Add-on Mirabegron in Pediatric Patients With Refractory Overactive Bladder

NCT02476175 | Completed Phase 3 Results DMC

• 1 other identifier: B14-06-1998-2
• Study Type: interventional
• Target: 35
• Locations: 0 countries
• Sites: N/A

Brief Summary

The objective is to evaluate the efficacy and safety of adding mirabegron to an antimuscarinic to treat urinary incontinence in children with Overactive Bladder that are refractory to antimuscarinics.

Conditions

Overactive Bladder; Urinary Incontinence

Keywords

mirabegron; antimuscarinics; beta 3 agonist; overactive bladder; children

Outcome Measures

Primary Outcomes (2)

• Response to Urinary Incontinence as a Composite Measure of Efficacy of add-on Mirabegron

  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).

  up to 52 weeks

• Number of Participants With Grade 2 or 3 Urgency Episodes as a Measure of Efficacy

  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.

  up to 52 weeks

Secondary Outcomes (3)

• Number of Participants With Cardio Vascular Safety

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

• Number of Participants Showing Improved Quality of Life Using the Patient Perception of Bladder Condition Scale and Voiding Diaries

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

• Number of Participants Without Variation in Heart Rate

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

Study Arms (1)

Add-on Mirabegron

EXPERIMENTAL

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).

Drug: Mirabegron

Interventions

Mirabegron DRUG

Patients will keep the antimuscarinic providing some efficacy and that is tolerated and Mirabegron will be added (dual therapy).

Also known as: Myrbetriq

Add-on Mirabegron

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female ≥ 5 years old and ≤17 years old
• OAB diagnostic according to the International Children Continence Society (ICCS) and less than 65% of the expected mean bladder capacity for age is confirmed (30 + (age in years x 30) mL) on a 3-day voiding diary.
• Weight and height are within the normal percentile (3rd to 97th percentile) and weight is ≥ 20 kg (3rd percentile of a 8 y.o. child, boy or girl), according to the CDC growth chart
• Ability to swallow pills
• Subjects/parents (vs. legal guardian) agree to participate to the following study and sign the informed consent
• Subjects/parents (vs. legal guardian) are able to comply with the study requirements and with the medication restrictions.
• Female subjects of childbearing potential must have a negative serum or urine pregnancy test at enrollment and must agree to maintain highly effective birth control during the study. Sexually active male subjects agree to use a barrier method of birth control with female partner for the duration of the study and at least one month after ending study treatment. Sexually active male subjects agree to use a condom for the duration of the study and for at least one month after ending study treatment and the female partner to use a reliable form of birth control for the duration of the study and for at least one month after ending study treatment.
• Patients without symptom improvement or with partial response under medical therapy (at least 2 different antimuscarinic agents).

You may not qualify if:

• Subject has a diagnostic of dysfunctional voiding
• Post-voiding residue \> 20 cc
• Polyuria (\> 75 ml/kg/b.w./24 hours)
• Nephrogenic of central diabetes insipidus
• Constipation at screening (if the patient is treated and the treatment is successful, the patient will be eligible to the study)
• Urinary tract infection at visit 2-3-4. If UTI is present at the screening visit, the UTI must be treated and the success of the treatment must be documented with a negative urinalysis at visit 2.
• QTc interval greater than 460 ms, or any increase of 30 ms on follow-up EKG (mean of 6 separate EKG-3 from visit week-2 and 3 from visit week 0). If a patient meets those criteria in the first month (initial dose), he will be excluded from the study. If the QTc change is noted after the up-titration, the dose will be decreased and EKG will be repeated within 1 week to ensure normalization of QTc.
• Clinically significant unstable medical condition or disorder
• Subject is pregnant or intends to become pregnant
• Serum creatinin more than or equal to 2 times the upper limit of normal
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) more than or equal to 2 times ULN, or bilirubin more than or equal to 1.5 times ULN.
• Known hypersensitivity to mirabegron or any contraindication to the use of the molecule, in accordance to the product monography (to the exception of pediatric age).
• Subject is taking medication that interact with mirabegron and this medication can't be discontinued (see appendix 1 of excluded drugs)
• Known urological pathology other than OAB that could explain urinary symptoms (as bladder stone…)
• Non-treated or non-controlled arterial hypertension

Sponsors & Collaborators

• CHU de Quebec-Universite Laval: lead

MeSH Terms

Conditions

Urinary Bladder, Overactive; Urinary Incontinence

Interventions

mirabegron

Condition Hierarchy (Ancestors)

Urinary Bladder Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Lower Urinary Tract Symptoms; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Urination Disorders

Results Point of Contact

• Title: Dr Stephane Bolduc
• Organization: CHU de Quebec-Universite Laval

Stephane.Bolduc@fmed.ulaval.ca

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Pediatric Urologist, MD, FRCSC, FAAP

Study Record Dates

• First Submitted: June 14, 2015
• First Posted: June 19, 2015
• Study Start: April 1, 2013
• Primary Completion: April 1, 2016
• Study Completion: June 1, 2016
• Last Updated: April 24, 2018
• Results First Posted: April 24, 2018

Record last verified: 2018-03

Data Sharing

• IPD Sharing: Will not share