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New treatment option for Autosomal Recessive Polycystic Kidney (ARPKD)
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?
Tolvaptan Suspension is an investigational medicine, being studied as a potential treatment for autosomal recessive polycystic kidney (arpkd).
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
- To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
- 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).
- people 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...
- 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's taking place
- Washington D.C., District of Columbia, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- New Orleans, Louisiana, United States
- Baltimore, Maryland, United States
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- Cincinnati, Ohio, United States
- Cleveland, Ohio, United States
- Pittsburgh, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Brussels, Brussels Capital, Belgium
- Ghent, Oost-Vlaanderen, Belgium
- Leuven, Vlaams Brabant, Belgium
- Cologne, North Rhine-Westphalia, Germany
- Warsaw, Masovian Voivodeship, Poland
- Bialystok, Poland
- Esplugues de Llobregat, Barcelona, Spain
- Sabadell, Barcelona, Spain
+ 3 more site(s).
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 - 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?
Study sites include Washington D.C., District of Columbia, United States; Atlanta, Georgia, United States; Chicago, Illinois, United States; Indianapolis, Indiana, United States; New Orleans, Louisiana, United States; Baltimore, Maryland, United States and 17 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.