Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC

Résumé

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

Éligibilité

Conception de l'étude

Critères de jugement

1. Critère de jugement principal

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

   Délai Optimized Treatment Week 24

2. Critère de jugement secondaire

   Change from Baseline in MCC

   Délai Optimized Week 12

3. Critère de jugement secondaire

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

   Délai Optimized Treatment Weeks 12 and 24

4. Critère de jugement secondaire

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

   Délai Optimized Treatment Weeks 12 and 24

5. Critère de jugement secondaire

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

   Délai Optimized Treatment Weeks 12 and 24

6. Critère de jugement secondaire

   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

   Délai Optimized Treatment Weeks 12 and 24

7. Critère de jugement secondaire

   Change from Baseline in average first morning catheterized volume

   Délai through study completion, an average of 52 weeks

8. Critère de jugement secondaire

   Change from Baseline in average catheterized volume per catheterization

   Délai through study completion, an average of 52 weeks

9. Critère de jugement secondaire

   Change from Baseline in average maximum catheterized volume per day

   Délai through study completion, an average of 52 weeks

10. Critère de jugement secondaire

    Change from Baseline in average maximum catheterized daytime volume

    Délai through study completion, an average of 52 weeks

11. Critère de jugement secondaire

    Change from Baseline in average number of leakage episodes per day

    Délai through study completion, an average of 52 weeks

12. Critère de jugement secondaire

    Change from Baseline in estimated number of dry (leakage-free) days/ 7 days

    Délai through study completion, an average of 52 weeks

13. Critère de jugement secondaire

    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.

    Délai through study completion, an average of 52 weeks

14. Critère de jugement secondaire

    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.

    Délai through study completion, an average of 52 weeks

15. Critère de jugement secondaire

    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.

    Délai through study completion, an average of 52 weeks

Dates

Réelle signifie que l'événement a eu lieu. Estimée signifie que le promoteur s'y attend. Les deux ont un sens différent.

Lieux

32 sites sont en recrutement

Documents de l'étude

Aucun document n'est lié dans cette fiche du registre.

Évolution au fil du temps

Aucun changement n'a été enregistré depuis que nous avons intégré cette fiche pour la première fois.

Un changement est enregistré chaque fois que le promoteur met à jour la fiche du registre. Le statut, les dates, le recrutement et les sites apparaissent ici au fur et à mesure de leur évolution.