Autosomal Recessive Polycystic Kidney study: Tolvaptan Suspension vs Tolvaptan Tablets
Official title 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)
ClinicalTrials.gov ID: NCT04782258
What this study is testing
What is Tolvaptan Suspension?
An investigational syrup being studied as a potential treatment for Autosomal Recessive Polycystic Kidney Disease (ARPKD). It is designed to block vasopressin V2 receptors, which may reduce cyst growth in the kidneys.
Also referred to as Tolvaptan.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether tolvaptan is safe for children with ARPKD.
- Who it's for
- You may be able to join if you are a child or teen with ARPKD and are between 28 days and 18 years old.
- What's involved
- You will take tolvaptan at home and have regular check‑ups with the study team. Blood and urine tests will be done to monitor safety.
- Time commitment
- The study lasts about 12 months, with visits every few weeks and occasional hospital visits for tests.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 28 to 18
You may be able to join if
- Male or female people 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...
You likely can't join if
- 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...
- 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.
See the full eligibility criteria
- Male or female people 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...
- 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.
- Cannot be monitored for fluid balance.
- Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.
- Has or at risk of having significant hypovolemia as determined by investigator.
- Clinically significant anemia, as determined by investigator.
- Platelets \< 50000 µL.
- Severe systolic dysfunction defined as ejection fraction \< 14%.
- Serum sodium levels \ 145 mmol/L.
- Taking any other experimental medications.
- Require ventilator support.
- Taking medications known to induce CYP3A4 (CYP = Cytochrome P).
- Having an infection including viral that would require therapy disruptive to IMP (Investigational Medicinal Product) dosing.
- Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP.
- people with a history of substance abuse (within the last 6 months).
- people who have bladder dysfunction and/or difficulty voiding.
- people taking a vasopressin agonist (eg, desmopressin).
- people with a history of persistent noncompliance with antihypertensive or other important medical therapy.
- people taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin...
- 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).
- Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide
- Intrauterine device
- Hormone-based contraceptives which are associated with inhibition of ovulation.
The study team makes the final eligibility decision.
Where it is enrolling
- Washington D.C., District of Columbia, United States
- Baltimore, Maryland, United States
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- Cincinnati, Ohio, United States
- Cleveland, Ohio, United States
- Brussels, Brussels Capital, Belgium
- Ghent, Oost-Vlaanderen, Belgium
- Leuven, Vlaams Brabant, Belgium
- Cologne, North Rhine-Westphalia, Germany
- Esplugues de Llobregat, Barcelona, Spain
- Barcelona, Spain
- London, 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, 28 days to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Washington D.C., District of Columbia, United States; Baltimore, Maryland, United States; Ann Arbor, Michigan, United States; Rochester, Minnesota, United States; Cincinnati, Ohio, United States; Cleveland, Ohio, United States and 7 more locations. Enter your location above to see the nearest enrolling 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 ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.