NCT04786574

A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. · Industry
• Sex: All
• Age: 4 Weeks – 12 Weeks
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy 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 24 months and closely monitored over the course of the study.

Conditions

• Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Interventions

Type	Name	Description
DRUG	Tolvaptan (OPC-41061)	Tolvaptan suspension will be administered orally or via nasogastric tube at doses of 0.15 mg/kg once daily in the AM, 0.30 mg/kg once daily in the AM, 0.5 mg/kg once daily in the AM, 0.75 mg/kg split dose (0.5 mg/kg AM and 0.25 mg/kg 8 hours later), and 1 mg/kg split dose (0.67 mg/kg AM and 0.33 mg/kg 8 hours later) based on age. Treatment duration is 2 years.

Timeline

• Start date: 2022-07-01
• Primary completion: 2025-12-10
• Completion: 2025-12-10
• First posted: 2021-03-08
• Last updated: 2025-12-24

Regulatory

• FDA-regulated drug study