Beta 3 Agonists in Treatment of Non-neurogenic Voiding Dysfunction in Children

Safety and Efficacy Beta 3 Agonists in Treatment of Non-neurogenic Voiding Dysfunction in Children, a Randomized Clinical Trial

A randomized clinical study to evaluate the safety and efficacy of using the Beta 3 agonist drug (Mirabegron) combined with standard behavioral therapy in comparison to using the anticholinergic drug (Solifenacin) combined with behavioral therapy, for children with non-neurogenic voiding dysfunction refractory to behavioral therapy alone, using improvement of dysfunctional voiding symptom score as a primary measurement of efficacy.

Study Overview

• Status: Not yet recruiting
• Conditions: Overactive Bladder; Voiding Disorders
• Intervention / Treatment: Drug: Beta3-Agonists, Adrenergic; Drug: Anticholinergic drug, Solifenacin

Detailed Description

The study aims to evaluate the beta 3 agonist drug (Mirabegron) as regard its safety and efficacy when used in children with non neurogenic voiding dysfunction, including children suffering from frequency, urgency, urgency incontinence and hesitancy who do not have neurological disease explaining these symptoms and who failed behavioral urotherapy.

The patients will be randomly allocated into two groups, one will receive standard behavioral therapy plus Mirabegron, the other will receive standard behavioral therapy plus the anticholinergic drug Solifenacin for three months. Patients will be assessed initially and after three months using:

• Medical and voiding history (bladder voiding diary, Dysfunctional voiding symptom score (DVSS) , Bristol Stool Scale)
• Dysfunctional voiding symptom score (DVSS) , Bristol Stool Scale
• Urine analysis and mid-stream urine culture.
• Uroflowmetry.
• Pelvic US and post-void residual urine (PVR).

• Study Type: Interventional
• Enrollment (Anticipated): 70
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Islam Mansour, MB ChB; Phone Number: +201147800607; Email: islamreda2205@gmail.com
• Study Contact Backup: Name: Ahmed Elhefnawy, PhD; Phone Number: +201224285870; Email: a_s_elhefnawy@yahoo.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Children with non-neurogenic voiding dysfunction predominantly filling phase dysfunctions or OAB, refractory to behavioral therapy as a primary monotherapy, with dysfunctional voiding symptom score of ≥6 for females and ≥9 for males, between the age of 5 & 18 years old.

Exclusion Criteria:

• Neurogenic or anatomical bladder problems.
• Patients with contraindications to Beta 3 agonists or anticholinergic drugs.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intervention; This arm will receive standard behavioral therapy plus Mirabegron with adjusted-dose regimen (25-50 mg), patients of 20-40 kg will receive 25-50 mg once a day; patients >40 kg will receive 50 mg once a day, for three months.	Drug: Beta3-Agonists, Adrenergic; The beta 3 agonist ,Mirabegron, has been shown to be effective for the treatment of OAB in adults through relaxation of bladder wall, with few side effects. This intervention aims to evaluate its efficacy and safety for children with non-neurogenic voiding dysfunction.; Other Names: Standard behavioral Urotherapy
Active Comparator: Control; This arm will receive will receive standard behavioral therapy plus Solifenacin with a daily dosage of (2.5-10 mg/kg), for three months.	Drug: Anticholinergic drug, Solifenacin; Anticholinergic drugs are widely used to treat overactive bladder by relaxation of bladder wall smooth muscles.; Other Names: Standard behavioral Urotherapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the Beta 3 agonist drug (Mirabegron) in improving symptoms of children with voiding dysfunction using dysfunctional voiding scoring system as a primary measure.	To evaluate the efficacy of using the Beta 3 agonist drug (Mirabegron) combined with standard behavioral therapy in comparison to using the anticholinergic drug (Solifenacin) combined with behavioral therapy, for children with non-neurogenic voiding dysfunction refractory to behavioral therapy alone, using improvement of dysfunctional voiding symptom score as a primary measurement of efficacy. This score consists of 10 voiding dysfunction parameters that are assigned scores of 0 to 3 according to prevalence, and possible total scores ranging from 0 to 30. The higher the score, the more severe the symptoms of voiding dysfunction. The documented cutoff score for voiding dysfunction is 6 and 9 for females and males respectively.	6 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the side effects of the beta 3 agonist drug (Mirabegron) in children.	This is planned to assess side effects and safety profile of the beta 3 agonist (Mirabegron) on children (5-18 years) from Egypt.	6 months
To validate an Arabic version of the Dysfunctional Voiding Scoring System as the documented cutoff score for voiding dysfunction is 6 and 9 for females and males respectively.	The investigators are planning to translate an Arabic version of the Dysfunctional Voiding Scoring System to use it to assess improvement of symptoms and patient recorded outcomes for Egyptian children before and after receiving Mirabegron.	6 months
To evaluate correlation between bladder ultrasound findings and severity of patients' symptoms. (normally it is less than 3 mm)	The investigators are planning to assess Ultrasound measurement of bladder wall thickness as an indicator of severity of patients' symptoms and whether there is bladder wall hypertrophy corresponding to severity of symptoms and whether the bladder wall thickness decreases with treatment.	6 months

Collaborators and Investigators

• Sponsor: Mansoura University
• Investigators: Principal Investigator: Islam Mansour, Mb ChB, Mansoura University

Study record dates

Study Major Dates

• Study Start (Anticipated): February 1, 2022
• Primary Completion (Anticipated): July 1, 2022
• Study Completion (Anticipated): December 1, 2022

Study Registration Dates

• First Submitted: December 11, 2021
• First Submitted That Met QC Criteria: February 13, 2022
• First Posted (Actual): February 15, 2022

Study Record Updates

• Last Update Posted (Actual): February 15, 2022
• Last Update Submitted That Met QC Criteria: February 13, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Urinary Bladder Diseases; Lower Urinary Tract Symptoms; Urological Manifestations; Urinary Bladder, Overactive; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Muscarinic Antagonists; Cholinergic Agents; Urological Agents; Adrenergic beta-Agonists; Adrenergic Agonists; Solifenacin Succinate; Adrenergic Agents; Cholinergic Antagonists; Adrenergic beta-3 Receptor Agonists
• Other Study ID Numbers: MS.21.09.1680

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No