A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 D… (NCT04786574) | Clinical Trial Compass
WithdrawnPhase 3
A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Stopped: Study stopped before enrolling any participants.
0Started 2022-07-01
Plain-language summary
The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)
Who can participate
Age range
4 Weeks – 12 Weeks
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 \< 12 weeks of age, inclusive at the time of enrollment.
. Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
. Ability for parent or 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.
Exclusion criteria
. Premature birth (≤ 32 weeks gestational 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
. Parents with renal cystic disease
. Need for chronic diuretic use
. Cannot be monitored for fluid balance
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
The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.
Timeframe: From Enrollment to 1 year of age
Trial details
NCT IDNCT04786574
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.