Neurogenic Detrusor Overactivity
Conditions
Keywords
Neurogenic Detrusor Overactivity, Vibegron, Clean Intermittent Catheterization, Beta-3 Adrenergic Receptor Agonist, Maximum Cystometric Capacity, Spinal Dysraphism, Spina Bifida, Myelomeningocele, Meningocele, Spinal cord injury, Transverse myelitis
Brief summary
The purpose of this study is to evaluate the safety, efficacy, and PK of Vibegron in pediatric participants with NDO who are regularly using CIC
Interventions
DRUGVibegron
Participants will be administered Vibegron orally, once daily (QD)
Sponsors
Urovant Sciences GmbH
Sumitomo Pharma America, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Male or female participants, age 2 years to \< 18 years and weighing at least 11 kg at the Screening Visit. * Participant has been diagnosed with NDO due to one of the following: spinal dysraphism, which includes spina bifida (eg, myelomeningocele, meningocele) and all forms of tethered cord; or acquired NDO from a spinal cord injury or spinal cord surgery, with the injury/surgery having occurred at least 6 months prior to the Screening Visit; or acquired NDO due to transverse myelitis with diagnosis at least 12 months prior to the Screening Visit. * Participant undergoes CIC at least 3 times per 24 hours (with the last CIC performed prior to going to sleep for the night) for at least 4 weeks prior to the Screening Visit.
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 had urethral dilatation or 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 acute fecal impaction or, within the 3 months prior to the Screening Visit, had fecal impaction that required hospitalization or ambulatory surgical treatment. * Participant had a urinary indwelling catheter in the 4 weeks prior to the Screening Visit. * Participant has moderate to severe dilating vesicoureteral reflux (Grade IV 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 |
|---|---|
| Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics | Optimized Treatment Week 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in MCC | Optimized Week 12 | — |
| Change from Baseline in number of overactive detrusor contractions until the end of bladder filling | Optimized Treatment Weeks 12 and 24 | — |
| Change from Baseline in detrusor pressure at the end of bladder filling | Optimized Treatment Weeks 12 and 24 | — |
| Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction | Optimized Treatment Weeks 12 and 24 | — |
| Change from Baseline in bladder compliance (mL/cm H2O) | Optimized Treatment Weeks 12 and 24 | Bladder compliance is calculated by dividing the change in volume by the change in detrusor pressure during the filling of the bladder |
| Change from Baseline in average first morning catheterized volume | through study completion, an average of 52 weeks | — |
| Change from Baseline in average catheterized volume per catheterization | through study completion, an average of 52 weeks | — |
| Change from Baseline in average maximum catheterized volume per day | through study completion, an average of 52 weeks | — |
| Change from Baseline in average maximum catheterized daytime volume | through study completion, an average of 52 weeks | — |
| Change from Baseline in average number of leakage episodes per day | through study completion, an average of 52 weeks | — |
| Change from Baseline in estimated number of dry (leakage-free) days/ 7 days | through study completion, an average of 52 weeks | — |
| Change from Baseline in Pediatric Incontinence Questionnaire (PIN-Q) | through study completion, an average of 52 weeks | PIN-Q is a 20-item questionnaire addressing quality of life for participants with bladder disorders. Each question was answered on a scale from 0 (no, never) to 4 (all the time). The total score ranged from 0 to 80, with higher scores indicating more impact on the quality of life. |
| Change from Baseline in Patient Global Impression of Severity (PGI-S) Scale | through study completion, an average of 52 weeks | PGI-S is a 5 point scale that determines the bladder condition of a participant with 0 being really bad and 4 as really good. Higher score indicates better bladder condition. |
| Change from Baseline in Clinical Global Impression of Change (CGI-C) Scale | through study completion, an average of 52 weeks | The CGI-C scale is used to determine the degree of change in participant's overall bladder symptoms since the start of the study on Day 1. The scale will be filled by the investigator by ticking on any of the following options: very much improved, much improved, minimally improved, no change, minimally worse, much worse and very much worse. |
Countries
Belgium, Canada, Croatia, Denmark, Georgia, Jordan, Latvia, Lithuania, Malaysia, Philippines, Poland, Romania, Serbia, Slovakia, Turkey (Türkiye), United States
Contacts
CONTACTStudy Director
STUDY_DIRECTORJanet Owens-Grillo
Sumitomo Pharma America
Outcome results
None listed