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

NCT05491525 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: URO-901-3007
• Study Type: interventional
• Target: 71
• Locations: 16 countries
• Sites: 38

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

Conditions

Neurogenic Detrusor Overactivity

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

Outcome Measures

Primary Outcomes (1)

• Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics

  Optimized Treatment Week 24

Secondary Outcomes (14)

• 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

Study Arms (3)

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.

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.

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.

Drug: Vibegron

Interventions

Vibegron DRUG

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

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

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Urovant Sciences GmbH: lead
• Sumitomo Pharma America, Inc.: collaborator

Study Sites (38)

Arkansas Childrens Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

Children's Hospital of Orange County

Orange, California, 92868-4568, United States

TERMINATED

Children's Hospital Colorado

Aurora, Colorado, 800045, United States

RECRUITING

Nemours Childrens Health, Jacksonville

Jacksonville, Florida, 32207, United States

RECRUITING

Wichita Urology Group

Wichita, Kansas, 67226, United States

RECRUITING

Childrens Hospital New Orleans

New Orleans, Louisiana, 70118, United States

WITHDRAWN

Albany Medical College

Albany, New York, 12208, United States

RECRUITING

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

RECRUITING

Duke University Medical Center

Durham, North Carolina, 27710, United States

TERMINATED

University of Oklahoma

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

WITHDRAWN

Universitair Ziekenhuis Antwerpen

Edegem, Antwerpen, 2650, Belgium

WITHDRAWN

UZ Gent

Ghent, East Flanders, 9000, Belgium

RECRUITING

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 1E8, Canada

RECRUITING

University Hospital Split - KBC Split

Split, 21000, Croatia

RECRUITING

Aarhus University Hospital - Department of Paediatrics and Adolescent Medicine

Aarhus, 8200, Denmark

RECRUITING

JSC Evex Hospital M. lashvili Childrens Central Hospital

Tbilisi, 0159, Georgia

RECRUITING

Istiklal Hospital

Amman, 11821, Jordan

RECRUITING

Jordan University Hospital

Amman, 11942, Jordan

RECRUITING

Irbid Specialty Hospital

Irbid, 21110, Jordan

RECRUITING

Childrens Clinical University Hospital

Riga, LV-1004, Latvia

WITHDRAWN

Hospital of Lithuanian University of Health Sciences Kauno klinikos

Kaunas, LT-50161, Lithuania

RECRUITING

Vilnius University Hospital Santaros Klinikos

Vilnius, LT-08406, Lithuania

RECRUITING

Kuala Lumpur Hospital

Kuala Lumpur, Kuala Lumpur, 50586, Malaysia

RECRUITING

Hospital Umum Sarawak

Kuching, Sarawak, 93586, Malaysia

RECRUITING

National Children's Hospital

Quezon City, National Capital Region, 1102, Philippines

RECRUITING

Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa

Bialystok, 15-274, Poland

RECRUITING

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

RECRUITING

Instytut Pomnik - Centrum Zdrowia Dziecka

Warsaw, 04-736, Poland

RECRUITING

Institutul Clinic Fundeni

Bucharest, Bucharest, 22328, Romania

RECRUITING

Maria Sklodowska Curie Childrens Clinical Emergency Hospital

Bucharest, Bucharest, 41451, Romania

RECRUITING

University Children's Hospital Tirsova

Belgrade, 11000, Serbia

RECRUITING

Children and Youth Health Care Institute of Vojvodina

Novi Sad, 21000, Serbia

RECRUITING

Detská fakultná nemocnica s poliklinikou/Národný ústav detských chorôb (NÚDCH)

Bratislava, 83301, Slovakia

RECRUITING

Urologická ambulancia J. BREZA MEDICAL s.r.o.

Bratislava, 85101, Slovakia

RECRUITING

Ankara University Faculty of Medicine Ibni Sina Hospital

Ankara, Altindag, 6230, Turkey (Türkiye)

RECRUITING

Mersin University, Dept. of Urology

Yenişehir, 33343, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Spinal Dysraphism; Meningomyelocele; Meningocele; Spinal Cord Injuries; Myelitis, Transverse

Interventions

N-(4-((5-(hydroxy(phenyl)methyl)pyrrolidin-2-yl)methyl)phenyl)-4-oxo-4,6,7,8-tetrahydropyrrolo(1,2-a)pyrimidine-6-carboxamide

Condition Hierarchy (Ancestors)

Neural Tube Defects; Nervous System Malformations; Nervous System Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hernia; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Spinal Cord Diseases; Central Nervous System Diseases; Trauma, Nervous System; Wounds and Injuries; Myelitis; Central Nervous System Infections; Infections; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Neurodegenerative Diseases; Neuroinflammatory Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Janet Owens-Grillo

  Sumitomo Pharma America

  STUDY DIRECTOR

Central Study Contacts

Study Director

CONTACT

833-876-8268; clinicaltrials@us.sumitomo-pharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2022
• First Posted: August 8, 2022
• Study Start: October 12, 2022
• Primary Completion (Estimated): March 1, 2030
• Study Completion (Estimated): September 1, 2030
• Last Updated: June 15, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: The data will be made available within 24 months after study completion and will be accessible for a time frame appropriate for the approved proposal.
• Access Criteria: Access to these clinical trial data can be requested by emailing medinfo@urovant.com and will be provided following Urovant review and approval of a research proposal and execution of a Data Sharing Agreement (DSA).

Locations

US (11); SL (2); MY (2); CA (2); PL (3); LI (2); JO (3); CR (1); LA (1); PH (1); DK (1); SE (2); BE (2); GE (1); TU (2); RO (2)