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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
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.
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Children's National Medical Center
Washington D.C., District of Columbia, United States
RECRUITINGEmory University Hospital
Atlanta, Georgia, United States
WITHDRAWNNorthwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
Chicago, Illinois, United States
WITHDRAWNRiley Hospital for Children
Indianapolis, Indiana, United States
WITHDRAWNChildren's Hospital - New Orleans
New Orleans, Louisiana, United States
WITHDRAWNJohns Hopkins Pediatric Specialty Clinic
Baltimore, Maryland, United States
RECRUITINGC.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
RECRUITINGMayo Clinic - Rochester
Rochester, Minnesota, United States
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGCleveland Clinic
Cleveland, Ohio, United States
RECRUITING...and 13 more locations
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