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)

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En resumen

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

Fase 2 / Fase 371 participantes buscados38 centros16 países

Categorías

Oncología20%Enfermedades infecciosas20%Neurología20%Inmunología y reumatología20%Enfermedades raras y genéticas20%

Registrado en 1 registro

ClinicalTrials.govNCT05491525Abrir este registro en ClinicalTrials.govSincronizado hace 2 meses

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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.

Resumen

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

Afecciones

Neurogenic Detrusor Overactivity

Elegibilidad

Elegibilidad
Sexo	Todos
Edades	2 Years – 17 Years
Voluntarios sanos	No

Elegibilidad tal como figura en el registro

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.

Elegibilidad en frases sencillas

Los criterios de este registro aún no se han desglosado en frases separadas. El texto del registro anterior está completo y es la versión autorizada.

Diseño del estudio

Diseño del estudio
Tipo de estudio	De intervención
Fase	Fase 2 / Fase 3
Asignación	No aleatorizado
Modelo de intervención	PARALLEL
Propósito principal	TREATMENT
Enmascaramiento	NONE (0)
Inscripción	71 participantes buscados

Patrocinador y colaboradores

Urovant Sciences GmbH Patrocinador
Sumitomo Pharma America, Inc. Colaboradores

Grupos e intervenciones

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.

Intervenciones

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

Variables de resultado

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

Fechas

Fechas
Fecha de inicio	12 de octubre de 2022 (real)
Finalización principal	1 de marzo de 2030 (estimada)
Finalización	1 de septiembre de 2030 (estimada)
Primera publicación	8 de agosto de 2022 (real)
Última actualización	15 de junio de 2026
Resultados publicados	No indicado en el registro
Estado verificado por última vez	junio de 2026

Real significa que el evento ocurrió. Estimada significa que el patrocinador lo prevé. Ambas cosas significan algo distinto.

Ubicaciones

32 centros están en reclutamiento

Belgium

Belgium
Centro	Ciudad	Estado o región	Estado
Universitair Ziekenhuis Antwerpen	Edegem	Antwerpen	Retirado
UZ Gent	Ghent	East Flanders	En reclutamiento

Canada

Canada
Centro	Ciudad	Estado o región	Estado
Alberta Children's Hospital	Calgary	Alberta	En reclutamiento
The Hospital for Sick Children	Toronto	Ontario	En reclutamiento

Croatia

Croatia
Centro	Ciudad	Estado o región	Estado
University Hospital Split - KBC Split	Split		En reclutamiento

Denmark

Denmark
Centro	Ciudad	Estado o región	Estado
Aarhus University Hospital - Department of Paediatrics and Adolescent Medicine	Aarhus		En reclutamiento

Georgia

Georgia
Centro	Ciudad	Estado o región	Estado
JSC Evex Hospital M. lashvili Childrens Central Hospital	Tbilisi		En reclutamiento

Jordan

Jordan
Centro	Ciudad	Estado
Jordan University Hospital	Amman	En reclutamiento
Istiklal Hospital	Amman	En reclutamiento
Irbid Specialty Hospital	Irbid	En reclutamiento

Latvia

Latvia
Centro	Ciudad	Estado o región	Estado
Childrens Clinical University Hospital	Riga		Retirado

Lithuania

Lithuania
Centro	Ciudad	Estado o región	Estado
Hospital of Lithuanian University of Health Sciences Kauno klinikos	Kaunas		En reclutamiento
Vilnius University Hospital Santaros Klinikos	Vilnius		En reclutamiento

Malaysia

Malaysia
Centro	Ciudad	Estado o región	Estado
Kuala Lumpur Hospital	Kuala Lumpur	Kuala Lumpur	En reclutamiento
Hospital Umum Sarawak	Kuching	Sarawak	En reclutamiento

Philippines

Philippines
Centro	Ciudad	Estado o región	Estado
National Children's Hospital	Quezon City	National Capital Region	En reclutamiento

Poland

Poland
Centro	Ciudad	Estado
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa	Bialystok	En reclutamiento
Uniwersyteckie Centrum Kliniczne	Gdansk	En reclutamiento
Instytut Pomnik - Centrum Zdrowia Dziecka	Warsaw	En reclutamiento

Romania

Romania
Centro	Ciudad	Estado o región	Estado
Institutul Clinic Fundeni	Bucharest	Bucharest	En reclutamiento
Maria Sklodowska Curie Childrens Clinical Emergency Hospital	Bucharest	Bucharest	En reclutamiento

Serbia

Serbia
Centro	Ciudad	Estado o región	Estado
University Children's Hospital Tirsova	Belgrade		En reclutamiento
Children and Youth Health Care Institute of Vojvodina	Novi Sad		En reclutamiento

Slovakia

Slovakia
Centro	Ciudad	Estado o región	Estado
Detská fakultná nemocnica s poliklinikou/Národný ústav detských chorôb (NÚDCH)	Bratislava		En reclutamiento
Urologická ambulancia J. BREZA MEDICAL s.r.o.	Bratislava		En reclutamiento

Turkey (Türkiye)

Turkey (Türkiye)
Centro	Ciudad	Estado o región	Estado
Ankara University Faculty of Medicine Ibni Sina Hospital	Ankara	Altindag	En reclutamiento
Mersin University, Dept. of Urology	Yenişehir		En reclutamiento

United States

United States
Centro	Ciudad	Estado o región	Estado
Albany Medical College	Albany	New York	En reclutamiento
Children's Hospital Colorado	Aurora	Colorado	En reclutamiento
Duke University Medical Center	Durham	North Carolina	Finalizado antes de tiempo
Nemours Childrens Health, Jacksonville	Jacksonville	Florida	En reclutamiento
Arkansas Childrens Hospital	Little Rock	Arkansas	En reclutamiento
Childrens Hospital New Orleans	New Orleans	Louisiana	Retirado
University of Oklahoma	Oklahoma City	Oklahoma	En reclutamiento
Children's Hospital of Orange County	Orange	California	Finalizado antes de tiempo
Oregon Health & Science University	Portland	Oregon	Retirado
SUNY Upstate Medical University	Syracuse	New York	En reclutamiento
Wichita Urology Group	Wichita	Kansas	En reclutamiento

Documentos del estudio

No hay documentos enlazados en este registro.

Cambios a lo largo del tiempo

No se han registrado cambios desde que incorporamos este registro por primera vez.

Se registra un cambio cada vez que el patrocinador actualiza el registro. El estado, las fechas, la inscripción y los centros aparecen aquí a medida que cambian.

Registro: ClinicalTrials.gov · NCT########

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