Status:
COMPLETED
Open-label Phase 3 Study With Mirabegron in Children From 3 to Less Than 18 Years of Age With Neurogenic Detrusor Overactivity
Lead Sponsor:
Astellas Pharma Europe B.V.
Conditions:
Neurogenic Detrusor Overactivity
Eligibility:
All Genders
3-17 years
Phase:
PHASE3
Brief Summary
The objective of the study was to evaluate the efficacy, safety, tolerability and pharmacokinetics of mirabegron after multiple-dose administration in the pediatric population.
Detailed Description
This was a phase 3, open-label, baseline-controlled, multicenter study. The study consisted of 3 periods: Pretreatment period: for a maximum of 28 days before baseline, including screening, washout (i...
Eligibility Criteria
Inclusion
- Subject has a body weight of greater than or equal to 11 kg.
- Subject suffers from NDO confirmed by urodynamic investigation at baseline. The diagnosis of NDO must be confirmed by the presence of at least 1 involuntary detrusor contraction \> 15 cm H2O from baseline detrusor pressure, and/or a decrease in compliance leading to an increase in baseline detrusor pressure of \> 20 cm H2O.
- Subject has been using CIC for at least 4 weeks prior to visit 1/screening.
- Subject has a current indication for drug therapy to manage NDO.
- Subject is able to take the study drug in accordance with the protocol
Exclusion
- Subject has a known genitourinary condition (other than NDO) that may cause overactive contractions or incontinence or kidney/bladder stones or another persistent urinary tract pathology that may cause symptoms.
- Subject has one of the following gastrointestinal problems: partial or complete obstruction, decreased motility such as paralytic ileus, subjects at risk of gastric retention.
- Subject has a urinary indwelling catheter within 4 weeks prior to visit 1/screening.
- Subject has a surgically treated underactive urethral sphincter
- Subject has vesico-ureteral reflux grade 3 to 5.
- Subject has undergone bladder augmentation surgery.
- Subject receives electrostimulation therapy, if started within 30 days before visit 1/screening or is expected to start during the study period. Subjects who are on an established regimen may remain on this for the duration of the study.
- Subject suffers from a symptomatic urinary tract infection (UTI) at baseline (symptomatic is defined as pain, fever, hematuria, new onset foul-smelling urine). If present at visit 1/screening or diagnosed between visit 1/screening and visit 3/baseline, the UTI should be treated successfully (clinical recovery) prior to baseline. If a symptomatic UTI is present at baseline, all baseline assessments are allowed to be postponed for a maximum of 7 days until the UTI is successfully treated (clinical recovery).
- Subject has a (mean) resting pulse rate \> 99th percentile \[Fleming et al, 2011\].
- Subject has an established hypertension and a systolic or diastolic blood pressure greater than the 99th percentile of the normal range determined by sex, age and height, plus 5mmHg \[NIH 2005\].
- Subject has a risk of QT prolongation (e.g., hypokalemia, long QT syndrome \[LQTS\]; or family history of LQTS, exercise-induced syncope).
- Subject has severe renal impairment (eGFR according to Larsson equation \< 30 mL/min).
- Subject's aspartate aminotransferase (AST) or alanine aminotransferase (ALT) is greater than or equal to 2 times the upper limit of normal (ULN) or total bilirubin (TBL) greater than or equal to 1.5 times the ULN according to age and sex.
- Subject has a history or presence of any malignancy prior to visit 1/screening.
- Subject has known or suspected hypersensitivity to mirabegron, any of the excipients used in the current formulations or previous severe hypersensitivity to any drug.
- Subject 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.
- Subject uses any of the following prohibited medications (after start of washout):
- Any medication, other than the study drug used, for the management of NDO;
- Any drugs that are sensitive CYP2D6 substrates with a narrow therapeutic index or sensitive P-glycoprotein (P-gp) substrates
- Any strong CYP3A4 inhibitors if the subject has a mild to moderate renal impairment (eGFR 30 - 89 mL/min).
- Subject has been administered intravesical botulinum toxin; except if given \> 4 months prior to visit 1/screening and the subject experiences symptoms comparable to those existing prior to the botulinum toxin injections.
Key Trial Info
Start Date :
June 17 2016
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 6 2019
Estimated Enrollment :
91 Patients enrolled
Trial Details
Trial ID
NCT02751931
Start Date
June 17 2016
End Date
May 6 2019
Last Update
November 12 2024
Active Locations (32)
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1
Site AU61002
Randwick, New South Wales, Australia, 2031
2
Site BE32004
Edegem, Belgium, B-2650
3
Site BE32001
Ghent, Belgium, 9000
4
Site HR38501
Zagreb, Croatia, 10000