Neurogenic Detrusor OveractivityPhase 2/3KANGUROONCT05491525
Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC
A Phase 2/3 study testing Vibegron in people with Neurogenic Detrusor Overactivity, run by Urovant Sciences GmbH at 38 sites in 16 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Neurogenic Detrusor Overactivity
- What is being tested?
- Vibegron
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 71 participants
- Where
- 38 sitesin 16 countries
- Study dates
- 12 Oct 2022to Sep 2030
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
38 sites in 16 countries. Contact the study team or a site near you to ask about taking part.
38 sites in 16 countries
United States11 sites, 7 recruiting
- Recruiting
Albany Medical College
Albany, New York, 12208
Alexandra Rehfuss
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 800045
Kyle Rove
- Terminated
Duke University Medical Center
Durham, North Carolina, 27710
- Recruiting
Nemours Childrens Health, Jacksonville
Jacksonville, Florida, 32207
Andrew Stec
- Recruiting
Arkansas Childrens Hospital
Little Rock, Arkansas, 72202
Ashay Patel
- Withdrawn
Childrens Hospital New Orleans
New Orleans, Louisiana, 70118
- Recruiting
University of Oklahoma
Oklahoma City, Oklahoma, 73104
Dominic Frimberger
- Terminated
Children's Hospital of Orange County
Orange, California, 92868-4568
- Withdrawn
Oregon Health & Science University
Portland, Oregon, 97239
- Recruiting
SUNY Upstate Medical University
Syracuse, New York, 13210
Jeffery Villanueva
- Recruiting
Wichita Urology Group
Wichita, Kansas, 67226
Kahlil N Saad
Jordan3 sites, 3 recruiting
- Recruiting
Istiklal Hospital
Amman, 11821
Mohammad Al-Zubi
- Recruiting
Jordan University Hospital
Amman, 11942
Raed Altaher
- Recruiting
Irbid Specialty Hospital
Irbid, 21110
Osama Bani-Hani
Poland3 sites, 3 recruiting
- Recruiting
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
Bialystok, 15-274
Anna Wasilewska
- Recruiting
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
Michal Maternik
- Recruiting
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, 04-736
Piotr Gastol
Belgium2 sites, 1 recruiting
- Withdrawn
Universitair Ziekenhuis Antwerpen
Edegem, Antwerpen, 2650
- Recruiting
UZ Gent
Ghent, East Flanders, 9000
Erik Van Laecke
Canada2 sites, 2 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Bryce Weber
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 1E8
Michael Chua
Lithuania2 sites, 2 recruiting
- Recruiting
Hospital of Lithuanian University of Health Sciences Kauno klinikos
Kaunas, LT-50161
Sarunas Rudaitis
- Recruiting
Vilnius University Hospital Santaros Klinikos
Vilnius, LT-08406
Beata Vincel
Malaysia2 sites, 2 recruiting
- Recruiting
Kuala Lumpur Hospital
Kuala Lumpur, Kuala Lumpur, 50586
Poongkodi Nagappan
- Recruiting
Hospital Umum Sarawak
Kuching, Sarawak, 93586
Ing Soon Ngu
Romania2 sites, 2 recruiting
- Recruiting
Institutul Clinic Fundeni
Bucharest, Bucharest, 22328
Robert Stoica
- Recruiting
Maria Sklodowska Curie Childrens Clinical Emergency Hospital
Bucharest, Bucharest, 41451
Beatrice Bunea
Serbia2 sites, 2 recruiting
- Recruiting
University Children's Hospital Tirsova
Belgrade, 11000
Dusan Parapovic
- Recruiting
Children and Youth Health Care Institute of Vojvodina
Novi Sad, 21000
Dragan Sarac
Slovakia2 sites, 2 recruiting
- Recruiting
Detská fakultná nemocnica s poliklinikou/Národný ústav detských chorôb (NÚDCH)
Bratislava, 83301
Peter Barton
- Recruiting
Urologická ambulancia J. BREZA MEDICAL s.r.o.
Bratislava, 85101
Jan Breza
Turkey (Türkiye)2 sites, 2 recruiting
- Recruiting
Ankara University Faculty of Medicine Ibni Sina Hospital
Ankara, Altindag, 6230
Berk Burgu
- Recruiting
Mersin University, Dept. of Urology
Yenişehir, 33343
Erim Erdem
Croatia1 site, 1 recruiting
- Recruiting
University Hospital Split - KBC Split
Split, 21000
Tanja Kovacevic
Denmark1 site, 1 recruiting
- Recruiting
Aarhus University Hospital - Department of Paediatrics and Adolescent Medicine
Aarhus, 8200
Kostas Kamperis
Georgia1 site, 1 recruiting
- Recruiting
JSC Evex Hospital M. lashvili Childrens Central Hospital
Tbilisi, 0159
Tinatin Davitaia
Philippines1 site, 1 recruiting
- Recruiting
National Children's Hospital
Quezon City, National Capital Region, 1102
Carlo Bisnar
About this study
Official title: 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)
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
- Conditions
- Neurogenic Detrusor Overactivity
- Being tested
- Vibegron
- 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
- Medical subject headings
- Spinal Dysraphism, Meningomyelocele, Meningocele, Spinal Cord Injuries, Myelitis, Transverse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VibegronDrug
Participants will be administered Vibegron orally, once daily (QD)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Weight >=41.5kg | Experimental | Part A: Participants will receive a dose of Vibegron based on their weight, with dose reduction based on individual clinical condition, PK, and safety/tolerability data. Participants may be dose-reduced up to 2 times.
Part B: Participants will receive a Data and Safety Monitoring Board (DSMB)-selected Vibegron dose for their weight determined from participants in their respective cohort and weight band of Part A. Receives: Drug: Vibegron |
| Cohort 2: Weight Range >=29.5 kg to <=41.4 kg | Experimental | Part A: participants will receive a dose of Vibegron based on their weight, with dose reduction based on individual clinical condition, PK, and safety/tolerability data. Participants may be dose-reduced up to 2 times.
Part B: Participants will receive a DSMB-selected Vibegron dose for their weight determined from participants in their respective cohort and weight band of Part A. Receives: Drug: Vibegron |
| Cohort 3: Weight range >=11 kg to <=29.4 kg | Experimental | Part A: Participants will receive a dose of Vibegron based on their weight, with dose reduction based on individual clinical condition, PK, and safety/tolerability data. Participants may be dose-reduced up to 2 times.
Part B: Participants will receive a DSMB-selected Vibegron dose for their weight determined from participants in their respective cohort and weight band of Part A. Receives: Drug: Vibegron |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics
Measured: Optimized Treatment Week 24
Secondary outcomes (14)
Change from Baseline in MCC
Measured: Optimized Week 12
Change from Baseline in number of overactive detrusor contractions until the end of bladder filling
Measured: Optimized Treatment Weeks 12 and 24
Change from Baseline in detrusor pressure at the end of bladder filling
Measured: Optimized Treatment Weeks 12 and 24
Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction
Measured: Optimized Treatment Weeks 12 and 24
Change from Baseline in bladder compliance (mL/cm H2O)
Bladder compliance is calculated by dividing the change in volume by the change in detrusor pressure during the filling of the bladder
Measured: Optimized Treatment Weeks 12 and 24
Change from Baseline in average first morning catheterized volume
Measured: through study completion, an average of 52 weeks
Change from Baseline in average catheterized volume per catheterization
Measured: through study completion, an average of 52 weeks
Change from Baseline in average maximum catheterized volume per day
Measured: through study completion, an average of 52 weeks
Change from Baseline in average maximum catheterized daytime volume
Measured: through study completion, an average of 52 weeks
Change from Baseline in average number of leakage episodes per day
Measured: through study completion, an average of 52 weeks
Change from Baseline in estimated number of dry (leakage-free) days/ 7 days
Measured: through study completion, an average of 52 weeks
Change from Baseline in Pediatric Incontinence Questionnaire (PIN-Q)
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.
Measured: through study completion, an average of 52 weeks
Change from Baseline in Patient Global Impression of Severity (PGI-S) Scale
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.
Measured: through study completion, an average of 52 weeks
Change from Baseline in Clinical Global Impression of Change (CGI-C) Scale
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.
Measured: through study completion, an average of 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2022
- Study start12 Oct 2022
- Primary completionMar 2030 (estimated)
- Study completionSep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurogenic Detrusor OveractivityStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 71 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Urovant Sciences GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sumitomo Pharma America, Inc.
- Responsible party
- Sponsor
- Investigators
- Janet Owens-Grillo, Study director, Sumitomo Pharma America
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05491525
- Sponsor's protocol ID
- URO-901-3007
- Acronym
- KANGUROO
- Status last verified
- Jun 2026
- First submitted
- 28 Jul 2022
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Urovant is committed to sharing patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Data requests will be reviewed and approved on the basis of scientific merit. All data provided will be anonymized according to applicable laws and regulations.