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)

NCT04786574 | Withdrawn Phase 3 DMC FDA Drug

• 1 other identifier: 156-12-204
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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)

Conditions

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Keywords

ARPKD; Tolvaptan; Polycystic Kidney Disease; Autosomal Recessive Polycystic Kidney Disease; Renal Cysts; Nephromegaly; Oligohydramnios; Anhydramnios

Outcome Measures

Primary Outcomes (1)

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

  From Enrollment to 1 year of age

Secondary Outcomes (3)

• Rate of change of eGFR by Schwartz formula from pre-treatment to after 2 years of treatment

  From Enrollment to 2 years of age

• Palatability of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose

  From Enrollment to 2 years of age

• Acceptance of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose

  From Enrollment to 2 years of age

Study Arms (1)

Tolvaptan (OPC-41061)

EXPERIMENTAL

Drug: Tolvaptan (OPC-41061)

Interventions

Tolvaptan (OPC-41061) DRUG

Tolvaptan suspension will be administered orally or via 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 2 years.

Tolvaptan (OPC-41061)

Eligibility Criteria

• Age: 4 Weeks - 12 Weeks
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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:
• Nephromegaly (\> 2 standard deviations from age-appropriate standard via ultrasound)
• Multiple renal cysts
• History of oligohydramnios or anhydramnios
• 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.

You may not qualify if:

• 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
• Has or at risk of having significant hypovolemia as determined by investigator
• Clinically significant anemia, as determined by investigator
• Severe systolic dysfunction defined as ejection fraction \< 14%
• Serum sodium levels \< 130 mmol/L or \>145 mmol/L
• Taking any other experimental medications
• Require ventilator support
• Taking medications known to induce CYP3A4

Sponsors & Collaborators

• Otsuka Pharmaceutical Development & Commercialization, Inc.: lead

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Recessive; Polycystic Kidney Diseases; Oligohydramnios

Interventions

Tolvaptan

Condition Hierarchy (Ancestors)

Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn; Pregnancy Complications

Intervention Hierarchy (Ancestors)

Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Olga Sergeyeva, MD

  Otsuka Pharmaceutical Development & Commercialization, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2021
• First Posted: March 8, 2021
• Study Start: July 1, 2022
• Primary Completion: December 10, 2025
• Study Completion: December 10, 2025
• Last Updated: December 24, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share