A Study of Vibegron in Pediatric Participants 2 Years to Less Than (<) 18 Years of Age With NDO and on CIC

Neurogenic Detrusor Overactivity Clinical Trial

— KANGUROO  

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05491525
• Other study ID #: URO-901-3007
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: October 12, 2022
• Est. completion date: September 2027

Study information

• Verified date: June 2023
• Source: Urovant Sciences GmbH
• Contact: Study Director
• Phone: 833-876-8268
• Email: clinicaltrials[@]urovant.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 85
• Est. completion date: September 2027
• Est. primary completion date: January 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Neurogenic Detrusor Overactivity
• Urinary Bladder, Overactive

Intervention

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

Locations

Country	Name	City	State
United States	Albany Medical College	Albany	New York
United States	Duke University Medical Center	Durham	North Carolina
United States	Nemours Childrens Health, Jacksonville	Jacksonville	Florida
United States	Childrens Hospital New Orleans	New Orleans	Louisiana
United States	Children's Hospital of Orange County	Orange	California
United States	Wichita Urology Group	Wichita	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Urovant Sciences GmbH

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics		Baseline and at Week 32
Secondary	Change from Baseline in MCC		Baseline and at Week 20
Secondary	Change from Baseline in number of overactive detrusor contractions until the end of bladder filling		Baseline and at Weeks 20 and 32
Secondary	Change from Baseline in detrusor pressure at the end of bladder filling		Baseline and at Weeks 20 and 32
Secondary	Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction		Baseline and at Weeks 20 and 32
Secondary	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	Baseline and at Weeks 20 and 32
Secondary	Change from Baseline in average first morning catheterized volume		Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Secondary	Change from Baseline in average catheterized volume per catheterization		Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Secondary	Change from Baseline in average maximum catheterized volume per day		Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Secondary	Change from Baseline in average maximum catheterized daytime volume		Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Secondary	Change from Baseline in average number of leakage episodes per day		Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Secondary	Change from Baseline in estimated number of dry (leakage-free) days/ 7 days		Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Secondary	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.	Baseline and at Weeks 20, 32 and 52
Secondary	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.	Baseline and at Weeks 20, 32 and 52
Secondary	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.	Baseline and at Weeks 20, 32 and 52