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

Primary Purpose

Neurogenic Detrusor Overactivity

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Vibegron
Sponsored by
Urovant Sciences GmbH
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neurogenic Detrusor Overactivity focused on measuring 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

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital of Orange CountyRecruiting
  • Nemours Childrens Health, JacksonvilleRecruiting
  • Wichita Urology GroupRecruiting
  • Childrens Hospital New OrleansRecruiting
  • Albany Medical CollegeRecruiting
  • Duke University Medical CenterRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

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

Cohort 2: Vibegron Children (2 to < 12 years)

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change from Baseline in MCC
Change from Baseline in number of overactive detrusor contractions until the end of bladder filling
Change from Baseline in detrusor pressure at the end of bladder filling
Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction
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
Change from Baseline in average first morning catheterized volume
Change from Baseline in average catheterized volume per catheterization
Change from Baseline in average maximum catheterized volume per day
Change from Baseline in average maximum catheterized daytime volume
Change from Baseline in average number of leakage episodes per day
Change from Baseline in estimated number of dry (leakage-free) days/ 7 days
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.
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.
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.

Full Information

First Posted
July 28, 2022
Last Updated
June 12, 2023
Sponsor
Urovant Sciences GmbH
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1. Study Identification

Unique Protocol Identification Number
NCT05491525
Brief Title
A Study of Vibegron in Pediatric Participants 2 Years to Less Than (<) 18 Years of Age With NDO and on CIC
Acronym
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)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 12, 2022 (Actual)
Primary Completion Date
January 2027 (Anticipated)
Study Completion Date
September 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Urovant Sciences GmbH

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
85 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1: Vibegron Adolescents (12 to < 18 years)
Arm Type
Experimental
Arm Description
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.
Arm Title
Cohort 2: Vibegron Children (2 to < 12 years)
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Vibegron
Intervention Description
Participants will be administered Vibegron orally, once daily (QD)
Primary Outcome Measure Information:
Title
Change from Baseline in maximum cystometric capacity (MCC) based on bladder filling urodynamics
Time Frame
Baseline and at Week 32
Secondary Outcome Measure Information:
Title
Change from Baseline in MCC
Time Frame
Baseline and at Week 20
Title
Change from Baseline in number of overactive detrusor contractions until the end of bladder filling
Time Frame
Baseline and at Weeks 20 and 32
Title
Change from Baseline in detrusor pressure at the end of bladder filling
Time Frame
Baseline and at Weeks 20 and 32
Title
Change from Baseline in bladder filling volume until first involuntary/hyperactive detrusor contraction
Time Frame
Baseline and at Weeks 20 and 32
Title
Change from Baseline in bladder compliance (mL/cm H2O)
Description
Bladder compliance is calculated by dividing the change in volume by the change in detrusor pressure during the filling of the bladder
Time Frame
Baseline and at Weeks 20 and 32
Title
Change from Baseline in average first morning catheterized volume
Time Frame
Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Title
Change from Baseline in average catheterized volume per catheterization
Time Frame
Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Title
Change from Baseline in average maximum catheterized volume per day
Time Frame
Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Title
Change from Baseline in average maximum catheterized daytime volume
Time Frame
Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Title
Change from Baseline in average number of leakage episodes per day
Time Frame
Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Title
Change from Baseline in estimated number of dry (leakage-free) days/ 7 days
Time Frame
Baseline and at Weeks 1, 4, 8, 20, 32, 48, and 52
Title
Change from Baseline in Pediatric Incontinence Questionnaire (PIN-Q)
Description
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.
Time Frame
Baseline and at Weeks 20, 32 and 52
Title
Change from Baseline in Patient Global Impression of Severity (PGI-S) Scale
Description
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.
Time Frame
Baseline and at Weeks 20, 32 and 52
Title
Change from Baseline in Clinical Global Impression of Change (CGI-C) Scale
Description
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.
Time Frame
Baseline and at Weeks 20, 32 and 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Director
Phone
833-876-8268
Email
clinicaltrials@urovant.com
Facility Information:
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868-4568
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Antoine Khoury
Facility Name
Nemours Childrens Health, Jacksonville
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Andrew Stec
Facility Name
Wichita Urology Group
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67226
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kahlil N Saad
Facility Name
Childrens Hospital New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70118
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christopher Roth
Facility Name
Albany Medical College
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alexandra Rehfuss
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
John S. Wiener

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Urovant is committed to sharing patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Data requests will be reviewed and approved on the basis of scientific merit. All data provided will be anonymized according to applicable laws and regulations.
IPD Sharing 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.
IPD Sharing 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).

Learn more about this trial

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

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