NCT05491525ClinicalTrials.gov

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, 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)

RecruitingTaking participants now, according to the registry record.

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In brief

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

Phase 2 / Phase 371 participants sought38 sites16 countries

Registered in 1 registry

ClinicalTrials.govNCT05491525Open this record at ClinicalTrials.govSynced 2 months ago

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Trial information is shown as published by the registry, in its original language.

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How this study is set up

The points below are drawn from the public registry record for this study. Each one cites the field or sentence it came from. A dash (—) means the record does not state something — not that it is missing or wrong. Many well-run studies, especially small ones, leave some of these blank.

Present: Registered before enrolment beganFirst posted 2022-08-08; recorded start 2022-10-12
Present: Has a defined primary outcomeA primary outcome measure is listed in the record
Present: The primary outcome states a time frameThe primary outcome measure records a time frame
Not stated: Participants are not randomly assignedAllocation is recorded as non-randomised
Present: Has a comparison groupThe record lists a placebo or comparator arm, or more than one study arm
Not stated: Open label (no blinding)Masking is recorded as none (open label)
Not stated: An ethics committee is not stated in the registry recordThis registry's ingested record has no ethics-committee field
Not stated: Regulatory oversight (such as an IND or IDE) is not stated in the registry recordThis registry's ingested record has no regulatory-authorisation field
Not stated: A data monitoring committee is not stated in the registry recordThis registry's ingested record has no data-monitoring-committee fieldA data monitoring committee is not required for many smaller studies, and its absence here is not unusual.
Present: No cost to participants is mentioned in the recordChecked the summary, description and eligibility text; no cost-to-participant phrase found
Not stated: No other studies from this sponsor are in this databaseCounted from the lead sponsor named in the record (Urovant Sciences GmbH)
Not stated: No posted results from this sponsor are in this database yetBased on the sponsor’s studies in this database

The record lists 1 condition.
Lead sponsor type recorded as: industry.
Intervention regulatory context: investigational, within a phased regulatory pathway.

These are facts from the public registry record, not a judgement about the study. Discuss any trial with your own doctor before taking part.

What these facts mean, and what to ask · ruleset v1

Trial stature

Three independent measures of this study, described from its registry record — not a recommendation about it. A rigorous study by investigators nobody has heard of is better evidence than a weak study led by a famous one. How these are scored.

Methodological rigourEXPLORATORY

An exploratory-stage design for a phase 3 study, judged from its ClinicalTrials.gov record.

How this score is built

Randomised allocation0/20
Non-randomised allocation
Blinding0/20
Open-label
Control arm0/15
No comparator arm stated in the record
Primary-outcome specificity10/10
Named primary outcome with a defined time frame
Endpoint type3/10
Surrogate or intermediate endpoint (conservative default)
Multi-centre8/8
Multi-centre: 38 sites
Data monitoring committee7/7
A data monitoring committee is in place
Prospective registration5/5
Registered before the study start date
Protocol / SAP posted0/5
No protocol or SAP posted to the registry

ScaleSMALL

A small study, international in scope: 71 participants (target), run at 38 sites, across 16 countries.

How this score is built

Enrolment17/40
71 participants (target)
Site count19/25
38 sites
Country count15/15
16 countries
Planned duration10/10
Planned over about 96 months
Sponsor scale0/10
Urovant Sciences GmbH has led 1 trials in our corpus

Investigator standingUNKNOWN

We have no verifiable track record for the investigator named on this trial. That is common for early-career investigators and for records held outside ClinicalTrials.gov — it is not a negative signal.

How this score is built

Investigator standing0/100
No investigator recorded in the registry for this trial

These describe the registry record only, and today we hold ClinicalTrials.gov data. Absent fields lower a score, and absence often reflects registration practice rather than study quality. A high-stature trial is not necessarily safer or a better choice for you — enrolling in a large definitive trial can mean a higher chance of receiving placebo, while a small early-phase study may be the only route to a new therapy.

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

Conditions

Neurogenic Detrusor Overactivity

Eligibility

Eligibility
Sex	All
Ages	2 Years – 17 Years
Healthy volunteers	No

Eligibility as written in the registry

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.

Eligibility in plain statements

This record's criteria have not been broken into separate statements yet. The registry text above is complete and is the authoritative version.

Study design

Study design
Study type	Interventional
Phase	Phase 2 / Phase 3
Allocation	Non-randomised
Intervention model	PARALLEL
Primary purpose	TREATMENT
Masking	NONE (0)
Enrolment	71 participants sought

Sponsor and collaborators

Urovant Sciences GmbH Sponsor
Sumitomo Pharma America, Inc. Collaborators

Arms and interventions

Cohort 1: Weight >=41.5kgEXPERIMENTAL
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.
Cohort 2: Weight Range >=29.5 kg to <=41.4 kgEXPERIMENTAL
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.
Cohort 3: Weight range >=11 kg to <=29.4 kgEXPERIMENTAL
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.

Interventions

Drug Vibegron
Participants will be administered Vibegron orally, once daily (QD)

Outcome measures

Primary outcome
Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics
Time frame Optimized Treatment Week 24
Secondary outcome
Change from Baseline in MCC
Time frame Optimized Week 12
Secondary outcome
Change from Baseline in number of overactive detrusor contractions until the end of bladder filling
Time frame Optimized Treatment Weeks 12 and 24
Secondary outcome
Change from Baseline in detrusor pressure at the end of bladder filling
Time frame Optimized Treatment Weeks 12 and 24
Secondary outcome
Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction
Time frame Optimized Treatment Weeks 12 and 24
Secondary outcome
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
Time frame Optimized Treatment Weeks 12 and 24
Secondary outcome
Change from Baseline in average first morning catheterized volume
Time frame through study completion, an average of 52 weeks
Secondary outcome
Change from Baseline in average catheterized volume per catheterization
Time frame through study completion, an average of 52 weeks
Secondary outcome
Change from Baseline in average maximum catheterized volume per day
Time frame through study completion, an average of 52 weeks
Secondary outcome
Change from Baseline in average maximum catheterized daytime volume
Time frame through study completion, an average of 52 weeks
Secondary outcome
Change from Baseline in average number of leakage episodes per day
Time frame through study completion, an average of 52 weeks
Secondary outcome
Change from Baseline in estimated number of dry (leakage-free) days/ 7 days
Time frame through study completion, an average of 52 weeks
Secondary outcome
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.
Time frame through study completion, an average of 52 weeks
Secondary outcome
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.
Time frame through study completion, an average of 52 weeks
Secondary outcome
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.
Time frame through study completion, an average of 52 weeks

Dates

Dates
Start date	October 12, 2022 (actual)
Primary completion	March 1, 2030 (estimated)
Completion	September 1, 2030 (estimated)
First posted	August 8, 2022 (actual)
Last updated	June 15, 2026
Results posted	Not stated in the registry record
Status last verified	June 2026

Actual means the event happened. Estimated means the sponsor expects it. The two mean different things.

Locations

32 sites are recruiting

Belgium

Belgium
Facility	City	State or region	Status
Universitair Ziekenhuis Antwerpen	Edegem	Antwerpen	Withdrawn
UZ Gent	Ghent	East Flanders	Recruiting

Canada

Canada
Facility	City	State or region	Status
Alberta Children's Hospital	Calgary	Alberta	Recruiting
The Hospital for Sick Children	Toronto	Ontario	Recruiting

Croatia

Croatia
Facility	City	State or region	Status
University Hospital Split - KBC Split	Split		Recruiting

Denmark

Denmark
Facility	City	State or region	Status
Aarhus University Hospital - Department of Paediatrics and Adolescent Medicine	Aarhus		Recruiting

Georgia

Georgia
Facility	City	State or region	Status
JSC Evex Hospital M. lashvili Childrens Central Hospital	Tbilisi		Recruiting

Jordan

Jordan
Facility	City	Status
Jordan University Hospital	Amman	Recruiting
Istiklal Hospital	Amman	Recruiting
Irbid Specialty Hospital	Irbid	Recruiting

Latvia

Latvia
Facility	City	State or region	Status
Childrens Clinical University Hospital	Riga		Withdrawn

Lithuania

Lithuania
Facility	City	State or region	Status
Hospital of Lithuanian University of Health Sciences Kauno klinikos	Kaunas		Recruiting
Vilnius University Hospital Santaros Klinikos	Vilnius		Recruiting

Malaysia

Malaysia
Facility	City	State or region	Status
Kuala Lumpur Hospital	Kuala Lumpur	Kuala Lumpur	Recruiting
Hospital Umum Sarawak	Kuching	Sarawak	Recruiting

Philippines

Philippines
Facility	City	State or region	Status
National Children's Hospital	Quezon City	National Capital Region	Recruiting

Poland

Poland
Facility	City	Status
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa	Bialystok	Recruiting
Uniwersyteckie Centrum Kliniczne	Gdansk	Recruiting
Instytut Pomnik - Centrum Zdrowia Dziecka	Warsaw	Recruiting

Romania

Romania
Facility	City	State or region	Status
Institutul Clinic Fundeni	Bucharest	Bucharest	Recruiting
Maria Sklodowska Curie Childrens Clinical Emergency Hospital	Bucharest	Bucharest	Recruiting

Serbia

Serbia
Facility	City	State or region	Status
University Children's Hospital Tirsova	Belgrade		Recruiting
Children and Youth Health Care Institute of Vojvodina	Novi Sad		Recruiting

Slovakia

Slovakia
Facility	City	State or region	Status
Detská fakultná nemocnica s poliklinikou/Národný ústav detských chorôb (NÚDCH)	Bratislava		Recruiting
Urologická ambulancia J. BREZA MEDICAL s.r.o.	Bratislava		Recruiting

Turkey (Türkiye)

Turkey (Türkiye)
Facility	City	State or region	Status
Ankara University Faculty of Medicine Ibni Sina Hospital	Ankara	Altindag	Recruiting
Mersin University, Dept. of Urology	Yenişehir		Recruiting

United States

United States
Facility	City	State or region	Status
Albany Medical College	Albany	New York	Recruiting
Children's Hospital Colorado	Aurora	Colorado	Recruiting
Duke University Medical Center	Durham	North Carolina	Terminated
Nemours Childrens Health, Jacksonville	Jacksonville	Florida	Recruiting
Arkansas Childrens Hospital	Little Rock	Arkansas	Recruiting
Childrens Hospital New Orleans	New Orleans	Louisiana	Withdrawn
University of Oklahoma	Oklahoma City	Oklahoma	Recruiting
Children's Hospital of Orange County	Orange	California	Terminated
Oregon Health & Science University	Portland	Oregon	Withdrawn
SUNY Upstate Medical University	Syracuse	New York	Recruiting
Wichita Urology Group	Wichita	Kansas	Recruiting

Study documents

No documents are linked in this registry record.

Changes over time

No changes have been recorded since we first ingested this record.

A change is recorded each time the sponsor updates the registry record. Status, dates, enrolment and sites appear here as they move.

Registry: ClinicalTrials.gov · NCT########

https://xmedica.io/trials/ctgov/NCT05491525

Categories

Registered in 1 registry

Interested in this study?

How this study is set up

Summary

Conditions

Eligibility

Eligibility as written in the registry

Eligibility in plain statements

Study design

Sponsor and collaborators

Arms and interventions

Interventions

Outcome measures

Dates

Locations

Belgium

Canada

Croatia

Denmark

Georgia

Jordan

Latvia

Lithuania

Malaysia

Philippines

Poland

Romania

Serbia

Slovakia

Turkey (Türkiye)

United States

Study documents

Changes over time