A Phase 3 interventional study of Tolvaptan Suspension and Tolvaptan Tablets in Autosomal Recessive Polycystic Kidney (ARPKD), sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc.. Recruiting at 23 sites in 6 countries. Open to participants aged 28 Days to 18 Years. Per ClinicalTrials.gov, last updated 2026-08-07.
Sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc. · Phase 3, Interventional, and Treatment
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
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 24 months over the course of the study.
The overall trial duration is expected to be approximately 5 years.
Exclusion Criteria:
Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP:
Tolvaptan suspension will be administered orally or via feeding/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 24 months.
Drug: Tolvaptan Suspension
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. Treatment duration is 24 months.
Drug: Tolvaptan Tablets
Syrup
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.
Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)
Number of participants with TEAEs will be assessed from Baseline to post-treatment after 24 months or End of Treatment (EOTx). The EOTx visit applies to participants who discontinue IMP before Month 24. TEAEs are defined as Adverse Events (AEs) with an onset date on or after the start of Investigational Medicinal Product (IMP) treatment. TEAEs are also events continuous from baseline which worsened, became serious, were IMP related, or resulted in death, discontinuation, interruption, or reduction of IMP. An AE is defined as any untoward medical occurrence in a clinical trial subject administered an IMP and which does not necessarily have a causal relationship with this treatment.
Time frame: From baseline to post-treatment after 24 months or EoTx
Annual rate of change of eGFR (by Schwartz formula) from baseline to post-treatment after 24 months
Annual rate of change of eGFR from Baseline to post-treatment after Month 24 or EOTx is calculated using eGFR Schwartz formula = 0.413 × height \[or length, centimeter (cm)\] /serum creatinine milligram per deciliter (mg/dL). The EOTx visit applies to participants who discontinue IMP before Month 24.
Time frame: From Baseline to post-treatment after Month 24 or EOTx
Change from baseline of eGFR (by Schwartz formula) while on treatment at Months 1, 6, 12, 18 and 24 or EOTx
Change from baseline of eGFR is calculated using eGFR Schwartz formula = 0.413 × height \[or length, cm\] /serum creatinine mg/dL) while on treatment at Months 1, 6, 12, 18, and 24 or EoTx. The EOTx visit applies to participants who discontinue IMP before Month 24.
Time frame: At Months 1, 6, 12, 18, and 24 or EOTx
The amount of time between enrollment and 24 months that a subject requires renal replacement therapy (RRT).
To evaluate the effect of tolvaptan on the need for RRT in pediatric participants with ARPKD.
Time frame: From enrollment to 24 months
The percentage of subjects that will receive renal replacement therapy (RRT) by 24 months
Percentage of subjects who receive RRT from baseline through Month 24. RRT is defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or kidney transplantation.
Time frame: From Baseline to Month 24
Plan to share: No
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Polycystic Kidney, Autosomal Recessive
Otsuka Pharmaceutical Development & Commercialization, Inc.