A Phase 2/3, Open-label, Baseline-controlled, Multicenter, Long-term Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age with Neurogenic Detrusor Overactivity (NDO) on Clean Intermittent Catheterization (CIC)
Trial status: Authorised
Secondary Efficacy Endpoints: Based on Urodynamics: - Change from baseline at Study Week 20 (Optimized Treatment Week 12) in MCC - Change from baseline at Study Weeks 20 and 32 (Optimized Treatment Week 12 and 24) in: Number of overactive detrusor contractions until end of filling; Detrusor pressure at end of filling; Filling volume until first involuntary/hyperactive detrusor contraction; Bladder compliance, Secondary Efficacy Endpoints: Based on Bladder Diary: - Change from baseline at Study Weeks 1, 4, 8, 20, 32, 48, and 52 in: average first morning catheterized volume; Average catheterized volume per catheterization; average maximum catheterized volume per day; Average maximum catheterized daytime volume; Average number of leakage episodes per day; Estimated number of dry days/7 days, Secondary Efficacy Endpoints: Based on Questionnaires: - Change from baseline at Study Weeks 20, 32, and 52 in PIN-Q - Change from baseline at Study Weeks 20, 32, and 52 in PGI-S Scale - CGI-C Scale assessment at Study Weeks 20, 32, and 52., Safety and Tolerability: - Incidence of AEs - Incidence of AESIs - Upper urinary tract ultrasound assessment - eGFR - Vital signs measured at clinic visits: pulse rate, systolic and diastolic blood pressure - 12-lead ECG, centrally read, Pharmacokinetics: - PK of vibegron in plasma: Cmax, tmax, AUC(0-24h), Ctrough, t1/2, and CL/F
Change from baseline at Study Week 32 (Optimized Treatment Week 24) in MCC based on filling urodynamics
Описание
Secondary Efficacy Endpoints: Based on Urodynamics: - Change from baseline at Study Week 20 (Optimized Treatment Week 12) in MCC - Change from baseline at Study Weeks 20 and 32 (Optimized Treatment Week 12 and 24) in: Number of overactive detrusor contractions until end of filling; Detrusor pressure at end of filling; Filling volume until first involuntary/hyperactive detrusor contraction; Bladder compliance, Secondary Efficacy Endpoints: Based on Bladder Diary: - Change from baseline at Study Weeks 1, 4, 8, 20, 32, 48, and 52 in: average first morning catheterized volume; Average catheterized volume per catheterization; average maximum catheterized volume per day; Average maximum catheterized daytime volume; Average number of leakage episodes per day; Estimated number of dry days/7 days, Secondary Efficacy Endpoints: Based on Questionnaires: - Change from baseline at Study Weeks 20, 32, and 52 in PIN-Q - Change from baseline at Study Weeks 20, 32, and 52 in PGI-S Scale - CGI-C Scale assessment at Study Weeks 20, 32, and 52., Safety and Tolerability: - Incidence of AEs - Incidence of AESIs - Upper urinary tract ultrasound assessment - eGFR - Vital signs measured at clinic visits: pulse rate, systolic and diastolic blood pressure - 12-lead ECG, centrally read, Pharmacokinetics: - PK of vibegron in plasma: Cmax, tmax, AUC(0-24h), Ctrough, t1/2, and CL/F
Кой може да участва
Age group: 0-17 years
Gender: Female, Male
Target enrollment: 48 participants