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Vasopressin V2 Receptor Antagonist

Tolvaptan for Polycystic Kidney Disease

Phase 3

Recruiting

Research Sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female subjects between 28 days and less than 18 years of age, with clinical features consistent with a diagnosis of ARPKD

Be younger than 65 years old

Must not have

Premature birth (≤ 32 weeks gestational age) for infants 28 days to < 12 weeks of age

Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial will test the safety of tolvaptan, a drug used to treat ARPKD, in children.

Who is the study for?

This trial is for infants and children from 28 days old to under 18 years with ARPKD. Participants need informed consent from parents or guardians, must be able to follow the trial's procedures, and not have been born prematurely if under 12 weeks old. They can't join if they require dialysis, have had a kidney transplant, severe anemia or heart issues, electrolyte imbalances, are on certain other medications including experimental drugs for PKD or CYP3A4 inducers.

What is being tested?

The study tests the safety of Tolvaptan in tablet and suspension forms in young patients with autosomal recessive polycystic kidney disease (ARPKD). It aims to see how well these pediatric subjects tolerate the medication.

What are the potential side effects?

While specific side effects for this age group aren't listed here, Tolvaptan in adults may cause thirstiness, increased urination frequency, liver enzyme elevations which could indicate liver injury risk; potential risks for children will be monitored closely.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am under 18 years old and have been diagnosed with ARPKD.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My baby was born at or before 32 weeks and is now between 28 days and less than 12 weeks old.

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I am on dialysis or have had a kidney transplant.

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I have an enlarged spleen or high blood pressure in the liver.

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I am not pregnant or breastfeeding.

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I have received or am scheduled for a liver transplant.

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I am currently taking medication that affects my body's water balance.

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I am on a diuretic that can't be changed after starting tolvaptan.

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One or both of my parents have kidney cysts.

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My heart's pumping ability is severely reduced.

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I have an infection that wouldn't interfere with the trial medication schedule.

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I have a history of not following my prescribed medical treatments consistently.

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I have signs of serious portal hypertension like varices or low platelet count.

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I have trouble emptying my bladder or have bladder control issues.

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I have a condition linked to kidney cysts, not including ARPKD.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Tolvaptan TabletsExperimental Treatment1 Intervention

Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets. Treatment duration is 18 months.

Group II: Tolvaptan SuspensionExperimental Treatment1 Intervention

Tolvaptan suspension will be administered orally or via feeding/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 18 months.

0 / 15Eligibility criteria met