To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD.
Ability for parent/legal guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial. Ability to provide written informed assent from all subjects old enough per local laws to provide assent.
Premature birth (≤ 32 weeks gestational age) for infants 28 days to < 12 weeks of age.
Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
Abnormal liver function tests including ALT and AST, > 1.2 × ULN (upper limit of normal).
Parallel
Tolvaptan Suspension
DrugSyrup
Tolvaptan Tablets
DrugTolvaptan (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.
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.
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.
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.
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.
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