Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 days to less than 12 weeks of Age with Autosomal Recessive Polycystic Kidney Disease (ARPKD).

This research study is about a medicine called Tolvaptan for very young babies, specifically those between 28 days and less than 12 weeks old, who have a rare kidney condition known as Autosomal Recessive Polycystic Kidney Disease (ARPKD). Doctors are trying to find out if Tolvaptan is safe for these children and if it can help their kidneys work better. They will also be checking to see how well the babies tolerate the medicine. The main goal is to see if taking Tolvaptan can reduce the number of babies who need kidney dialysis or a kidney transplant by their first birthday. This is a Phase 3 study, which means the medicine has already been tested to some extent, and now they are confirming its effects in a larger group.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Otsuka Pharmaceutical Development & Commercialization Inc.

Enrolment target

Start

29 Apr 2024

Autosomal Recessive Polycystic Kidney Disease

What is this study about?

This study is looking into a new medicine called Tolvaptan for very young babies and children (from 28 days to under 12 weeks old) who have a serious kidney problem called Autosomal Recessive Polycystic Kidney Disease, or ARPKD. This condition affects how the kidneys develop and work, leading to cysts (small fluid-filled sacs) that can damage the kidneys over time.

Doctors want to find out if Tolvaptan can help slow down the damage to the kidneys in babies with ARPKD. They are particularly interested in whether this medicine can reduce the number of children who need serious kidney treatments, like dialysis or a kidney transplant, by the time they are one year old. They are also carefully watching to see if the medicine is safe and if the babies can take it without too many problems.

This is an important step in trying to find better ways to treat ARPKD in its earliest stages. By understanding how Tolvaptan works in these young patients, researchers hope to improve the long-term health and well-being of children living with this challenging kidney condition.

Key takeaways

This study is for babies with a kidney condition called ARPKD.
It's testing a medicine called Tolvaptan to see if it helps their kidneys.
The main goal is to see if it reduces the need for dialysis or transplant by age one.
Participants will have regular check-ups and scans for two years.
It's a Phase 3 study, meaning previous research has already shown some promise.

Who may be eligible?

This study is open to both baby boys and baby girls.

To join, babies must be between 28 days old and less than 12 weeks old when they start the study. All babies joining the study will have Autosomal Recessive Polycystic Kidney Disease (ARPKD). Your healthcare team will confirm if your child meets all the specific health requirements for the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child between 28 days and less than 12 weeks old?
Has my child been diagnosed with Autosomal Recessive Polycystic Kidney Disease (ARPKD)?
Is my child generally healthy enough to take part in a clinical study?
Am I able to bring my child to regular hospital or clinic appointments?
Am I willing to have my child take the study medicine for two years?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will be given the medicine Tolvaptan. You and your child will need to visit the study clinic regularly for check-ups over a period of two years. These visits will involve various checks, such as measuring their growth (height, weight, head size), taking blood and urine samples, checking their blood pressure, and having ultrasound scans of their kidneys to see how they are changing. The study team will also ask about how your child is feeling and if they are having any side effects. After two years of treatment, there will be further follow-up to monitor their health.

Potential risks and benefits

Taking part in this study could potentially benefit your child by providing them with a new treatment that might help slow down their kidney disease. However, as with any medicine, there are potential risks and side effects that your child might experience. The study team will carefully monitor your child for any problems and explain all known side effects. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Belgium
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Unverified
Spain
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Unverified
Germany
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Unverified
Poland

Common questions

What is ARPKD?

ARPKD stands for Autosomal Recessive Polycystic Kidney Disease. It's a rare genetic condition that causes cysts (small fluid-filled sacs) to grow in the kidneys, which can stop them from working properly.

What is Tolvaptan?

Tolvaptan is a medicine being studied to see if it can help slow down kidney damage in babies with ARPKD. It's thought to work by affecting how the kidneys handle water.

How long will my child need to take the medicine?

Children in this study will receive the medicine for two years. After that, they will still have follow-up visits to check on their health.

Will my child definitely get the study medicine?

Yes, in this study, all children who are eligible and join will receive the study medicine, Tolvaptan.

What does 'RRT' mean?

RRT stands for Renal Replacement Therapy. This refers to treatments like dialysis (a machine that cleans the blood) or a kidney transplant, which are needed when kidneys can no longer do their job.