A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 days to less than 18 years of Age with Autosomal Recessive Polycystic Kidney Disease (ARPKD)
EU CTIS ID: 2023-508218-41-00
What this study is testing
To evaluate the safety of tolvaptan in pediatric subjects with ARPKD
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- Premature birth (≤ 32 weeks gestational age) for infants 28 days to < 12 weeks of age.
- Has or at risk of having significant hypovolemia (eg, subjects that lack free access to water [inability to respond to thirst, depending on age], without adequate fluid monitoring and management) as determined by investigator.
- Clinically significant anemia, as determined by investigator.
- Platelets < 50000 µL.
- Severe systolic dysfunction defined as ejection fraction < 14%.
- Taking any other experimental medications.
See the full eligibility 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.
- Premature birth (≤ 32 weeks gestational age) for infants 28 days to < 12 weeks of age.
- Has or at risk of having significant hypovolemia (eg, subjects that lack free access to water [inability to respond to thirst, depending on age], without adequate fluid monitoring and management) as determined by investigator.
- Clinically significant anemia, as determined by investigator.
- Platelets < 50000 µL.
- Severe systolic dysfunction defined as ejection fraction < 14%.
- Taking any other experimental medications.
- Require ventilator support.
- Taking medications known to induce CYP3A4.
- Having an active infection including viral that would require therapy disruptive to IMP dosing.
- Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP.
- Subjects with a history of substance abuse within the last 6 months (depending on age).
- Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
- Subjects who have bladder dysfunction and/or difficulty voiding.
- Subjects taking a vasopressin agonist (eg, desmopressin).
- Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy.
- Subjects having concomitant illnesses or taking medications likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin antagonists, anti-sense RNA therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (ie, octreotide, sandostatin).
- Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP.
- Received or are scheduled to receive a liver transplant.
- History of cholangitis within the last 6 months.
- Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia).
- Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
- Abnormal liver function tests including ALT and AST, > 1.2 × ULN.
- Has splenomegaly or portal hypertension.
- Parents with renal cystic disease.
- Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
- Cannot be monitored for fluid balance.
- Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Belgium
- Spain
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Belgium; Spain; United States; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from EU CTIS. The study team decides eligibility, and your own doctor gives medical advice.