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)

• Status: Not yet recruiting
• Trial end date: 2025-07-01
• Target enrollment: 0
• Participant gender: All

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)

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.
• Treatments: Tolvaptan

Criteria

Inclusion Criteria:

1. Male or female subjects between 28 days and < 12 weeks of age, inclusive.

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

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. Critical electrolyte imbalances, as determined by the investigator

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

13. Cannot be 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