A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Brief Summary:
The primary objective of this study is to evaluate the safety of tolvaptan in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)
Detailed Description:
Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 3), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).
The trial will be the first trial of tolvaptan in a pediatric ARPKD population.
Participants in this study will be assigned to tolvaptan for 18 months and closely monitored over the course of the study.
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 20 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Estimated Study Start Date: April 1, 2021
Estimated Primary Completion Date: March 31, 2025
Estimated Study Completion Date: June 1, 2025
Arm:
- Experimental: Tolvaptan Suspension
- Experimental: Tolvaptan Tablets
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 20 |
| Estimated Study start date | 01 April 2021 |
| Estimated Study Completion Date | 01 June 2025 |