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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) | Clinical Trials | Clareo Health

WITHDRAWNPHASE3

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)

A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

NCT04786574•Otsuka Pharmaceutical Development & Commercialization, Inc.

View on ClinicalTrials.gov

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)

Detailed Description

Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 3), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV). The trial will be the first trial of tolvaptan in a pediatric ARPKD population. Participants in this study will be assigned to tolvaptan for 24 months and closely monitored over the course of the study.

Eligibility

Age

0 - 0 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
•2. Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
•* Nephromegaly (\> 2 standard deviations from age-appropriate standard via ultrasound)
•* Multiple renal cysts
•* History of oligohydramnios or anhydramnios
•3. 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

•1. Premature birth (≤ 32 weeks gestational age)
•2. Anuria or RRT, defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation
•3. Evidence of syndromic conditions associated with renal cysts (other than ARPKD)
•4. Abnormal liver function tests including ALT and AST, \> 1.2 × ULN
•5. Parents with renal cystic disease
•6. Need for chronic diuretic use
•7. Cannot be monitored for fluid balance
•8. Has or at risk of having sodium and potassium electrolyte imbalances
•9. Has or at risk of having significant hypovolemia as determined by investigator
•10. Clinically significant anemia, as determined by investigator
+13 more criteria

Key Dates

Start Date

July 1, 2022

Primary Completion Date

December 10, 2025

Completion Date

December 10, 2025

Last Updated

December 24, 2025

Conditions

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Interventions

Tolvaptan (OPC-41061)

DRUG