A Study of Vibegron in Pediatric Participants 2 Years to Less Than (<) 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: 85
• Locations: 1 country
• Sites: 6

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

  Baseline and at Week 32

Secondary Outcomes (14)

• Change from Baseline in MCC

  Baseline and at Week 20

• Change from Baseline in number of overactive detrusor contractions until the end of bladder filling

  Baseline and at Weeks 20 and 32

• Change from Baseline in detrusor pressure at the end of bladder filling

  Baseline and at Weeks 20 and 32

• Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction

  Baseline and at Weeks 20 and 32

• Change from Baseline in bladder compliance (mL/cm H2O)

  Baseline and at Weeks 20 and 32

Study Arms (2)

Cohort 1: Vibegron Adolescents (12 to < 18 years)

EXPERIMENTAL

Part A: Participants aged 12 to \< 18 years will receive 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: Vibegron Children (2 to < 12 years)

EXPERIMENTAL

Part A: Participants aged 2 to \< 12 years will receive vibegron based on their weight, after DSMB review of Cohort 1, Part A data, 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: Vibegron Adolescents (12 to < 18 years); Cohort 2: Vibegron Children (2 to < 12 years)

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 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.

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 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.

Sponsors & Collaborators

• Urovant Sciences GmbH: lead

Study Sites (6)

Children's Hospital of Orange County

Orange, California, 92868-4568, 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

RECRUITING

Albany Medical College

Albany, New York, 12208, United States

RECRUITING

Duke University Medical Center

Durham, North Carolina, 27710, United States

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

Central Study Contacts

Study Director

CONTACT

833-876-8268; clinicaltrials@urovant.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• 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): January 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: July 3, 2024

Record last verified: 2024-07

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 (6)