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Study on the Safety and Effects of Tolvaptan for Children with Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Verified siteRegistered drugNo 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 in infants and children. The trial will use a medication called Tolvaptan, which is available in different forms such as oral suspension and tablets. The tablets are marketed under names like Jinarc and Samsca. The purpose of the study is to evaluate the safety of Tolvaptan in young patients with ARPKD.

Participants in the study will receive Tolvaptan over a period of up to 18 months. The study will monitor the safety and tolerability of the medication, which means checking for any side effects or reactions. The trial will also look at how the medication affects the growth and development of the children, including changes in kidney size and function. The study will involve regular check-ups and assessments to ensure the well-being of the participants.

Throughout the trial, researchers will gather information on how well Tolvaptan works in managing ARPKD in children. This includes observing any changes in kidney function and overall health. The study aims to provide valuable insights into the treatment of ARPKD, potentially improving the quality of life for those affected by this condition. Participants will be closely monitored by healthcare professionals to ensure their safety and to gather accurate data on the effects of the medication.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the trial, the patient will be assessed to ensure they meet the criteria for participation. This includes being between 28 days and less than 18 years of age and having clinical features consistent with autosomal recessive polycystic kidney disease (ARPKD).

    Consent must be provided by a parent or legal guardian, and assent from the patient if they are old enough according to local laws.

  2. Step 2

    Medication administration

    The patient will receive tolvaptan, which is administered orally. The specific formulations include Jinarc 15 mg tablets, Samsca 7.5 mg tablets, and Jinarc 30 mg tablets.

    The dosage and frequency will be determined by the healthcare provider based on the patient's specific needs and condition.

  3. Step 3

    Monitoring and assessments

    Throughout the trial, the patient will undergo regular safety assessments. These include monitoring for any adverse events, checking vital signs, and conducting clinical laboratory assessments.

    The trial will also track changes in kidney function using the eGFR (estimated glomerular filtration rate) Schwartz formula, which involves measuring height and serum creatinine levels.

  4. Step 4

    Progress evaluation

    The trial will evaluate the annual rate of change in eGFR from the start of the trial to 18 months after treatment.

    Additional evaluations include changes in kidney size, growth percentiles for height, weight, and head circumference, and age-appropriate assessments of the medication's palatability and acceptability.

  5. Step 5

    Completion of the trial

    The trial is expected to continue until December 17, 2025. During this period, the patient's progress will be closely monitored, and any necessary adjustments to the treatment plan will be made based on the assessments.

Who can join the trial?

5 criteria

  • Participants must be **male or female** and between **28 days and less than 18 years old**.
  • Participants should have **clinical features** that match a diagnosis of **Autosomal Recessive Polycystic Kidney Disease (ARPKD)**. This is a condition that affects the kidneys.
  • The **parent or legal guardian** must be able to give **written, informed consent** before any trial-related activities begin. This means they agree to let their child participate after understanding what the trial involves.
  • Participants who are old enough, according to local laws, must be able to give their own **written informed assent**. This means they agree to participate in the trial.
  • Participants must be able, in the opinion of the main doctor in charge of the trial, to follow all the trial requirements.

Who cannot join the trial?

9 criteria

  • Patients who do not have Autosomal Recessive Polycystic Kidney Disease (ARPKD) cannot participate. ARPKD is a genetic condition that affects the kidneys.
  • Patients who are not within the specified age range for the study cannot participate. The study is for children aged 2 years and older.
  • Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
  • Patients who have other medical conditions that might interfere with the study or make it unsafe for them to participate cannot join the study.
  • Patients who are taking other medications that might interfere with the study medication cannot participate.
  • Patients who have had a recent surgery or medical procedure that might affect the study results cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate in the study.
  • Patients who have a history of allergic reactions to similar medications cannot participate.
  • Patients who are part of another clinical trial cannot participate in this study.
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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. The trial aims to determine how well children tolerate the medication and if it can help manage their condition.

What is already known about the treatment

Tolvaptan – Tolvaptan is administered orally in tablet form. It is currently being studied in clinical trials for its safety and effectiveness in treating Autosomal Recessive Polycystic Kidney Disease (ARPKD) in children. The main therapeutic indication for Tolvaptan is to slow the progression of kidney disease by reducing cyst formation and growth. At the molecular level, Tolvaptan works by blocking the action of vasopressin, a hormone that can contribute to cyst growth in the kidneys. It is classified as a vasopressin receptor antagonist.

Investigated diseases

Autosomal Recessive Polycystic Kidney Disease – This is a genetic disorder that affects the kidneys and liver, leading to the development of numerous fluid-filled cysts. It is inherited in an autosomal recessive pattern, meaning both parents must carry the gene for a child to be affected. The disease often begins in infancy or early childhood, causing the kidneys to become enlarged and potentially leading to kidney dysfunction. Over time, the cysts can interfere with the normal function of the kidneys and liver, leading to complications. 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 ID2023-508218-41-00Protocol code156-201-00307Estimated enrolment21 patientsSponsorOtsuka Pharmaceutical Development & Commercialization Inc.

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