A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less… (NCT04782258) | Clinical Trial Compass
RecruitingPhase 3
A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
United States, Belgium, Germany20 participantsStarted 2023-01-23
Plain-language summary
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
Who can participate
Age range
28 Days – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD.
. Ability for parent/legal guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial. Ability to provide written informed assent from all subjects old enough per local laws to provide assent.
Exclusion criteria
. Premature birth (≤ 32 weeks gestational age) for infants 28 days to \< 12 weeks of age.
. Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
. Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
. Abnormal liver function tests including ALT and AST, \> 1.2 × ULN (upper limit of normal).
. Has splenomegaly or portal hypertension (HTN).
. Parents with renal cystic disease.
. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)
Timeframe: From baseline to post-treatment after 24 months or EoTx
Trial details
NCT IDNCT04782258
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.