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)

מגייסמקבל משתתפים כעת, לפי רשומת המרשם.

יצירת קשר עם המחקר

בקצרה

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

שלב 2 / שלב 3דרושים 71 משתתפים38 אתרים16 מדינות

קטגוריות

אונקולוגיה20%מחלות זיהומיות20%נוירולוגיה20%אימונולוגיה וראומטולוגיה20%מחלות נדירות וגנטיות20%

רשום במרשם אחד

ClinicalTrials.govNCT05491525פתיחת רשומה זו ב־ClinicalTrials.govסונכרן לפני חודשיים

מחקר יחיד יכול להיות רשום בכמה מרשמים. אנו מציגים אותו פעם אחת ומקשרים לכל רשומה שברשותנו.

מידע הניסוי מוצג כפי שפורסם על ידי המרשם, בשפת המקור שלו.

מתעניינים במחקר הזה?

התחברות או צרו חשבון כדי לרשום את התעניינותכם ולעקוב אחר המחקר הזה.

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.

תקציר

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

מצבים רפואיים

Neurogenic Detrusor Overactivity

זכאות

זכאות
מין	הכול
גילאים	2 Years – 17 Years
מתנדבים בריאים	לא

זכאות כפי שנכתבה במרשם

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.

זכאות במשפטים פשוטים

הקריטריונים של רשומה זו טרם פורקו למשפטים נפרדים. טקסט המרשם שלמעלה שלם והוא הגרסה המחייבת.

תכנון המחקר

תכנון המחקר
סוג המחקר	התערבותי
שלב	שלב 2 / שלב 3
הקצאה	לא אקראי
מודל ההתערבות	PARALLEL
מטרה ראשית	TREATMENT
הסתרה	NONE (0)
גיוס	דרושים 71 משתתפים

נותן חסות ושותפים

Urovant Sciences GmbH נותן חסות
Sumitomo Pharma America, Inc. שותפים

זרועות והתערבויות

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.

התערבויות

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

מדדי תוצאה

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

תאריכים

תאריכים
תאריך התחלה	12 באוקטובר 2022 (בפועל)
סיום ראשוני	1 במרץ 2030 (משוער)
סיום	1 בספטמבר 2030 (משוער)
פורסם לראשונה	8 באוגוסט 2022 (בפועל)
עודכן לאחרונה	15 ביוני 2026
התוצאות פורסמו	לא צוין ברשומת המרשם
המצב אומת לאחרונה	יוני 2026

בפועל פירושו שהאירוע התרחש. משוער פירושו שנותן החסות צופה אותו. שתי המשמעויות שונות זו מזו.

מיקומים

32 אתרים מגייסים

Belgium

Belgium
מתקן	עיר	מדינה או אזור	מצב
Universitair Ziekenhuis Antwerpen	Edegem	Antwerpen	בוטל
UZ Gent	Ghent	East Flanders	מגייס

Canada

Canada
מתקן	עיר	מדינה או אזור	מצב
Alberta Children's Hospital	Calgary	Alberta	מגייס
The Hospital for Sick Children	Toronto	Ontario	מגייס

Croatia

Croatia
מתקן	עיר	מדינה או אזור	מצב
University Hospital Split - KBC Split	Split		מגייס

Denmark

Denmark
מתקן	עיר	מדינה או אזור	מצב
Aarhus University Hospital - Department of Paediatrics and Adolescent Medicine	Aarhus		מגייס

Georgia

Georgia
מתקן	עיר	מדינה או אזור	מצב
JSC Evex Hospital M. lashvili Childrens Central Hospital	Tbilisi		מגייס

Jordan

Jordan
מתקן	עיר	מצב
Jordan University Hospital	Amman	מגייס
Istiklal Hospital	Amman	מגייס
Irbid Specialty Hospital	Irbid	מגייס

Latvia

Latvia
מתקן	עיר	מדינה או אזור	מצב
Childrens Clinical University Hospital	Riga		בוטל

Lithuania

Lithuania
מתקן	עיר	מדינה או אזור	מצב
Hospital of Lithuanian University of Health Sciences Kauno klinikos	Kaunas		מגייס
Vilnius University Hospital Santaros Klinikos	Vilnius		מגייס

Malaysia

Malaysia
מתקן	עיר	מדינה או אזור	מצב
Kuala Lumpur Hospital	Kuala Lumpur	Kuala Lumpur	מגייס
Hospital Umum Sarawak	Kuching	Sarawak	מגייס

Philippines

Philippines
מתקן	עיר	מדינה או אזור	מצב
National Children's Hospital	Quezon City	National Capital Region	מגייס

Poland

Poland
מתקן	עיר	מצב
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa	Bialystok	מגייס
Uniwersyteckie Centrum Kliniczne	Gdansk	מגייס
Instytut Pomnik - Centrum Zdrowia Dziecka	Warsaw	מגייס

Romania

Romania
מתקן	עיר	מדינה או אזור	מצב
Institutul Clinic Fundeni	Bucharest	Bucharest	מגייס
Maria Sklodowska Curie Childrens Clinical Emergency Hospital	Bucharest	Bucharest	מגייס

Serbia

Serbia
מתקן	עיר	מדינה או אזור	מצב
University Children's Hospital Tirsova	Belgrade		מגייס
Children and Youth Health Care Institute of Vojvodina	Novi Sad		מגייס

Slovakia

Slovakia
מתקן	עיר	מדינה או אזור	מצב
Detská fakultná nemocnica s poliklinikou/Národný ústav detských chorôb (NÚDCH)	Bratislava		מגייס
Urologická ambulancia J. BREZA MEDICAL s.r.o.	Bratislava		מגייס

Turkey (Türkiye)

Turkey (Türkiye)
מתקן	עיר	מדינה או אזור	מצב
Ankara University Faculty of Medicine Ibni Sina Hospital	Ankara	Altindag	מגייס
Mersin University, Dept. of Urology	Yenişehir		מגייס

United States

United States
מתקן	עיר	מדינה או אזור	מצב
Albany Medical College	Albany	New York	מגייס
Children's Hospital Colorado	Aurora	Colorado	מגייס
Duke University Medical Center	Durham	North Carolina	הופסק
Nemours Childrens Health, Jacksonville	Jacksonville	Florida	מגייס
Arkansas Childrens Hospital	Little Rock	Arkansas	מגייס
Childrens Hospital New Orleans	New Orleans	Louisiana	בוטל
University of Oklahoma	Oklahoma City	Oklahoma	מגייס
Children's Hospital of Orange County	Orange	California	הופסק
Oregon Health & Science University	Portland	Oregon	בוטל
SUNY Upstate Medical University	Syracuse	New York	מגייס
Wichita Urology Group	Wichita	Kansas	מגייס

מסמכי המחקר

לא צורפו מסמכים ברשומת מרשם זו.

שינויים לאורך זמן

לא נרשמו שינויים מאז שקלטנו רשומה זו לראשונה.

שינוי נרשם בכל פעם שנותן החסות מעדכן את רשומת המרשם. מצב, תאריכים, גיוס ואתרים מופיעים כאן ככל שהם משתנים.

מרשם: ClinicalTrials.gov · NCT########

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