Evaluating Vibegron for Children with Neurogenic Detrusor Overactivity

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)

This study is testing if vibegron is safe and effective for children aged 2 to under 18 with neurogenic detrusor overactivity who use catheters.

Quick facts

• Phase: PHASE2; PHASE3
• Study type: Interventional
• Enrollment: 85 (estimated)
• Ages: 2 Years to 17 Years
• Sex: All
• Sponsor: Urovant Sciences GmbH (industry)
• Locations: 6 sites (Orange, California and 5 other locations)
• Trial ID: NCT05491525 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and effectiveness of vibegron in pediatric patients aged 2 to less than 18 years who have been diagnosed with neurogenic detrusor overactivity (NDO) and are using clean intermittent catheterization (CIC). Participants will be divided into two cohorts based on age and weight, and will receive vibegron to evaluate its pharmacokinetics and therapeutic outcomes. The study will monitor participants for a range of safety and efficacy endpoints over the course of the trial.

Who should consider this trial

Good fit: Ideal candidates are children aged 2 to less than 18 years with a diagnosis of NDO who are currently using CIC.

Not a fit: Patients with conditions such as cerebral palsy or uncontrolled epilepsy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve bladder function and quality of life for children suffering from NDO.

How similar studies have performed: Previous studies have shown promise with beta-3 adrenergic receptor agonists in treating bladder dysfunction, indicating potential for success with this approach.

Eligibility criteria

Inclusion Criteria:

* Male or female participants, age 2 years to \< 18 years at the Screening Visit. Participants age 12 to \< 18 years (Cohort 1) must weigh at least 29.5 kilograms (kg). Participants age 2 to \< 12 years (Cohort 2) must weigh at least 11 kg.
* 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 urethral dilatation or has had 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 fecal impaction or a history of fecal impaction requiring hospitalization or ambulatory surgical treatment in the 3 months prior to the Screening Visit.
* Participant has a urinary indwelling catheter in the 4 weeks prior to the Screening Visit.
* Participant has moderate to severe dilating vesicoureteral reflux (Grade III 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.

Where this trial is running

Orange, California and 5 other locations

• Children's Hospital of Orange County — Orange, California, United States (RECRUITING)
• Nemours Childrens Health, Jacksonville — Jacksonville, Florida, United States (RECRUITING)
• Wichita Urology Group — Wichita, Kansas, United States (RECRUITING)
• Childrens Hospital New Orleans — New Orleans, Louisiana, United States (RECRUITING)
• Albany Medical College — Albany, New York, United States (RECRUITING)
• Duke University Medical Center — Durham, North Carolina, United States (RECRUITING)

Study contacts

• Study coordinator: Study Director

Conditions: Neurogenic Detrusor Overactivity, Vibegron, Clean Intermittent Catheterization, Beta-3 Adrenergic Receptor Agonist, Maximum Cystometric Capacity, Spinal Dysraphism, Spina Bifida, Myelomeningocele