Vibegron: Participants will be administered Vibegron orally, once daily (QD)
Study 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
Eligibility
Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics — Optimized Treatment Week 24
Trial sites (38)
Facility
City
Region
Status
Arkansas Childrens Hospital
Little Rock
Arkansas
Recruiting
Children's Hospital of Orange County
Orange
California
Terminated
Children's Hospital Colorado
Aurora
Colorado
Recruiting
Nemours Childrens Health, Jacksonville
Jacksonville
Florida
Recruiting
Wichita Urology Group
Wichita
Kansas
Recruiting
Childrens Hospital New Orleans
New Orleans
Louisiana
Withdrawn
Albany Medical College
Albany
New York
Recruiting
SUNY Upstate Medical University
Syracuse
New York
Recruiting
Duke University Medical Center
Durham
North Carolina
Terminated
University of Oklahoma
Oklahoma City
Oklahoma
Recruiting
Oregon Health & Science University
Portland
Oregon
Withdrawn
Universitair Ziekenhuis Antwerpen
Edegem
Antwerpen
Withdrawn
UZ Gent
Ghent
East Flanders
Recruiting
Alberta Children's Hospital
Calgary
Alberta
Recruiting
The Hospital for Sick Children
Toronto
Ontario
Recruiting
University Hospital Split - KBC Split
Split
Croatia
Recruiting
Aarhus University Hospital - Department of Paediatrics and Adolescent Medicine
Aarhus
Denmark
Recruiting
JSC Evex Hospital M. lashvili Childrens Central Hospital
Tbilisi
Georgia
Recruiting
Istiklal Hospital
Amman
Jordan
Recruiting
Jordan University Hospital
Amman
Jordan
Recruiting
Irbid Specialty Hospital
Irbid
Jordan
Recruiting
Childrens Clinical University Hospital
Riga
Latvia
Withdrawn
Hospital of Lithuanian University of Health Sciences Kauno klinikos
Kaunas
Lithuania
Recruiting
Vilnius University Hospital Santaros Klinikos
Vilnius
Lithuania
Recruiting
Kuala Lumpur Hospital
Kuala Lumpur
Kuala Lumpur
Recruiting
Hospital Umum Sarawak
Kuching
Sarawak
Recruiting
National Children's Hospital
Quezon City
National Capital Region
Recruiting
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
Bialystok
Poland
Recruiting
Uniwersyteckie Centrum Kliniczne
Gdansk
Poland
Recruiting
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw
Poland
Recruiting
Institutul Clinic Fundeni
Bucharest
Bucharest
Recruiting
Maria Sklodowska Curie Childrens Clinical Emergency Hospital
Bucharest
Bucharest
Recruiting
University Children's Hospital Tirsova
Belgrade
Serbia
Recruiting
Children and Youth Health Care Institute of Vojvodina
Novi Sad
Serbia
Recruiting
Detská fakultná nemocnica s poliklinikou/Národný ústav detských chorôb (NÚDCH)
Bratislava
Slovakia
Recruiting
Urologická ambulancia J. BREZA MEDICAL s.r.o.
Bratislava
Slovakia
Recruiting
Ankara University Faculty of Medicine Ibni Sina Hospital
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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