NCT04782258

A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal 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 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. · Industry
• Sex: All
• Age: 28 Days – 18 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety of tolvaptan in pediatric subjects with ARPKD

Detailed description

This study is a multinational, multicenter, open-label, non-randomized trial. The study consist of three periods: Screening Period, Treatment period and Follow-up period. Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 4), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV). Participants in this study will be assigned to tolvaptan and followed for 18 months over the course of the study. The overall trial duration is expected to be approximately 3.5 years.

Conditions

• Autosomal Recessive Polycystic Kidney (ARPKD)

Interventions

Type	Name	Description
DRUG	Tolvaptan Suspension	Syrup
DRUG	Tolvaptan Tablets	Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.

Timeline

• Start date: 2023-01-23
• Primary completion: 2028-07-31
• Completion: 2028-08-14
• First posted: 2021-03-04
• Last updated: 2026-02-17

Locations

23 sites across 6 countries: United States, Belgium, Germany, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study