Trial Outcomes & Findings for Pilot Study of Mirabegron in Pediatric Patients With Overactive Bladder (NCT NCT02468830)

NCT ID: NCT02468830

Last Updated: 2018-06-20

Results Overview

Percent change in the frequency of urinary incontinence episodes as a Measure of Efficacy.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 58 participants
• Primary outcome timeframe: Participants will be followed for the duration of the study, up to 52 weeks
• Results posted on: 2018-06-20

Participant Flow

Participant milestones

Measure	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. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Overall Study STARTED	58
Overall Study COMPLETED	55
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Study of Mirabegron in Pediatric Patients With Overactive Bladder

Baseline characteristics by cohort

Measure	Mirabegron n=58 Participants Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron (initial dosage 25 mg this dose wil be maintain or increase to a maximum of 50 mg based on persistent of symptomsand side effect profile): Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Age, Categorical <=18 years	58 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	44 Participants n=5 Participants
Region of Enrollment Canada	58 participants n=5 Participants

PRIMARY outcome

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

Percent change in the frequency of urinary incontinence episodes as a Measure of Efficacy.

Outcome measures

Measure	Mirabegron n=58 Participants Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron Complete elimination of urinary incontinence:100%	13 Participants
Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron Significant response > 90%-	14 Participants
Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron Partial response: 50-89%	25 Participants
Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron Failure < 50%	6 Participants

PRIMARY outcome

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

Change in mean bladder capacity from baseline to final visit based on voiding diary.

Outcome measures

Measure	Mirabegron n=58 Participants Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron baseline	150 milliliter Interval 125.0 to 200.0
Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron Final visit	200 milliliter Interval 171.0 to 300.0

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).

Parameters to be measure at each visit but particularly at visit 2 (Week 0, first dose on site), to be obtained before and 1 hour after taking the medication).

Outcome measures

Measure	Mirabegron n=58 Participants Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Number of Participants With Cardio Vascular Safety No variation:systolic ±20 mmHg in blood pressure	58 Participants
Number of Participants With Cardio Vascular Safety No variation:Diastolic ±15 mmHg in blood pressure	58 Participants

SECONDARY outcome

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

The Patient Perception of Bladder Condition (PPBC) scale on a 6-point score scale at baseline and final visit.

Explanation of possible answer:

1. does not cause me any problems at all,

2. causes me some very minor problems,

3. causes me some minor problems,

4. causes me (some) moderate problems,

5. causes me severe problems,

6. causes me many severe problems

Outcome measures

Measure	Mirabegron n=58 Participants Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Improved Quality of Life Using the Patient Perception of Bladder Condition (PPBC) Scale baseline	4.0 units on a scale Interval 4.0 to 5.0
Improved Quality of Life Using the Patient Perception of Bladder Condition (PPBC) Scale Final visit	2.0 units on a scale Interval 1.0 to 3.0

SECONDARY outcome

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

Cardiovascular safety: mean difference in heart rate (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

Measure	Mirabegron n=58 Participants Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability of Mirabegron No change in heart rate over 20%	58 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability of Mirabegron No significant change in heart rate	58 Participants

Adverse Events

Mirabegron

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Mirabegron n=58 participants at risk Patients without symptom improvement or with partial response under intensive behavioural protocol and medical therapy (at least 2 different antimuscarinic agents) will be recruited. Patients with significantly bothersome S/E on antimuscarinics will also be included. mirabegron: Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.
Gastrointestinal disorders Transient abdominal colic	3.4% 2/58 • Number of events 2
Gastrointestinal disorders Constipation	3.4% 2/58 • Number of events 2
Eye disorders Blurred vision	1.7% 1/58 • Number of events 1
Gastrointestinal disorders Nausea	1.7% 1/58 • Number of events 1
General disorders Change in behavior	1.7% 1/58 • Number of events 1
General disorders Nasopharyngitis	1.7% 1/58 • Number of events 1

Additional Information

Dr Stephane Bolduc

CHU de Quebec-Universite Laval

Email: Stephane.Bolduc@fmed.ulaval.ca

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place