	166.8 Centimeters STANDARD_DEVIATION 9.92 • n=5 Participants	165.1 Centimeters STANDARD_DEVIATION 11.45 • n=7 Participants	164.4 Centimeters STANDARD_DEVIATION 11.67 • n=5 Participants	165.4 Centimeters STANDARD_DEVIATION 11.10 • n=4 Participants
Weight	80.5 Kilograms STANDARD_DEVIATION 16.88 • n=5 Participants	78.2 Kilograms STANDARD_DEVIATION 18.93 • n=7 Participants	78.8 Kilograms STANDARD_DEVIATION 18.47 • n=5 Participants	79.2 Kilograms STANDARD_DEVIATION 18.15 • n=4 Participants
Body mass index	28.887 kg/m^2 STANDARD_DEVIATION 5.4002 • n=5 Participants	28.496 kg/m^2 STANDARD_DEVIATION 5.5886 • n=7 Participants	28.948 kg/m^2 STANDARD_DEVIATION 5.6271 • n=5 Participants	28.776 kg/m^2 STANDARD_DEVIATION 5.5432 • n=4 Participants

PRIMARY outcome

Timeframe: From randomization up to a discontinuation of randomized treatment or a change in maintenance therapy. The mean and median reporting period for all participants was 44 and 48 weeks, respectively.

Population: All participants that were randomized, took at least one puff of randomized treatment, and had at least one efficacy assessment, excluding participants confirmed as duplicates (n = 4). As children \<12 years of age were not randomized to the BDA MDI 160/180μg dose, comparison was made to the participants \>=12 years of age within the AS MDI 180μg arm.

Outcome measures

Outcome measures
Measure	BDA MDI 160/180 μg (Full Analysis Set; >=12 Years) n=1013 Participants All participants who are randomized to BDA MDI 160/180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set; >=12 Years) n=1014 Participants All participants who are randomized to AS MDI 180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	BDA MDI 80/180 μg (Full Analysis Set) n=1054 Participants All participants who are randomized to BDA MDI 80/180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set) n=1056 Participants All participants who are randomized to AS MDI 180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.
Number of Participants With a Severe Asthma Exacerbation Event	207 Participants	266 Participants	241 Participants	276 Participants

SECONDARY outcome

Timeframe: From randomization up to discontinuation of randomized treatment or a change in maintenance therapy. The mean and median reporting period for all participants was 44 and 48 weeks, respectively.

The annualized severe exacerbation rate (severe exacerbations per year) is estimated from a negative binomial model with treatment, age group, region, and number of severe exacerbations in the last 12 months prior to randomization as categorical covariates. The logarithm of the time at risk is included as an offset variable.

Outcome measures

Outcome measures
Measure	BDA MDI 160/180 μg (Full Analysis Set; >=12 Years) n=1013 Participants All participants who are randomized to BDA MDI 160/180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set; >=12 Years) n=1014 Participants All participants who are randomized to AS MDI 180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	BDA MDI 80/180 μg (Full Analysis Set) n=1054 Participants All participants who are randomized to BDA MDI 80/180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set) n=1056 Participants All participants who are randomized to AS MDI 180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.
Annualized Severe Exacerbation Rate	0.45 Severe exacerbations per year Interval 0.34 to 0.6	0.59 Severe exacerbations per year Interval 0.44 to 0.78	0.49 Severe exacerbations per year Interval 0.37 to 0.64	0.61 Severe exacerbations per year Interval 0.46 to 0.8

SECONDARY outcome

This endpoint includes all systemic corticosteroids (SCS) taken in response to a severe exacerbation event from randomization up to randomized treatment discontinuation or a change in maintenance therapy. All SCS are standardized to equipotent doses of prednisone before deriving the total dose. The total annualized dose is calculated as the total dose of SCS divided by the duration of the randomized treatment period.

Outcome measures

Outcome measures
Measure	BDA MDI 160/180 μg (Full Analysis Set; >=12 Years) n=1012 Participants All participants who are randomized to BDA MDI 160/180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set; >=12 Years) n=1011 Participants All participants who are randomized to AS MDI 180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	BDA MDI 80/180 μg (Full Analysis Set) n=1052 Participants All participants who are randomized to BDA MDI 80/180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set) n=1052 Participants All participants who are randomized to AS MDI 180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.
Total Annualized Dose of Systemic Corticosteroid (SCS)	86.2 Milligram(s) Interval 0.0 to 3678.0	129.3 Milligram(s) Interval 0.0 to 18941.0	95.5 Milligram(s) Interval 0.0 to 4974.0	127.1 Milligram(s) Interval 0.0 to 18941.0

SECONDARY outcome

Timeframe: From baseline to Week 24

The ACQ-5 consists of 5 questions on symptom control, with each scored on a 7-point scale (0 = excellent asthma control; 6 = extremely poor control). The overall score (0 = excellent asthma control; 6 = extremely poor control, so a lower score is the better outcome) is the mean of the 5 symptom items. A responder is defined as a participant with a decrease from baseline to Week 24 overall ACQ-5 score of 0.5 or more. ACQ-5 is not validated for children less than 6 years old, data for participants who were 4 or 5 years old was excluded from the analysis of ACQ-5.

Outcome measures

Outcome measures
Measure	BDA MDI 160/180 μg (Full Analysis Set; >=12 Years) n=1013 Participants All participants who are randomized to BDA MDI 160/180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set; >=12 Years) n=1014 Participants All participants who are randomized to AS MDI 180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	BDA MDI 80/180 μg (Full Analysis Set) n=1052 Participants All participants who are randomized to BDA MDI 80/180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set) n=1055 Participants All participants who are randomized to AS MDI 180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.
Asthma Control Questionnaire-5 (ACQ-5) - Number of Participants Who Were Responders at Week 24	677 Participants	630 Participants	681 Participants	650 Participants

SECONDARY outcome

Timeframe: From baseline to 24 weeks

The AQLQ+12 consists of 32 questions in 4 domains and is assessed on separate 7-point Likert scales from 1 to 7, with higher values indicating better health-related quality of life. The overall score is the mean of all responses. A responder is defined as a participant with an increase from baseline to week 24 AQLQ-12 score of at least 0.5.

Outcome measures

Outcome measures
Measure	BDA MDI 160/180 μg (Full Analysis Set; >=12 Years) n=994 Participants All participants who are randomized to BDA MDI 160/180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set; >=12 Years) n=987 Participants All participants who are randomized to AS MDI 180 μg, are aged \>=12 years at randomization, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	BDA MDI 80/180 μg (Full Analysis Set) n=993 Participants All participants who are randomized to BDA MDI 80/180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.	AS MDI 180 μg (Full Analysis Set) All participants who are randomized to AS MDI 180 μg, take at least one puff of randomized treatment and have at least one efficacy assessment, excluding participants identified as confirmed duplicates.
Asthma Quality of Life Questionnaire for Participants Aged 12 Years and Older (AQLQ+12) - Number of Participants Who Were Responders at Week 24	508 Participants	489 Participants	461 Participants	—

Adverse Events

BDA MDI (PT027) 160/180 μg

Serious events: 53 serious events

Other events: 244 other events

Deaths: 4 deaths

BDA MDI (PT027) 80/180 μg

Serious events: 40 serious events

Other events: 253 other events

Deaths: 2 deaths

AS MDI (PT007) 180 µg

Serious events: 48 serious events

Other events: 243 other events

Deaths: 2 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	BDA MDI (PT027) 160/180 μg n=1015 participants at risk Budesonide/albuterol sulfate, BDA MDI, PT027 high dose Budesonide/albuterol sulfate metered-dose inhaler 160/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	BDA MDI (PT027) 80/180 μg n=1055 participants at risk Budesonide/albuterol sulfate, BDA MDI, PT027 low dose Budesonide/albuterol sulfate metered-dose inhaler 80/180 μg: Budesonide/albuterol sulfate combination inhalation aerosol	AS MDI (PT007) 180 µg n=1057 participants at risk Albuterol sulfate MDI, PT007 Albuterol sulfate metered-dose inhaler 180 μg: Albuterol sulfate inhalation aerosol
Vascular disorders Hypertension	2.2% 22/1015 • Number of events 23 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.6% 27/1055 • Number of events 27 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.5% 26/1057 • Number of events 27 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Nervous system disorders Headache	4.3% 44/1015 • Number of events 71 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	4.7% 50/1055 • Number of events 77 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	4.6% 49/1057 • Number of events 76 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Musculoskeletal and connective tissue disorders Back pain	2.7% 27/1015 • Number of events 29 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.2% 23/1055 • Number of events 23 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	1.9% 20/1057 • Number of events 20 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Infections and infestations Nasopharyngitis	7.5% 76/1015 • Number of events 95 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	5.8% 61/1055 • Number of events 68 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	5.1% 54/1057 • Number of events 67 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Infections and infestations COVID-19	3.3% 33/1015 • Number of events 33 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	4.5% 47/1055 • Number of events 47 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	3.6% 38/1057 • Number of events 38 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Infections and infestations Upper respiratory tract infection	2.6% 26/1015 • Number of events 31 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.9% 31/1055 • Number of events 35 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.5% 26/1057 • Number of events 28 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Infections and infestations Bronchitis	2.5% 25/1015 • Number of events 27 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.6% 27/1055 • Number of events 30 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.6% 28/1057 • Number of events 32 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Infections and infestations Sinusitis	1.5% 15/1015 • Number of events 16 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	1.6% 17/1055 • Number of events 17 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	2.4% 25/1057 • Number of events 28 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.
Infections and infestations Influenza	2.1% 21/1015 • Number of events 23 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	1.9% 20/1055 • Number of events 22 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.	1.3% 14/1057 • Number of events 16 • Adverse events (AEs) were collected from the time of signed informed consent/assent and up to the safety follow up period. Reported AEs are those that occurred from the date of first dose of randomized treatment up to the date of treatment discontinuation, which per protocol, was expected to be at least 24 weeks. The mean and median duration of exposure to randomized treatment for the reporting period for all participants was 44 and 48 weeks, respectively.

Additional Information

Chief Medical Officer

Avillion LLP

Phone: +44 (0)203 764 9530

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Data or results obtained from this study must not be published without prior approval from the Sponsor.
Publication restrictions are in place

Restriction type: OTHER