Clinical trial • Phase II/III • Other
VIBEGRON for Neurogenic Detrusor Overactivity (NDO) | Neurogenic bladder
Phase II/III trial of VIBEGRON for Neurogenic Detrusor Overactivity (NDO) | Neurogenic bladder.
Overview
- Trial Therapeutic Area
- Other
- Trial Disease
- Neurogenic Detrusor Overactivity (NDO) | Neurogenic bladder
- Trial Stage
- Phase II/III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 14-06-2024
- First CTIS Authorization Date
- 22-07-2024
Trial design
open-label, none/not specified (baseline-controlled design; no active comparator arm specified), adaptive Phase II/III trial across 17 sites in Lithuania, Belgium, Denmark and others.
- Open Label
- Yes
- Comparator
- None/Not specified (baseline-controlled design; no active comparator arm specified)
- Adaptive
- Yes
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 53
- Trial Duration For Participant
- 378
Stratification factors
- age cohort (2 to <12 vs 12 to <18)
- weight within cohort
Eligibility
Recruits 53 paediatric patients.
- Pregnancy Exclusion
- 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.
- Vulnerable Population
- The trial enrols pediatric participants (age 2 years to <18 years). Vulnerable population handling includes age-specific assent and parent/guardian consent processes: subject information sheets, parent/guardian informed consent forms and child assent forms are provided (age-specific forms present for 2-4, 5-11, 12-17 years and adolescent assent). Multiple language versions and parent/legal guardian ICFs are provided. Consent is obtained from parent(s)/legal guardian(s); assent is obtained from children per age-appropriate assent forms as documented in the submitted ICF/assent materials.
Inclusion criteria
- {"criterion_text":"- 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\n- 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.\n- 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
- {"criterion_text":"- Participant has cerebral palsy, uncontrolled epilepsy, diabetes insipidus, or Stage 2 hypertension\n- 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.\n- 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.\n- Participant has a urinary indwelling catheter in the 4 weeks prior to the Screening Visit\n- Participant has moderate to severe dilating vesicoureteral reflux (Grade III to V) or severe renal failure.\n- Participant started electrostimulation/neuromodulation therapy in the 4 weeks before the Screening Visit, or is expected to start this therapy during the study period.\n- Participant has participated in another clinical trial and/or has taken an investigational drug within 4 weeks prior to the Screening Visit.\n- Participant is unable, or parent/caregiver is not willing, to washout any medication for the management of NDO.\n- 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.\n- 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.\n- Participant has an active malignancy in the 12 months prior to the Screening Visit.\n- Participant has been administered intravesical botulinum toxin within 9 months prior to the Screening Visit and should remain off this therapy during the study.\n- Participant is taking digoxin or lithium within 10 days prior to Screening Visit or plans to start taking either during the study.\n- Participant currently uses or plans to use a baclofen pump during the study.\n- Participant has urethral dilatation or has had urethral surgery in the 3 months prior to the Screening Visit\n- Participant has undergone bladder augmentation surgery\n- 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.\n- 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."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline at Study Week 32 (Optimized Treatment Week 24) in MCC based on filling urodynamics","definition_or_measurement_approach":"Maximum cystometric capacity (MCC) measured by filling urodynamics; endpoint is change from baseline measured at Study Week 32 (Optimized Treatment Week 24)."}
Secondary endpoints
- {"endpoint_text":"- Secondary Efficacy Endpoints: Based on Urodynamics: - Change from baseline at Study Week 20 (Optimized Treatment Week 12) in MCC - Change from baseline at Study Weeks 20 and 32 (Optimized Treatment Week 12 and 24) in: Number of overactive detrusor contractions until end of filling; Detrusor pressure at end of filling; Filling volume until first involuntary/hyperactive detrusor contraction; Bladder compliance","definition_or_measurement_approach":"Urodynamic-based measures: change from baseline in MCC at specified weeks; counts of overactive detrusor contractions, detrusor pressure at end of filling, filling volume to first involuntary contraction, and bladder compliance assessed by filling urodynamics at Study Weeks 20 and 32."}
- {"endpoint_text":"- Secondary Efficacy Endpoints: Based on Bladder Diary: - Change from baseline at Study Weeks 1, 4, 8, 20, 32, 48, and 52 in: average first morning catheterized volume; Average catheterized volume per catheterization; average maximum catheterized volume per day; Average maximum catheterized daytime volume; Average number of leakage episodes per day; Estimated number of dry days/7 days","definition_or_measurement_approach":"Bladder diary-based measures recorded by caregivers/subjects at specified study weeks: volumes per catheterization, maximum volumes, leakage episodes per day, and estimated dry days per week; change from baseline at listed timepoints."}
- {"endpoint_text":"- Secondary Efficacy Endpoints: Based on Questionnaires: - Change from baseline at Study Weeks 20, 32, and 52 in PIN-Q - Change from baseline at Study Weeks 20, 32, and 52 in PGI-S Scale - CGI-C Scale assessment at Study Weeks 20, 32, and 52.","definition_or_measurement_approach":"Questionnaire-based endpoints: Change from baseline in Pediatric Incontinence Questionnaire (PIN-Q), Patient Global Impression of Severity (PGI-S) at specified weeks; Clinical Global Impression of Change (CGI-C) assessed at specified weeks."}
- {"endpoint_text":"- Safety and Tolerability: - Incidence of AEs - Incidence of AESIs - Upper urinary tract ultrasound assessment - eGFR - Vital signs measured at clinic visits: pulse rate, systolic and diastolic blood pressure - 12-lead ECG, centrally read","definition_or_measurement_approach":"Safety endpoints include adverse event incidence (AEs), adverse events of special interest (AESIs), upper urinary tract ultrasound findings, estimated glomerular filtration rate (eGFR), clinic vital signs (pulse, systolic/diastolic BP), and centrally read 12-lead ECG."}
- {"endpoint_text":"- Pharmacokinetics: - PK of vibegron in plasma: Cmax, tmax, AUC(0-24h), Ctrough, t1/2, and CL/F","definition_or_measurement_approach":"PK endpoints: plasma PK parameters (Cmax, tmax, AUC0-24h, Ctrough, t1/2, CL/F) after multiple-dose administration measured per PK sampling schedule."}
Recruitment
- Planned Sample Size
- 53
- Recruitment Window Months
- 61
- Consent Approach
- Parent(s)/legal guardian(s) provide informed consent. Age-appropriate assent is obtained from child participants using dedicated assent forms (age group-specific forms present for 2-4 years, 5-11 years, 12-17 years/adolescents). Subject information sheets, parent/guardian ICFs, and assent documents are provided in multiple languages as documented in the submission (examples include English and local language versions such as Lithuanian, Russian, Croatian, Danish, Romanian, Slovak, Polish, Dutch, French, Latvian).
Geography
- Total Number Of Sites
- 17
- Total Number Of Participants
- 48
Lithuania
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 24-07-2024
- Processing Time Days
- 26
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- Lietuvos sveikatos mokslu universiteto ligonine Kauno klinikos
- Principal Investigator Name
- Sarunas Rudaitis
- Principal Investigator Email
- rsarunas@gmail.com
- Contact Person Name
- Sarunas Rudaitis
- Contact Person Email
- rsarunas@gmail.com
- Site Name
- Vilniaus Universiteto Ligonine Santaros Klinikos Vsi
- Principal Investigator Name
- Beata Vincel
- Principal Investigator Email
- beata.vincel@santa.lt
- Contact Person Name
- Beata Vincel
- Contact Person Email
- beata.vincel@santa.lt
Belgium
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Urology
- Principal Investigator Name
- Erik Van Laecke
- Principal Investigator Email
- erik.vanlaecke@uzgent.be
- Contact Person Name
- Erik Van Laecke
- Contact Person Email
- erik.vanlaecke@uzgent.be
- Site Name
- Antwerp University Hospital
- Department Name
- Urology
- Principal Investigator Name
- Gunter De Win
- Principal Investigator Email
- gunter.dewin@uza.be
- Contact Person Name
- Gunter De Win
- Contact Person Email
- gunter.dewin@uza.be
Denmark
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Region Midtjylland
- Department Name
- Department of Paediatrics and Adolescent Medicine
- Principal Investigator Name
- Konstantinos Kamperis
- Principal Investigator Email
- konskamp@rn.dk
- Contact Person Name
- Konstantinos Kamperis
- Contact Person Email
- konskamp@rn.dk
Romania
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Spitalul Clinic de Urgenta pentru Copii “Marie Sklodowska Curie”
- Department Name
- Pediatric Surgery
- Principal Investigator Name
- Beatrice Bunea
- Principal Investigator Email
- beatricemihaelabunea@gmail.com
- Contact Person Name
- Beatrice Bunea
- Contact Person Email
- beatricemihaelabunea@gmail.com
- Site Name
- Institutul Clinic Fundeni
- Department Name
- Urology
- Principal Investigator Name
- Robert Stoica
- Principal Investigator Email
- stoicarobert@gmail.com
- Contact Person Name
- Robert Stoica
- Contact Person Email
- stoicarobert@gmail.com
Latvia
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Bernu Kliniska Universitates Slimnica VSIA
- Principal Investigator Name
- Ainars Gilis
- Principal Investigator Email
- ainars.gilis@bkus.lv
- Contact Person Name
- Ainars Gilis
- Contact Person Email
- ainars.gilis@bkus.lv
Croatia
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Children's Hospital Zagreb
- Department Name
- Department of Pediatrics
- Principal Investigator Name
- Slaven Abdovic
- Principal Investigator Email
- sabdovic@mefhr.org
- Contact Person Name
- Slaven Abdovic
- Contact Person Email
- sabdovic@mefhr.org
- Site Name
- KBC Split
- Department Name
- Department of Pediatrics
- Principal Investigator Name
- Tanja Kovacevic
- Principal Investigator Email
- tanjakovacevic74@yahoo.com
- Contact Person Name
- Tanja Kovacevic
- Contact Person Email
- tanjakovacevic74@yahoo.com
Slovakia
- Earliest CTIS Part Ii Submission Date
- 2024-06-28
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Narodny Ustav Detskych Chorob
- Department Name
- Klinika pediatrickej urologie LF UK a NUDCH
- Principal Investigator Name
- Peter Barton
- Principal Investigator Email
- Peter.Barton@nudch.eu
- Contact Person Name
- Peter Barton
- Contact Person Email
- Peter.Barton@nudch.eu
- Site Name
- J. Breza Medical s.r.o.
- Department Name
- Urologická ambulancia
- Principal Investigator Name
- Jan Breza
- Principal Investigator Email
- janbreza@yahoo.com
- Contact Person Name
- Jan Breza
- Contact Person Email
- janbreza@yahoo.com
Norway
- Earliest CTIS Part Ii Submission Date
- 2025-07-24
- Latest Decision Or Authorization Date
- 30-07-2025
- Processing Time Days
- 6
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Helse Bergen HF
- Department Name
- Department of Clinical Science
- Principal Investigator Name
- Camilla Tondel
- Principal Investigator Email
- camilla.tondel@helse-bergen.no
- Contact Person Name
- Camilla Tondel
- Contact Person Email
- camilla.tondel@helse-bergen.no
- Site Name
- Oslo University Hospital HF
- Department Name
- Department of Pediatric
- Principal Investigator Name
- Petra Aden
- Principal Investigator Email
- UXPEAD@Ous-hf.no
- Contact Person Name
- Petra Aden
- Contact Person Email
- UXPEAD@Ous-hf.no
Poland
- Earliest CTIS Part Ii Submission Date
- 2025-07-07
- Latest Decision Or Authorization Date
- 11-08-2025
- Processing Time Days
- 35
- Number Of Sites
- 3
- Number Of Participants
- 10
Sites
- Site Name
- Instytut Pomnik Centrum Zdrowia Dziecka
- Department Name
- Klinika Urologii Dzieciecej
- Principal Investigator Name
- Piotr Gastol
- Principal Investigator Email
- p.gastol@ipczd.pl
- Contact Person Name
- Piotr Gastol
- Contact Person Email
- p.gastol@ipczd.pl
- Site Name
- Uniwersytecki Dzieciecy Szpital Kliniczny Im. L. Zamenhofa W Bialymstoku
- Department Name
- Klinika Pediatrii i Nefrologii
- Principal Investigator Name
- Anna Wasilewska
- Principal Investigator Email
- anna.wasilewska@udsk.pl
- Contact Person Name
- Anna Wasilewska
- Contact Person Email
- anna.wasilewska@udsk.pl
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Klinika Chorob Nerek i Nadcisnienia Dzieci i Mlodziezy
- Principal Investigator Name
- Michal Maternik
- Principal Investigator Email
- mmaternik@gumed.edu.pl
- Contact Person Name
- Michal Maternik
- Contact Person Email
- mmaternik@gumed.edu.pl
Sponsor
Primary sponsor
- Full Name
- Sumitomo Pharma America Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Investigational products
- Investigational Product Name
- Vibegron (FILM-COATED TABLET)
- Active Substance
- VIBEGRON
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- prodAuthStatus: 1
- Starting Dose
- Cohort 1 (12 to <18 years): ≥41.5 kg = 75 mg; ≥29.5 kg to 41.4 kg = 50 mg. Cohort 2 (2 to <12 years): ≥29.5 kg = 50 mg; ≥11 kg to 29.4 kg = 20 mg
- Dose Levels
- 75 mg | 50 mg | 20 mg | 10 mg | 5 mg
- Frequency
- Once daily (QD)
- Maximum Dose
- 75 mg
- Dose Escalation Increase
- Initial doses assigned by weight (20 mg, 50 mg, 75 mg); doses may be adjusted based on PK/safety and DSMB review.
- Investigational Product Name
- Vibegron (GRANULES) - formulation variants
- Active Substance
- VIBEGRON
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- prodAuthStatus: 1
- Starting Dose
- Cohort 1 (12 to <18 years): ≥41.5 kg = 75 mg; ≥29.5 kg to 41.4 kg = 50 mg. Cohort 2 (2 to <12 years): ≥29.5 kg = 50 mg; ≥11 kg to 29.4 kg = 20 mg
- Dose Levels
- 75 mg | 50 mg | 20 mg | 10 mg | 5 mg
- Frequency
- Once daily (QD)
- Maximum Dose
- 75 mg
- Dose Escalation Increase
- Initial doses assigned by weight (20 mg, 50 mg, 75 mg); doses may be adjusted based on PK/safety and DSMB review.
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