Neurogenic Detrusor Overactivity (NDO) MedDRA version: 21.1 Level: PT Classification code 10029279 Term: Neurogenic bladder System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Sponsors
Urovant Sciences GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female participants, age 2 years to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Participant has cerebral palsy, uncontrolled epilepsy, diabetes insipidus, or Stage 2 hypertension - Participant has an active malignancy in the 12 months prior to the Screening Visit. - Participant has been administered intravesical botulinum toxin within 9 months prior to the Screening Visit and should remain off this therapy during the study. - Participant is taking digoxin or lithium within 10 days prior to Screening Visit or plans to start taking either during the study. - Participant currently uses or plans to use a baclofen pump during the study. - Participant has urethral dilatation or has had urethral surgery in the 3 months prior to the Screening Visit. - Participant has undergone bladder augmentation surgery. - Participant has a known genitourinary condition (other than NDO) that may cause overactive contractions or incontinence (bladder exstrophy, urinary tract obstruction, urethral diverticulum or fistula) or bladder stones or another persistent urinary tract pathology that may cause symptoms. - Participant has an insufficient urethral sphincter, has had implantation of an artificial sphincter, has a surgically-treated underactive urethral sphincter, or, in the 6 months prior to the Screening Visit, has undergone pelvic gender reassignment surgery. - Participant has one of the following gastrointestinal problems: partial or complete obstruction, decreased motility such as paralytic ileus, risk of gastric retention, or malabsorption syndrome of any form. - Participant has fecal impaction or a history of fecal impaction requiring hospitalization or ambulatory surgical treatment in the 3 months prior to the Screening Visit. - Participant has a urinary indwelling catheter in the 4 weeks prior to the Screening Visit. - Participant has moderate to severe dilating vesicoureteral reflux (Grade III to V) or severe renal failure. - Participant started electrostimulation/neuromodulation therapy in the 4 weeks before the Screening Visit, or is expected to start this therapy during the study period. - Participant has participated in another clinical trial and/or has taken an investigational drug within 4 weeks prior to the Screening Visit. - Participant is unable, or parent/caregiver is not willing, to washout any medication for the management of NDO. - Participant is a female of childbearing potential who is unwilling or unable to use a highly effective method of contraception for the duration of the study. - Female participants who are currently breastfeeding or plan to breastfeed any time from the Screening Visit until 28 days after the final study drug administration.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of QD vibegron administration in pediatric subjects 2 years to < 18 years with NDO;Secondary Objective: - To evaluate safety and tolerability of vibegron in pediatric subjects 2 years to <18 years with NDO - To evaluate the pharmacokinetic (PK) profile of vibegron after multiple-dose administration in pediatric subjects 2 years to <18 years with NDO ;Primary end point(s): - Change from baseline at Study Week 32 (Optimized Treatment Week 24) in MCC based on filling urodynamics;Timepoint(s) of evaluation of this end point: As defined in the primary end points | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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: o Number of overactive detrusor contractions (> 15 cm H2O) until end of filling o Detrusor pressure at end of filling o Filling volume until first involuntary/hyperactive detrusor contraction (> 15 cm H2O) o Bladder compliance (?V/?P) Based on Bladder Diary: - Change from baseline at Study Weeks 1, 4, 8, 20, 32, 48, and 52 in: o Average first morning catheterized volume (collected in supine position) o Average catheterized volume per catheterization o Average maximum catheterized volume per day (day and night time) o Average maximum catheterized daytime volume o Average number of leakage episodes per day (day and night time) o Estimated number of dry (leakage-free) days/7 days (day and night time) 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;Timepoint(s) of evaluation of this end point: - Change from baseline at Study Week 20 in MCC - Change from baseline at Study Weeks 20 and 32 - Change from baseline at Study Weeks 1, 4, 8, 20, 32, 48, and 52 - Change from baseline at Study Weeks 20, 32, and 52 - AEs & AESIs (Part-A & B)- Screening to follow-up - Upper urinary tract ultrasound (Part-A & B)- Day 1, Week 52 or Early Withdrawal (WD) - eGFR (Part-A)- Screening, Week 20, Week 32, Week 52 or Early WD - eGFR (Part-B) - Screening, Week 12, Week | — |
Countries
Armenia, Belgium, Canada, Croatia, Denmark, Georgia, Jordan, Latvia, Lithuania, Malaysia, Mexico, Norway, Philippines, Poland, Romania, Serbia, Slovakia, Türkiye, United States
Contacts
Public ContactTina Lamberts
Syneos Health
Outcome results
None listed