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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
Must have clinical and imaging features consistent with a diagnosis of ARPKD with specific characteristics: Nephromegaly, multiple renal cysts, history of oligohydramnios or anhydramnios
Male or female subjects between 28 days and < 12 weeks of age, inclusive at the time of enrollment
Must not have
Has or at risk of having sodium and potassium electrolyte imbalances
Significant Portal Hypertension
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial is testing tolvaptan, a medication that slows kidney damage, in children with a severe kidney disease called ARPKD. Tolvaptan works by blocking a hormone that causes the kidneys to hold too much water, helping to reduce cysts and protect kidney function. The study will monitor children taking tolvaptan for a period of time. Tolvaptan has been shown to slow the progression of renal disease in adults with ADPKD, but its use in children is still being studied.

Who is the study for?
This trial is for infants aged between 28 days and less than 12 weeks with autosomal recessive polycystic kidney disease (ARPKD). They should have specific symptoms like enlarged kidneys and multiple cysts. Babies born prematurely or those needing dialysis, with liver issues, low platelets, severe anemia, or electrolyte imbalances can't participate.
What is being tested?
The study tests if Tolvaptan can help delay the need for dialysis in young children with ARPKD. It aims to see how effective this medication is at managing their kidney function over time.
What are the potential side effects?
Tolvaptan may cause thirst, frequent urination, fatigue, liver enzyme increases leading to potential liver damage, and possible electrolyte imbalance such as low sodium levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have ARPKD with enlarged kidneys, kidney cysts, and a history of low or no amniotic fluid.
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My child is between 28 days and less than 12 weeks old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have or might have imbalances in my body's sodium and potassium levels.
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I have severe high blood pressure in the veins of my liver.
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I do not have an infection that would interfere with the trial medication.
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I need to take water pills regularly.
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I have a condition linked to kidney cysts, not including ARPKD.
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I have trouble emptying my bladder.
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I am taking medications that affect liver enzyme levels.
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I have had cholangitis before.
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One or both of my parents have kidney cysts.
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I am on dialysis or have had a kidney transplant.
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I have received or am scheduled for a liver transplant.
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I am currently taking medication that acts like vasopressin.
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My heart's pumping ability is severely low.

Timeline

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

Side effects data

From 2017 Phase 3 trial • 1370 Patients • NCT02160145
32%
Polyuria
29%
Thirst
21%
Nocturia
10%
Polydipsia
9%
Dry mouth
100%
80%
60%
40%
20%
0%
Study treatment Arm
Tolvaptan (Single-blind Treatment Period)
Tolvaptan (Double-blind Treatment Period)
Placebo (Double-blind Treatment Period)

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Tolvaptan (OPC-41061)Experimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tolvaptan (OPC-41061)
2014
Completed Phase 3
~1370

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Polycystic Kidney Disease (PKD) include Vasopressin V2 Receptor Antagonists like Tolvaptan. Tolvaptan works by inhibiting the action of vasopressin, a hormone that promotes water reabsorption in the kidneys. By blocking vasopressin, Tolvaptan reduces cyst growth and slows the decline in kidney function, which is crucial for delaying the progression to end-stage renal disease. This mechanism is particularly important for PKD patients as it helps manage the disease's progression and associated symptoms, potentially improving quality of life and extending the time before more invasive treatments like dialysis or transplantation are needed.

Find a Location

Who is running the clinical trial?

Otsuka Pharmaceutical Development & Commercialization, Inc.Lead Sponsor
264 Previous Clinical Trials
170,059 Total Patients Enrolled
Rosa Real, MDStudy DirectorOtsuka Pharmaceutical Development & Commercialization, Inc.
3 Previous Clinical Trials
107 Total Patients Enrolled
Nataliya Agafonova, MDStudy DirectorOtsuka Pharmaceutical Development & Commercialization, Inc.
1 Previous Clinical Trials
289 Total Patients Enrolled

Media Library

Tolvaptan (Vasopressin V2 receptor antagonist) Clinical Trial Eligibility Overview. Trial Name: NCT04786574 — Phase 3
ARPKD Research Study Groups: Tolvaptan (OPC-41061)
ARPKD Clinical Trial 2023: Tolvaptan Highlights & Side Effects. Trial Name: NCT04786574 — Phase 3
Tolvaptan (Vasopressin V2 receptor antagonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04786574 — Phase 3
~11 spots leftby Oct 2027
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