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Evaluating the effectiveness of Mirabegron in young children with bladder control issues

A Phase 3, Open Label, Multicenter, Baseline-Controlled Sequential Dose Titration Study Followed by a Fixed Dose Observation Period to Evaluate Pharmacokinetics, Efficacy and Safety of Mirabegron Prolonged-Release Microgranula-Based Suspension in Children From 6 Months to Less Than 3 Years of Age With Neurogenic Detrusor Overactivity

Phase 3 Interventional Astellas Pharma Inc · NCT05621616

This study is testing if the medication Mirabegron can help young children with bladder control problems by increasing how much their bladder can hold.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment10 (estimated)
Ages6 Months to 3 Years
SexAll
SponsorAstellas Pharma Inc Industry-sponsored
Locations11 sites (Palo Alto, California and 10 other locations)
Trial IDNCT05621616 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and effectiveness of Mirabegron in children aged 6 months to less than 3 years who have neurogenic detrusor overactivity (NDO). The study will measure how well Mirabegron increases the bladder's maximum capacity, which could help prevent incontinence caused by high bladder pressure. Participants must be fitted with a catheter for urine drainage and meet specific weight and health criteria. The trial will involve regular monitoring and adherence to a catheterization regimen.

Who should consider this trial

Good fit: Ideal candidates are children aged 6 months to less than 3 years with a diagnosis of neurogenic detrusor overactivity and who weigh at least 9 kg.

Not a fit: Patients who do not have neurogenic detrusor overactivity or are unable to use clean intermittent catheterization may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve bladder control and quality of life for young children with NDO.

How similar studies have performed: Previous studies have shown that Mirabegron is effective in older children and adults, suggesting potential success in this younger population.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant's weight is a minimum of 9 kg.
* Participant has a previous myelomeningocele (documented at the screening visit).
* Participant has a diagnosis of neurogenic detrusor overactivity (NDO) confirmed by urodynamic investigation at baseline (day 1). The diagnosis of NDO should be confirmed by the presence of ≥ 1 involuntary detrusor contraction \> 15 cm H2O from baseline detrusor pressure, and/or a decrease in bladder compliance leading to an increase in baseline detrusor pressure of \> 20 cm H2O.
* Participant has a diagnosis of detrusor sphincter dyssynergia (DSD).
* Participant is using clean intermittent catheterization (CIC).
* Participant is suitable for a regimen of 4 to 6 CICs per day, fixed for the duration of the study using the 7-day baseline e-diary.
* Participant is able to swallow the study drug.
* Participant's legally authorized representative (LAR) is willing and able to comply with the study requirements (including compliant use of the e-diary) and with the concomitant medication restrictions.
* Participant's LAR agree not to allow participant to participate in another interventional study while receiving study intervention and throughout the pretreatment period.

Exclusion Criteria:

* Participant has a bladder capacity less than 25% of expected age-related capacity, confirmed by urodynamic investigation at baseline (day 1).
* Participant has vesicoureteral reflux grade 3 to 5.
* Participant has a known genitourinary condition, other than NDO, that may cause overactive contractions and/or incontinence (e.g., bladder exstrophy, urinary tract obstruction, urethral diverticulum or fistula) or kidney/bladder stones or another persistent local pathology that may cause urinary symptoms.
* Participant has had an indwelling urinary catheter within 4 weeks prior to the baseline visit.
* Participant has undergone bladder augmentation surgery.
* Participant with surgically corrected underactive sphincter.
* Participant receives electrostimulation therapy, if started within 30 days before visit 1 screening or is expected to start during the study period. Participants who are on an established regimen (defined as starting more than 30 days before visit 1 screening) may remain on this for the duration of the study.
* Participant has been administered intravesical botulinum toxin; except if given \> 4 months prior to visit 1 screening and the participant experiences symptoms comparable to those existing prior to the botulinum toxin injections.
* Participant has a current symptomatic urinary tract infection (UTI) confirmed by urinalysis (urine culture containing \> 100,000 cfu/mL) at baseline. If at screening and start of washout a UTI is present, the participant will be eligible for enrollment if the UTI has been treated successfully prior to baseline. If a symptomatic UTI is present at baseline, all baseline assessments should be postponed for a maximum of 7 days until the UTI is successfully treated. Successful treatment is defined as a symptom free patient with a white blood cell count in the urine \< 100/microliter and urine culture below 100,000 cfu/mL.
* Participant is using prohibited medications.
* Participant has a diagnosis of central or congenital nephrogenic diabetes insipidus.
* Participant with severe gastrointestinal (GI) condition (including toxic megacolon) or any of the following GI conditions: partial or complete obstruction, decreased motility like paralytic ileus or at risk for gastric retention.
* Participant suffers from malnutrition or is severely overweight.
* Participant has an average QT interval corrected by Bazett's formula (QTcB) \> 440 ms (based on the QTcB mean from the screening and baseline ECG triplicates), history of QTc prolongation or risk of QT prolongation (e.g., hypokalemia, Long QT Syndrome (LQTS), or family history of LQTS, exercise induced syncope).
* Participant has severe renal impairment (estimated glomerular filtration rate (eGFR) \< 30 mL/min per 1.73 m\^2 for participants 1 year of age and older; serum creatinine ≥ 2 × ULN, with ULN defined as 97.5th percentile for participants 6 to \< 12 months of age.).
* Participant's aspartate aminotransferase (AST) or alanine aminotransferase (ALT) is ≥ 2 × upper limit of normal (ULN) or total bilirubin (TBL) greater than or equal to 1.5 × ULN.
* Participant has a current or previous history of epilepsy.
* Participant has a history or presence of any malignancy prior to visit 1 screening.
* Participant has any other clinically significant out of range results of urinalysis, biochemistry, hematology or coagulation.
* Participant has an established hypertension and systolic or diastolic blood pressure greater than the 99th percentile of their normal range determined by gender, body size and age, plus 5 mmHg.
* Participant has a (median) resting heart rate \> 99th percentile.
* Participant has any clinically significant or unstable medical condition or disorder which precludes the participant from participating in the study.
* Participant has known or suspected hypersensitivity to mirabegron, any of the excipients used in the current formulation or previous severe hypersensitivity to any drug.
* Participant has participated in another clinical trial and/or has taken an investigational drug within 30 days (or 5 half-lives of the drug, or the limit set by national law, whichever is longer) prior to visit 1 screening.
* Participant is being breast-fed by a woman taking any prohibited medication or fed with a milk product in which the presence of prohibited medication ingredients cannot be excluded.

Where this trial is running

Palo Alto, California and 10 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Neurogenic Detrusor OveractivityPediatric acceptabilityBladder capacityPharmacokinetics of mirabegron
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.