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Study on the Safety and Effects of Tolvaptan for Infants and Young Children with Autosomal Recessive Polycystic Kidney Disease

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Autosomal Recessive Polycystic Kidney Disease (ARPKD), which affects the kidneys of infants and young children. The trial will explore the effects of a medication called Tolvaptan, also known by its code name OPC-41061. Tolvaptan is an oral suspension, meaning it is a liquid medicine taken by mouth. It is designed to help manage the symptoms of ARPKD by potentially reducing the need for certain medical treatments that support kidney function.

The purpose of this study is to evaluate how well Tolvaptan works in infants and children aged 28 days to less than 12 weeks who have been diagnosed with ARPKD. The trial will observe the safety, tolerability, and effectiveness of Tolvaptan over a period of time. Participants will receive the medication and be monitored for any changes in their condition, including the size of their kidneys and overall growth. The study will also look at how the medication is tolerated by the young participants and any side effects that may occur.

Throughout the study, various assessments will be conducted to track the progress of the participants. These assessments will include regular check-ups to measure kidney function, growth, and other health indicators. The trial aims to provide valuable information on how Tolvaptan can be used to help manage ARPKD in young children, potentially improving their quality of life and reducing the need for more intensive treatments. The study is expected to continue until 2027, allowing for comprehensive data collection and analysis.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    The trial is designed for infants and children aged 28 days to less than 12 weeks with a condition called autosomal recessive polycystic kidney disease (ARPKD).

    Participation requires a diagnosis of ARPKD, characterized by enlarged kidneys, multiple kidney cysts, and a history of low or no amniotic fluid during pregnancy.

    A parent or legal guardian must provide written consent for the child to participate.

  2. Step 2

    Medication administration

    The medication used in this trial is called Tolvaptan, which is administered as an oral suspension.

    The purpose of the medication is to evaluate its effect on the need for renal replacement therapy (RRT) in children with ARPKD.

  3. Step 3

    Monitoring and assessments

    The primary goal is to assess the percentage of children requiring RRT by the age of one year.

    Secondary assessments include changes in kidney function, measured by the rate of change in estimated glomerular filtration rate (eGFR), and changes in kidney size over 24 months.

    Other evaluations include the palatability and acceptability of the medication, changes in sodium levels, growth measurements, vital signs, and any adverse events.

  4. Step 4

    Duration of the trial

    The trial is expected to continue until July 22, 2027.

    Participants will be monitored and assessed at various intervals throughout the trial period, including at 3, 6, 12, 18, and 24 months.

Who can join the trial?

6 criteria

  • The child must be a boy or girl between 28 days and less than 12 weeks old.
  • The child must show signs and test results that match a diagnosis of Autosomal Recessive Polycystic Kidney Disease (ARPKD). This includes:
  • Nephromegaly: This means the kidneys are larger than normal for the child's age, as seen on an ultrasound.
  • Having many cysts in the kidneys.
  • A history of oligohydramnios or anhydramnios, which means there was too little or no amniotic fluid around the baby during pregnancy.
  • The child's parent or legal guardian must be able to give written, informed consent before any trial-related activities start. They must also be able, in the opinion of the main doctor in charge, to follow all the trial's requirements.

Who cannot join the trial?

8 criteria

  • Patients who have a different kidney disease than Autosomal Recessive Polycystic Kidney Disease (ARPKD) cannot participate. ARPKD is a genetic condition affecting the kidneys.
  • Patients who are not within the specified age range for the study cannot participate. The study is for children.
  • Patients who are not able to follow the study procedures or take the study medication cannot participate.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are taking medications that might interfere with the study drug cannot participate.
  • Patients who have had a kidney transplant cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
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Investigated drugs

Tolvaptan is a medication being studied for its safety, tolerability, and effectiveness in treating infants and children with Autosomal Recessive Polycystic Kidney Disease (ARPKD). This condition causes cysts to form in the kidneys, leading to kidney damage. Tolvaptan works by slowing down the growth of these cysts, potentially reducing the need for renal replacement therapy (RRT), which includes treatments like dialysis or kidney transplantation.

What is already known about the treatment

Tolvaptan – Tolvaptan is administered orally and is currently being studied in clinical trials for its safety, tolerability, and effectiveness in treating Autosomal Recessive Polycystic Kidney Disease (ARPKD) in infants and children. It is a medication that is already used in other conditions and is being evaluated for this specific use. The main therapeutic indication for Tolvaptan in this trial is to potentially reduce the need for renal replacement therapy in young patients with ARPKD. At the molecular level, Tolvaptan works by blocking the action of vasopressin, a hormone that can contribute to kidney cyst growth. It is classified as a vasopressin receptor antagonist.

Investigated diseases

Autosomal Recessive Polycystic Kidney Disease – This is a genetic disorder characterized by the development of numerous fluid-filled cysts in the kidneys. It is inherited in an autosomal recessive pattern, meaning both parents must carry the gene for a child to be affected. The disease often presents in infancy or early childhood, leading to enlarged kidneys and potential kidney dysfunction. As the condition progresses, it can cause high blood pressure and issues with kidney function. The liver may also be affected, leading to complications such as fibrosis. The progression of the disease can vary, with some individuals experiencing more severe symptoms than others.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2023-508217-17-00Protocol code156-12-204Estimated enrolment20 patientsSponsorOtsuka Pharmaceutical Development & Commercialization Inc.

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