What side effects are associated with this type of intervention?

Tolvaptan, a Vasopressin V2 Receptor Antagonist, is commonly used in the treatment of Polycystic Kidney Disease (PKD). Known side effects of Tolvaptan include increased thirst, frequent urination, dry mouth, and potential liver injury, which necessitates regular liver function monitoring. Other treatments for PKD, such as antihypertensive medications, may cause side effects like dizziness, fatigue, and electrolyte imbalances. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

Tolvaptan, a vasopressin V2 receptor antagonist, has been approved by the FDA for the treatment of autosomal dominant polycystic kidney disease (ADPKD). It is marketed under the brand name Jynarque and is used to slow the progression of kidney function decline in adults at risk of rapidly progressing ADPKD. Tolvaptan works by inhibiting the action of vasopressin, thereby reducing cyst growth and preserving kidney function. There are no other vasopressin V2 receptor antagonists currently approved by the FDA specifically for the treatment of PKD.

What other types of research exist?

Currently, there are no other disease-modifying agents recommended for routine clinical use in PKD besides Tolvaptan. Somatostatin analogs like octreotide and lanreotide have been studied but are not recommended due to lack of demonstrated benefit and adverse effects. Therefore, Tolvaptan remains the primary treatment option for PKD as of 2024.

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Vasopressin V2 receptor antagonist

Tolvaptan for Polycystic Kidney Disease

Q: What is the mechanism of action of this treatment?

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.

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

Select...

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 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

Side effects data

From 2017 Phase 3 trial • 1370 Patients • NCT02160145

32%

Polyuria

29%

Thirst

21%

Nocturia

10%

Polydipsia

Dry mouth

100%

80%

60%

40%

20%

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

0 / 15Eligibility criteria met

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.