RecruitingPHASE3INTERVENTIONAL

A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)

This research study is looking at a medicine called tolvaptan for children and teenagers (from 28 days old up to just under 18 years) who have a kidney condition called Autosomal Recessive Polycystic Kidney Disease (ARPKD). The main purpose is to check if tolvaptan is safe for them. We know that this medicine has helped adults with a similar kidney disease (ADPKD) by slowing down kidney damage. This study will follow participants for 18 months, with the total study lasting about three and a half years. All participants will receive tolvaptan, and doctors will carefully monitor how they get on. This is an important step to understand if tolvaptan could be a future treatment option for young people with ARPKD.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Otsuka Pharmaceutical Development & Commercialization, Inc.

Enrolment target

Start

23 Jan 2023

Estimated completion

14 Aug 2028

Autosomal Recessive Polycystic Kidney (ARPKD)

What is this study about?

This study is about a medicine called tolvaptan, and it's being tested in children and teenagers (from 28 days old up to just under 18 years) who have a specific kidney condition called Autosomal Recessive Polycystic Kidney Disease, or ARPKD. ARPKD is a condition where many cysts (fluid-filled sacs) grow in the kidneys, which can stop them from working properly over time.

The main aim of this study is to find out if tolvaptan is safe for young people with ARPKD. We already know that tolvaptan has been helpful for adults with a very similar kidney condition called ADPKD. In adults, it helped to slow down the damage to their kidneys. Researchers hope to learn if it could offer similar benefits, and most importantly, be safe for children with ARPKD.

Taking part would mean receiving the tolvaptan medicine and having regular checks over 18 months. The research team will be looking very closely at how participants respond to the treatment and ensuring their safety throughout the study. This kind of research is vital for finding new ways to help children with ARPKD.

Key takeaways

This study is testing if a medicine called tolvaptan is safe for children with ARPKD.
Tolvaptan has helped adults with a similar kidney condition by slowing down kidney damage.
Children aged 28 days to under 18 years with ARPKD may be able to join.
Participants will receive tolvaptan and be followed closely for 18 months.
The study aims to find new potential treatments for children with ARPKD.

Who may be eligible?

To join this study, children need to be between 28 days old and just under 18 years old, and have been diagnosed with ARPKD. Their parents or legal guardians must be able to understand and sign consent forms, and everyone involved must be able to follow the study rules. If the child is old enough according to local laws, they will also be asked to agree to take part.

There are several reasons why a child might not be able to join. For example, very premature babies (born at 32 weeks or less) who are less than 12 weeks old cannot take part. Children who already need dialysis or have had a kidney transplant, or those with very poor liver function or certain other health issues like severe heart problems, will not be able to participate. Also, if a child cannot be properly monitored for how much fluid they drink and pass, or if they have certain blood problems, they would not be suitable.

Children currently taking other experimental medicines, or those who need continuous breathing support (ventilator), or certain specific medications that could interfere with the study drug, also cannot join. For girls, they cannot be breastfeeding or pregnant. The study team will review all of these details carefully with you to see if your child is a good fit.

Quick self-check

Is my child between 28 days and less than 18 years old?
Does my child have a diagnosis of ARPKD?
Is my child currently on dialysis or has had a kidney transplant?
Does my child have any other severe health problems as listed by the doctor?
Can we commit to regular hospital visits and follow-up for 18 months?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child takes part in this study, they will receive the study medicine, tolvaptan, which comes as either a liquid ('suspension') or tablets. They will be followed very closely for 18 months. This will involve regular visits to the hospital for check-ups, blood tests, and other assessments to see how they are responding to the medicine and to monitor their health generally.

The total study is expected to last about three and a half years from start to finish, with your child's individual treatment and follow-up lasting 18 months. The study is 'open-label,' meaning both you and the study doctors will know that your child is receiving tolvaptan. There won't be a placebo group. The doctors will also carefully monitor your child’s fluid intake and output throughout the study.

Potential risks and benefits

Taking part in a study like this could potentially offer earlier access to a medicine that might help slow down kidney damage in ARPKD, based on its benefits in similar adult conditions. However, there's no guarantee that it will help every child, and it's also possible that tolvaptan could have side effects, just like any medicine. The study team will closely monitor your child for any problems, but potential risks could include issues with liver function, blood salt levels, or fluid balance. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their usual medical care.

Locations (23)

Children's National Medical Center
Washington D.C., United States· Recruiting
Emory University Hospital
Atlanta, United States· Withdrawn
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
Chicago, United States· Withdrawn
Riley Hospital for Children
Indianapolis, United States· Withdrawn
Children's Hospital - New Orleans
New Orleans, United States· Withdrawn
Johns Hopkins Pediatric Specialty Clinic
Baltimore, United States· Recruiting
C.S. Mott Children's Hospital
Ann Arbor, United States· Recruiting
Mayo Clinic - Rochester
Rochester, United States· Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, United States· Recruiting
Cleveland Clinic
Cleveland, United States· Recruiting
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, United States· Withdrawn
Primary Children's Hospital
Salt Lake City, United States· Withdrawn

+11 more sites — see the official record for the full list.

Common questions

What is ARPKD?

ARPKD is a kidney condition that affects children. It causes many small, fluid-filled sacs (cysts) to grow in the kidneys, which can stop them from working properly.

What is tolvaptan?

Tolvaptan is a medicine that has been shown to help adults with a similar kidney condition by slowing down kidney damage. This study is testing if it's safe for children with ARPKD.

Will my child definitely get the medicine?

Yes, if your child qualifies for this study and you agree to participate, they will receive the tolvaptan medicine.

How long will my child be in the study?

Your child will receive the medicine and be followed for 18 months.

Can I take my child out of the study if we change our mind?

Yes, you can choose to withdraw your child from the study at any time, and it won't affect their regular medical care.

How to find out more

Otsuka Call Center

Otsuka-ProfessionalServices@otsuka-us.com 844-687-8522 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.