Trial Outcomes & Findings for A Study to Evaluate Mirabegron in Pediatric Participants From 5 to Less Than 18 Years of Age With Overactive Bladder (OAB) (NCT NCT04641975)

Results Overview

A micturition was defined as any voluntary act of passing urine (excluding incontinence only episodes). The mean number of micturitions per 24 hours was calculated as the average number of times a participant urinated per day during the 7-day micturition diary period. The analysis was performed with imputation of missing visit 7/week 12 data using the last observation carried forward (LOCF) method.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

26 participants

Primary outcome timeframe

Baseline, week 12

Results posted on

2024-11-14

Participant Flow

Participants who had received 4 weeks of urotherapy prior to randomization were enrolled in the study.

Standard urotherapy included information on and demystification of voiding function and dysfunction, instruction on voiding habits, lifestyle advice regarding fluid intake, prevention of constipation, recording of symptoms and voiding habits in bladder diaries and support via regular follow-up. Specific interventions included various forms of pelvic floor training, behavioral modification, electrical stimulation, catherization and biofeedback and elements of cognitive behavioral therapy.

Participant milestones

Participant milestones
Measure	Mirabegron (5 to < 12 Years) Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (5 to < 12 Years) Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Overall Study STARTED	11	12	2	1
Overall Study COMPLETED	9	10	1	0
Overall Study NOT COMPLETED	2	2	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Mirabegron (5 to < 12 Years) Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (5 to < 12 Years) Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Overall Study Protocol Violation	1	2	0	0
Overall Study Adverse Event	0	0	1	1
Overall Study Withdrawal by Subject	1	0	0	0

Baseline Characteristics

Participants in the SAF with available data were analyzed.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Mirabegron (5 to < 12 Years) n=11 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (5 to < 12 Years) n=12 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) n=2 Participants Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) n=1 Participants Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Total n=26 Participants Total of all reporting groups
Age, Continuous	8.4 Years STANDARD_DEVIATION 1.9 • n=11 Participants	7.7 Years STANDARD_DEVIATION 1.7 • n=12 Participants	16 Years STANDARD_DEVIATION 1.4 • n=2 Participants	12 Years STANDARD_DEVIATION NA • n=1 Participants	8.8 Years STANDARD_DEVIATION 2.8 • n=26 Participants
Sex: Female, Male Female	2 Participants n=11 Participants	7 Participants n=12 Participants	2 Participants n=2 Participants	0 Participants n=1 Participants	11 Participants n=26 Participants
Sex: Female, Male Male	9 Participants n=11 Participants	5 Participants n=12 Participants	0 Participants n=2 Participants	1 Participants n=1 Participants	15 Participants n=26 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=11 Participants	1 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	1 Participants n=26 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=11 Participants	11 Participants n=12 Participants	2 Participants n=2 Participants	1 Participants n=1 Participants	23 Participants n=26 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=11 Participants	0 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	2 Participants n=26 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=11 Participants	0 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=26 Participants
Race (NIH/OMB) Asian	2 Participants n=11 Participants	6 Participants n=12 Participants	1 Participants n=2 Participants	1 Participants n=1 Participants	10 Participants n=26 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=11 Participants	0 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=26 Participants
Race (NIH/OMB) Black or African American	0 Participants n=11 Participants	0 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=26 Participants
Race (NIH/OMB) White	7 Participants n=11 Participants	6 Participants n=12 Participants	1 Participants n=2 Participants	0 Participants n=1 Participants	14 Participants n=26 Participants
Race (NIH/OMB) More than one race	0 Participants n=11 Participants	0 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=26 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=11 Participants	0 Participants n=12 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	2 Participants n=26 Participants
Mean Number of Micturitions per 24 hour	9.48 Micturitions per 24 hours STANDARD_DEVIATION 4.53 • n=11 Participants • Participants in the SAF with available data were analyzed.	9.72 Micturitions per 24 hours STANDARD_DEVIATION 4.95 • n=11 Participants • Participants in the SAF with available data were analyzed.	19.43 Micturitions per 24 hours STANDARD_DEVIATION 3.16 • n=2 Participants • Participants in the SAF with available data were analyzed.	9.29 Micturitions per 24 hours n=1 Participants • Participants in the SAF with available data were analyzed.	10.37 Micturitions per 24 hours STANDARD_DEVIATION 5.16 • n=25 Participants • Participants in the SAF with available data were analyzed.

PRIMARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis set: All participants who were randomized and received at least 1 dose of study drug and had at least 1 post baseline measurement for mean number of micturitions per 24 hours.

A micturition was defined as any voluntary act of passing urine (excluding incontinence only episodes). The mean number of micturitions per 24 hours was calculated as the average number of times a participant urinated per day during the 7-day micturition diary period. The analysis was performed with imputation of missing visit 7/week 12 data using the last observation carried forward (LOCF) method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=10 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=9 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Mean Number of Micturitions Per 24 Hours for Age Group 5 to <12 Years	-3.84 micturitions per 24 hours Standard Error 0.89	—	—	-1.62 micturitions per 24 hours Standard Error 0.89

SECONDARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis Set with available data was analyzed.

Mean volume voided was derived from "Pee Volume" of the 2-day Weekend Episodic Diary. Mean volume voided per day was calculated as the sum of the volumes voided on that (valid diary) day divided by the number of times a volume was recorded on that day in the 2-day Weekend Episodic Diary. The analysis was performed with LOCF and without LOCF method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=8 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=8 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Mean Volume Voided Per 24 Hours for Age Group 5 to <12 Years Week 12 (without LOCF)	24.55 milliliter (mL)/24 hours Standard Deviation 32.32	—	—	18.38 milliliter (mL)/24 hours Standard Deviation 33.67
Change From Baseline to Week 12/EoT in Mean Volume Voided Per 24 Hours for Age Group 5 to <12 Years Week 12 (with LOCF)	24.55 milliliter (mL)/24 hours Standard Deviation 32.32	—	—	18.38 milliliter (mL)/24 hours Standard Deviation 33.67

SECONDARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis Set with available data was analyzed.

MVV data was derived from "Pee Volume" of the 2-day Weekend Episodic Diary. The MVV was the largest (non-zero) volume recorded over both of the 2 (valid) measuring days in the diary. The analysis was performed with LOCF and without LOCF method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=8 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=8 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Maximum Volume Voided (MVV) for Age Group 5 to <12 Years Week 12 (without LOCF)	26.38 mL Standard Deviation 60.75	—	—	26.00 mL Standard Deviation 51.46
Change From Baseline to Week 12/EoT in Maximum Volume Voided (MVV) for Age Group 5 to <12 Years Week 12 (With LOCF)	26.38 mL Standard Deviation 60.75	—	—	26.00 mL Standard Deviation 51.46

SECONDARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis Set with available data was analyzed.

A daytime incontinence episode was defined as the complaint of any involuntary leakage of urine during daytime hours. Daytime was defined as time between waking up in the morning and going to sleep later the same day or next day. The mean number of daytime incontinence episodes per 24 hours was calculated by taking the sum of all daytime urinary incontinence episodes recorded in the participant diary, divided by the number of valid diary days. The analysis was performed with LOCF and without LOCF method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=9 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=8 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Mean Number of Daytime Incontinence Episodes Per 24 Hours for Age Group 5 to <12 Years Week 12 (without LOCF)	-1.28 incontinence episodes per 24 hours Standard Deviation 1.73	—	—	-1.29 incontinence episodes per 24 hours Standard Deviation 1.04
Change From Baseline to Week 12/EoT in Mean Number of Daytime Incontinence Episodes Per 24 Hours for Age Group 5 to <12 Years Week 12 (with LOCF)	-1.09 incontinence episodes per 24 hours Standard Deviation 1.74	—	—	-1.20 incontinence episodes per 24 hours Standard Deviation 1.02

SECONDARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis Set.

A nighttime incontinence episode was defined as the complaint of any involuntary leakage of urine during nighttime hours. Nightime was defined as time between between going to sleep on a day and waking up on the same or next day. The mean number of nighttime incontinence episodes per 24 hours was calculated by taking the sum of all nighttime urinary incontinence episodes recorded in the participant diary, divided by the number of valid diary days. The analysis was performed with LOCF and without LOCF method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=10 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=9 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Mean Number of Nighttime Incontinence Episodes Per 24 Hours for Age Group 5 to <12 Years Week 12 (without LOCF)	-1.34 incontinence episodes per 24 hours Standard Deviation 1.51	—	—	-0.64 incontinence episodes per 24 hours Standard Deviation 0.55
Change From Baseline to Week 12/EoT in Mean Number of Nighttime Incontinence Episodes Per 24 Hours for Age Group 5 to <12 Years Week 12 (with LOCF)	-1.34 incontinence episodes per 24 hours Standard Deviation 1.51	—	—	-0.64 incontinence episodes per 24 hours Standard Deviation 0.55

SECONDARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis Set

For a week day the daytime micturitions was derived from "Number of Times using the Toilet During the Day" was entered into the 5-day Week Diary. For a weekend day the daytime micturitions was derived from the number of times a "Pee in Toilet" or a "Pee in Toilet and Leakage" was entered into the 2-day Weekend Episodic Diary between the time the participant woke-up (exclusive). The total number of micturitions per weekend day was equal to the total number of times, in the diary, an amount of pee was recorded during daytime for that day. For each participant, the mean number of daytime micturitions was calculated as: Sum of the Number of Daytime Micturitions (per day) over the Valid Diary Days prior to Visit/Number of Valid Diary Days. The analysis was performed with LOCF and without LOCF method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=10 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=9 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Mean Number of Daytime Micturitions Per 24 Hours for Age Group 5 to <12 Years Week 12 (without LOCF)	-3.21 micturitions per 24 hours Standard Deviation 6.65	—	—	-0.85 micturitions per 24 hours Standard Deviation 2.67
Change From Baseline to Week 12/EoT in Mean Number of Daytime Micturitions Per 24 Hours for Age Group 5 to <12 Years Week 12 (with LOCF)	-3.21 micturitions per 24 hours Standard Deviation 6.27	—	—	-0.85 micturitions per 24 hours Standard Deviation 2.67

SECONDARY outcome

Timeframe: Baseline, week 12

Population: Full Analysis Set with available data was analyzed.

A dry (incontinence free) day was defined as a day where the response is "Dry" to the question "How was your Day" and to "How was your Night". For a weekend day a "Dry (incontinence free) Day" was defined a day where no "New pee or leakage" was reported. Let Ddry be the number of valid diary days where the response to both questions was "Dry". Let Dwet be the number of valid diary days where the response to one of the two questions or to both questions was "Wet". If (Ddry + Dwet) \> 3, the number of dry days per 7 days was calculated as Ddry/(Ddry + Dwet)\* 7, otherwise the value was missing. The analysis was performed with LOCF and without LOCF method.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=9 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=9 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline to Week 12/EoT in Number of Dry (Incontinence-free) Days Per 7 Days for Age Group 5 to <12 Years Week 12 (without LOCF)	1.45 incontinence-free days Standard Deviation 2.33	—	—	3.12 incontinence-free days Standard Deviation 3.36
Change From Baseline to Week 12/EoT in Number of Dry (Incontinence-free) Days Per 7 Days for Age Group 5 to <12 Years Week 12 (with LOCF)	1.46 incontinence-free days Standard Deviation 2.18	—	—	2.94 incontinence-free days Standard Deviation 3.47

SECONDARY outcome

Timeframe: From first dose up to week 14

Population: SAF

An AE was any untoward medical occurrence in a participant administered a study drug and which did not necessarily have to have a causal relationship with this treatment. An AE could therefore, be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug whether or not considered related to the study drug. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted a congenital anomaly/birth defect or other medically important event. A TEAE was defined as an AE observed after starting administration of the study drug until 30 days after last dose.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=12 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) n=2 Participants Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) n=1 Participants Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=11 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs Treatment emergent adverse events	7 Participants	1 Participants	1 Participants	5 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs Serious treatment emergent adverse events	0 Participants	1 Participants	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, weeks 4, 12, 14

Population: SAF with available data was analyzed.

PVR was assessed by ultrasonography.

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=10 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) n=2 Participants Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) n=1 Participants Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=10 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Change From Baseline in Post Void Residual (PVR) Volume Week 4	8.80 mL Standard Deviation 29.90	-3.00 mL Standard Deviation 9.90	—	-0.56 mL Standard Deviation 11.59
Change From Baseline in Post Void Residual (PVR) Volume Week 12	5.43 mL Standard Deviation 12.79	5.00 mL Standard Deviation NA Only 1 participant was analyzed, therefore, SD was not possible to be calculated.	0.00 mL Standard Deviation NA Only 1 participant was analyzed, therefore, SD was not possible to be calculated.	1.63 mL Standard Deviation 4.27
Change From Baseline in Post Void Residual (PVR) Volume Week 14	11.0 mL Standard Deviation 33.60	4.50 mL Standard Deviation 0.71	0.00 mL Standard Deviation NA Only 1 participant was analyzed, therefore, SD was not possible to be calculated.	2.20 mL Standard Deviation 8.44

SECONDARY outcome

Timeframe: Week 12

Population: SAF with available data was analyzed.

Participants evaluated the taste of the study drug/tablets by ticking 1 of the following categories: "Really Bad" (0), "Bad" (1), "Not Bad, Not Good" (2), "Good" (3) \& "Really Good" (4). Participants evaluated the swallow of the study drug/tablets by ticking one of the following categories: "Really Difficult" (0), "Difficult" (1), "Not Difficult, Not Easy" (2), "Easy" (3) and "Really Easy" (4).

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=1 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) n=2 Participants Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=3 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Number of Participants With Study Drug Acceptability and Palatability for Tablets Taste: Really bad	0 Participants	0 Participants	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Taste: Bad	0 Participants	0 Participants	—	1 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Taste: Not Bad, Not Good	1 Participants	2 Participants	—	1 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Taste: Good	0 Participants	0 Participants	—	1 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Taste: Really Good	0 Participants	0 Participants	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Swallow: Really Difficult	0 Participants	0 Participants	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Swallow: Difficult	0 Participants	0 Participants	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Swallow: Not Difficult, Not Easy	0 Participants	1 Participants	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Swallow: Easy	0 Participants	0 Participants	—	2 Participants
Number of Participants With Study Drug Acceptability and Palatability for Tablets Swallow: Really Easy	1 Participants	1 Participants	—	1 Participants

SECONDARY outcome

Timeframe: Week 12

Population: SAF with available data was analyzed.

Participants evaluated the taste of the study drug/oral suspension by ticking 1 of the following categories: "Really Bad" (0), "Bad" (1), "Not Bad, Not Good" (2), "Good" (3) \& "Really Good" (4). Participants evaluated the smell of the study drug/oral suspension by ticking 1 of the following categories: "Really Bad" (0), "Bad" (1),"Not Bad, Not Good" (2), "Good" (3) \& "Really Good" (4). Participants evaluated the consumption of the study drug/oral suspension by ticking 1 of the following categories: "Really Difficult" (0),"Difficult" (1), "Not Difficult, Not Easy" (2), "Easy" (3) \& "Really Easy" (4). Participants evaluated the preparation of the study drug/oral suspension by ticking 1 of the following categories: "Really Difficult" (0), "Difficult" (1), "Not Difficult, Not Easy" (2), "Easy" (3) \& "Really Easy" (4).

Outcome measures

Outcome measures
Measure	Placebo (5 to < 12 Years) n=7 Participants Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (12 to < 18 Years) Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron (5 to < 12 Years) n=6 Participants Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taste: Really Bad	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taste: Bad	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taste: Not Bad, Not Good	6 Participants	—	—	4 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taste: Good	1 Participants	—	—	1 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taste: Really Good	0 Participants	—	—	1 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Smell: Really Bad	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Smell: Bad	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Smell: Not Bad, Not Good	4 Participants	—	—	3 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Smell: Good	2 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Smell: Really Good	1 Participants	—	—	3 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taking: Really Difficult	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taking: Difficult	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taking: Not Difficult, Not Easy	1 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taking: Easy	5 Participants	—	—	4 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Taking: Really Easy	1 Participants	—	—	2 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Preparing: Really Difficult	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Preparing: Difficult	0 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Preparing: Not Difficult, Not Easy	2 Participants	—	—	0 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Preparing: Easy	5 Participants	—	—	4 Participants
Number of Participants With Study Drug Acceptability and Palatability for Oral Suspension Preparing: Really Easy	0 Participants	—	—	2 Participants

Adverse Events

Mirabegron(5 to<12 Years)

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Placebo (5 to < 12 Years)

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Mirabegron(12 to<18 Years)

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo (12 to < 18 Years)

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Mirabegron(5 to<12 Years) n=11 participants at risk Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (5 to < 12 Years) n=12 participants at risk Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron(12 to<18 Years) n=2 participants at risk Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) n=1 participants at risk Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Injury, poisoning and procedural complications Diffuse axonal injury	0.00% 0/11 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	50.0% 1/2 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Investigations Electrocardiogram QT prolonged	0.00% 0/11 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	100.0% 1/1 • Number of events 1 • From first dose up to week 14 SAF

Other adverse events

Other adverse events
Measure	Mirabegron(5 to<12 Years) n=11 participants at risk Participants aged 5 to \< 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (5 to < 12 Years) n=12 participants at risk Participants aged 5 to \< 12 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Mirabegron(12 to<18 Years) n=2 participants at risk Participants aged 12 to \< 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.	Placebo (12 to < 18 Years) n=1 participants at risk Participants aged 12 to \< 18 years received placebo matched to mirabegron orally once daily based on weight PED25 on day 1. Participants with a body weight ≥ 35 kg received tablet and participants with a body weight\< 35 kg or those who could not be dosed with the tablet received an oral suspension. At week 4, participants were up-titrated to the PED50 based on the given dose titration criteria up to week 12. Urotherapy continued throughout the study treatment period until week 12.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Gastrointestinal disorders Constipation	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 2 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Gastrointestinal disorders Dry mouth	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Gastrointestinal disorders Vomiting	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
General disorders Fatigue	9.1% 1/11 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
General disorders Medical device pain	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
General disorders Pyrexia	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 3 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Infections and infestations COVID-19	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Infections and infestations Gastroenteritis viral	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Infections and infestations Nasopharyngitis	18.2% 2/11 • Number of events 2 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 2 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Infections and infestations Respiratory tract infection viral	9.1% 1/11 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Infections and infestations Upper respiratory tract infection	9.1% 1/11 • Number of events 1 • From first dose up to week 14 SAF	16.7% 2/12 • Number of events 2 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Infections and infestations Viral infection	0.00% 0/11 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	50.0% 1/2 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Injury, poisoning and procedural complications Eyelid abrasion	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Injury, poisoning and procedural complications Head injury	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Injury, poisoning and procedural complications Skin abrasion	0.00% 0/11 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	50.0% 1/2 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Investigations Electrocardiogram QT prolonged	9.1% 1/11 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Nervous system disorders Headache	9.1% 1/11 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	100.0% 1/1 • Number of events 1 • From first dose up to week 14 SAF
Psychiatric disorders Mood swings	9.1% 1/11 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/12 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Reproductive system and breast disorders Genital erythema	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF
Skin and subcutaneous tissue disorders Dermatitis	0.00% 0/11 • From first dose up to week 14 SAF	8.3% 1/12 • Number of events 1 • From first dose up to week 14 SAF	0.00% 0/2 • From first dose up to week 14 SAF	0.00% 0/1 • From first dose up to week 14 SAF

Additional Information

Clinical Trial Transparency

Astellas Pharma Global Development Inc

Phone: 8008887704

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Institute and/or Principal Investigator may publish trial data generated at their specific study site after Sponsor publication of the multi-center data. Sponsor must receive a site's manuscript prior to publication for review and comment as specified in the Investigator Agreement.
Publication restrictions are in place

Restriction type: OTHER