Trial Outcomes & Findings for Add-on Mirabegron in Pediatric Patients With Refractory Overactive Bladder (NCT NCT02476175)

Results Overview

Results were based on the International Children's Continence Sociéty classification. Parents or patients supervised by their parents rated symptom relief on a questionnaire as complete cure (definied as dryness) or partial response (reduction of 50% to 99% in incontinence episodes).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

35 participants

Primary outcome timeframe

up to 52 weeks

Results posted on

2018-04-24

Participant Flow

Participant milestones

Participant milestones
Measure	Add-on Mirabegron Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Overall Study STARTED	35
Overall Study COMPLETED	33
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Add-on Mirabegron Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Overall Study Adverse Event	2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Add-on Mirabegron n=35 Participants Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Age, Categorical <=18 years	35 Participants n=35 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=35 Participants
Age, Categorical >=65 years	0 Participants n=35 Participants
Sex: Female, Male Female	7 Participants n=35 Participants
Sex: Female, Male Male	28 Participants n=35 Participants
Region of Enrollment Canada	35 Participants n=35 Participants

PRIMARY outcome

Timeframe: up to 52 weeks

Results were based on the International Children's Continence Sociéty classification. Parents or patients supervised by their parents rated symptom relief on a questionnaire as complete cure (definied as dryness) or partial response (reduction of 50% to 99% in incontinence episodes).

Outcome measures

Outcome measures
Measure	Add-on Mirabegron n=35 Participants Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Response to Urinary Incontinence as a Composite Measure of Efficacy of add-on Mirabegron Complete response:100%improvement of incontinence	12 Participants
Response to Urinary Incontinence as a Composite Measure of Efficacy of add-on Mirabegron Partial response: 50 to 99 % reduction	23 Participants

PRIMARY outcome

Timeframe: up to 52 weeks

On voiding diary, participants described their urgency according to the Canadian Urological Association voiding diary, range 0 to 3 at study entry and at study end.

Outcome measures

Outcome measures
Measure	Add-on Mirabegron n=35 Participants Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Number of Participants With Grade 2 or 3 Urgency Episodes as a Measure of Efficacy Participants with urgency 2-3 at entry	32 Participants
Number of Participants With Grade 2 or 3 Urgency Episodes as a Measure of Efficacy Participants without urgency2-3 at week 52studyend	19 Participants

SECONDARY outcome

Timeframe: Participants will be followed for the duration of the study, up to 52 weeks

Cardiovascular safety: mean difference in blood pressure (Variation in blood pressure: systolic ±20 mmHg, diastolic ±15 mmHg), blood pressure was taken at each visit and at the study end.

Outcome measures

Outcome measures
Measure	Add-on Mirabegron n=35 Participants Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Number of Participants With Cardio Vascular Safety No variation:systolic ±20 mmHg in blood pressure	35 Participants
Number of Participants With Cardio Vascular Safety No variation:Diastolic ±15 mmHg in blood pressure	35 Participants

SECONDARY outcome

Timeframe: Participants will be followed for the duration of the study, up to 52 weeks

Effectiveness will also be assessed using the Patient Perception of Bladder Condition (PPBC) scale on a 6-point scale ranging from 1 to 6 ( 1 is the best score and 6 is the worst score), at study initiation, every visit and at the study end. Results will be documented based on subjective relief of symptoms and objective voiding diaries. Participants perception of bladder condition score at initiation of treatment was:4 and at last follow up score was: 2.

Outcome measures

Outcome measures
Measure	Add-on Mirabegron n=35 Participants Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Number of Participants Showing Improved Quality of Life Using the Patient Perception of Bladder Condition Scale and Voiding Diaries	35 Participants

SECONDARY outcome

Timeframe: Participants will be followed for the duration of the study, up to 52 weeks

Cardiovascular safety: mean difference in heart rate (with variation in heart rate increase of more than 20%). Heart rate was taken at initiation of study drug, at each visit and at the study end.

Outcome measures

Outcome measures
Measure	Add-on Mirabegron n=35 Participants Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Number of Participants Without Variation in Heart Rate	35 Participants

Adverse Events

Add-on Mirabegron

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Add-on Mirabegron n=35 participants at risk Experimental: Add-on Mirabegron Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. They will keep the antimuscarinic and Mirabegron will be added (dual therapy). Mirabegron: Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).
Gastrointestinal disorders Transcient constipation	5.7% 2/35 • Number of events 2
Gastrointestinal disorders Abdominal colic	2.9% 1/35 • Number of events 1
Eye disorders Temporary blurred vision	2.9% 1/35 • Number of events 1
General disorders Rhinitis	2.9% 1/35 • Number of events 1

Additional Information

Dr Stephane Bolduc

CHU de Quebec-Universite Laval

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place