Pilot Study of Mirabegron in Pediatric Patients With Overactive Bladder

NCT02468830 | Completed Phase 3 Results DMC

• 1 other identifier: B14-06-1998
• Study Type: interventional
• Target: 58
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Overactive Bladder; Urinary Incontinence

Keywords

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

Outcome Measures

Primary Outcomes (2)

• Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron

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

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

• Improved Overactive Bladder Symptoms as a Measure of Efficacy of Mirabegron

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

  Participants will be followed for duration of the study, 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

• Improved Quality of Life Using the Patient Perception of Bladder Condition (PPBC) Scale

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

• Number of Participants With Adverse Events as a Measure of Safety and Tolerability of Mirabegron

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

Study Arms (1)

Mirabegron

EXPERIMENTAL

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.

Drug: mirabegron

Interventions

mirabegron DRUG

Switch treatment from antimuscarinic to study medication. A dose up-titration will be possible if well tolerated and sub-optimal efficacy.

Also known as: Myrbetriq

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) or with significantly bothersome S/E on antimuscarinics.

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

Related Publications (1)

• Blais AS, Nadeau G, Moore K, Genois L, Bolduc S. Prospective Pilot Study of Mirabegron in Pediatric Patients with Overactive Bladder. Eur Urol. 2016 Jul;70(1):9-13. doi: 10.1016/j.eururo.2016.02.007. Epub 2016 Feb 11.

  PMID: 26876327 RESULT

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: May 30, 2015
• First Posted: June 11, 2015
• Study Start: April 1, 2013
• Primary Completion: January 1, 2016
• Study Completion: March 1, 2016
• Last Updated: June 20, 2018
• Results First Posted: June 20, 2018

Record last verified: 2018-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR
• Time Frame: March 2018
• Access Criteria: publication