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A study to learn how effective and safe the drug “mirabegron” is and how long it stays in the body of children aged 6 months to less than 3 years of age with neurogenic detrusor overactivity
EU CTIS ID: 2023-507903-74-00
What this study is testing
To evaluate the efficacy of mirabegron prolonged-release microgranula-based suspension after multiple dose administration in the pediatric population.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. IRB/IEC approved written informed consent and privacy language as per national regulations (e.g., Health Insurance Portability and Accountability Act authorization for US study sites) must be obtained from the participant’s LAR prior to any study-related procedures (including withdrawal of prohibited medication, if applicable).
- 10. Participant’s LAR is willing and able to comply with the study requirements (including compliant use of the e-diary) and with the concomitant medication restrictions.
- 11. Participant’s LAR agree not to allow participant to participate in another interventional study while receiving study intervention and throughout the pretreatment period.
- 2. Participant is male or female and 6 months to less than 3 years of age.
- 3. Participant’s weight is a minimum of 9 kg.
- 4. Participant has a previous myelomeningocele (documented at the screening visit).
You likely can't join if
- 1. Participant has a bladder capacity less than 25% of expected age-related capacity, confirmed by urodynamic investigation at baseline (day 1).
- 10. Participant is using prohibited medications listed in [Section 10.5].
- 11. Participant has a diagnosis of central or congenital nephrogenic diabetes insipidus.
- 12. 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.
- 13. Participant suffers from malnutrition or is severely overweight, in the opinion of the investigator. See [Section 10.8.1].
- 14. Participant has an average 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, LQTS, or family history of LQTS, exercise induced syncope).
See the full eligibility criteria
Who can join
- 1. IRB/IEC approved written informed consent and privacy language as per national regulations (e.g., Health Insurance Portability and Accountability Act authorization for US study sites) must be obtained from the participant’s LAR prior to any study-related procedures (including withdrawal of prohibited medication, if applicable).
- 10. Participant’s LAR is willing and able to comply with the study requirements (including compliant use of the e-diary) and with the concomitant medication restrictions.
- 11. Participant’s LAR agree not to allow participant to participate in another interventional study while receiving study intervention and throughout the pretreatment period.
- 2. Participant is male or female and 6 months to less than 3 years of age.
- 3. Participant’s weight is a minimum of 9 kg.
- 4. Participant has a previous myelomeningocele (documented at the screening visit).
- 5. Participant has a diagnosis of 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.
- 6. Participant has a diagnosis of DSD.
- 7. Participant is using CIC.
- 8. Participant is suitable for a regimen of 4 to 6 CICs per day, fixed for the duration of the study in the opinion of the investigator using the 7-day baseline e-diary.
- 9. Participant is able to swallow the study drug.
What rules you out
- 1. Participant has a bladder capacity less than 25% of expected age-related capacity, confirmed by urodynamic investigation at baseline (day 1).
- 10. Participant is using prohibited medications listed in [Section 10.5].
- 11. Participant has a diagnosis of central or congenital nephrogenic diabetes insipidus.
- 12. 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.
- 13. Participant suffers from malnutrition or is severely overweight, in the opinion of the investigator. See [Section 10.8.1].
- 14. Participant has an average 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, LQTS, or family history of LQTS, exercise induced syncope).
- 15. Participant has severe renal impairment (eGFR < 30 mL/min per 1.73 m2 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 [Table 9; Boer et al, 2010]).
- 16. Participant’s AST or ALT is ≥ 2 × ULN or TBL greater than or equal to 1.5 × ULN.
- 17. Participant has a current or previous history of epilepsy
- 18. Participant has a history or presence of any malignancy prior to visit 1 screening.
- 19. Participant has any other clinically significant out of range results of urinalysis, biochemistry, hematology or coagulation as per the investigator’s interpretation.
- 2. Participant has vesicoureteral reflux grade 3 to 5 in the opinion of the investigator.
- 20. 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 (reference for normal blood pressure for children < 1 year old is [Report of the second task force on blood pressure control in children, 1987] and for children ≥ 1 years old is [Flynn et al, 2017]).
- 21. Participant has a (median) resting heart rate > 99th percentile [Section 10.7.3].
- 22. Participant has any clinically significant or unstable medical condition or disorder which, in the opinion of the investigator, precludes the participant from participating in the study.
- 23. Participant has known or suspected hypersensitivity to mirabegron, any of the excipients used in the current formulation or previous severe hypersensitivity to any drug.
- 24. 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.
- 25. 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.
- 3. 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.
- 4. Participant has had an indwelling urinary catheter within 4 weeks prior to the baseline visit.
- 5. Participant has undergone bladder augmentation surgery.
- 6. Participant with surgically corrected underactive sphincter.
- 7. 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.
- 8. 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.
- 9. Participant has a current symptomatic 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 successfullu 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.
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- United States
- Philippines
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; United States; Philippines. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.