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)

NCT ID: NCT04786574

Last Updated: 2025-12-24

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-07-01
• Study Completion Date: 2025-12-10

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)

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.

Conditions

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Keywords

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Tolvaptan (OPC-41061)

Group Type: EXPERIMENTAL

Tolvaptan (OPC-41061)

Intervention Type: 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.

Interventions

Tolvaptan (OPC-41061)

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.

Intervention Type: DRUG

Eligibility Criteria

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

11. Severe systolic dysfunction defined as ejection fraction < 14%

12. Serum sodium levels < 130 mmol/L or >145 mmol/L

13. Taking any other experimental medications

14. Require ventilator support

15. Taking medications known to induce CYP3A4

16. Having an infection including viral that would require therapy disruptive to IMP dosing

17. Platelet count <50,000 µL

18. Significant Portal Hypertension

19. Bladder dysfunction or difficulty voiding

20. Taking vasopressin agonist

21. Having concomitant illness or taking medications that are likely to confound endpoint assessments.

22. History of cholangitis

23. Received or scheduled to receive a liver transplant

• Minimum Eligible Age: 4 Weeks
• Maximum Eligible Age: 12 Weeks
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Otsuka Pharmaceutical Development & Commercialization, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Olga Sergeyeva, MD

Role: STUDY_DIRECTOR

Otsuka Pharmaceutical Development & Commercialization, Inc.

Countries

Belgium; Germany; Italy; Poland; Spain; United Kingdom; United States

Other Identifiers

156-12-204

Identifier Type: -

Identifier Source: org_study_id