WithdrawnPhase 3

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)

Stopped: Study stopped before enrolling any participants.

0Started 2022-07-01

Plain-language 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)

Who can participate

Age range4 Weeks – 12 Weeks

SexALL

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Inclusion criteria

✓. Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
✓. Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
✓. Ability for parent or 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.

Exclusion criteria

✕. Premature birth (≤ 32 weeks gestational 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
✕. Parents with renal cystic disease
✕. Need for chronic diuretic use
✕. Cannot be monitored for fluid balance
✕. Has or at risk of having sodium and potassium electrolyte imbalances

What they're measuring

The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.

Timeframe: From Enrollment to 1 year of age

Trial details

NCT IDNCT04786574

SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-12-10

View on ClinicalTrials.gov More Autosomal Recessive Polycystic Kidney Disease (ARPKD) trials