A trial 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)
- Conditions
- Autosomal Recessive Polycystic Kidney Disease (ARPKD)MedDRA version: 20.0Level: LLTClassification code 10036047Term: Polycystic kidney, autosomal recessiveSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2020-005991-36-BE
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
1. Male or female subjects between 28 days and < 12 weeks of age, inclusive.
2. Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
- Nephromegaly (> 2 standard deviations from age appropriate standard via ultrasound)
- Multiple renal cysts
- History of oligohydramnios or anhydramnios
3. 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Premature birth (= 32 weeks gestational age)
2. Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration, or history of kidney transplantation
3. Evidence of syndromic conditions associated with renal cysts (other than ARPKD)
4. Abnormal liver function tests including ALT and AST, > 1.2 × ULN
5. Parents with renal cystic disease
6. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation
7. Cannot be monitored for fluid balance
8. Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator
9. Has or at risk of having significant hypovolemia (eg, subjects that lack free access to water, without adequate fluid monitoring and management) as determined by investigator
10. Clinically significant anemia, as determined by investigator
11. Severe systolic dysfunction defined as ejection fraction < 14%
12. Serum sodium levels < 130 mmol/L or >145 mmol/L (or the ULN of the local laboratory, whichever is lower).
13. Taking any other experimental medications
14. Require ventilator support
15. Taking medications known to induce CYP3A4
16. Having an infection including viral that would require therapy disruptive to IMP dosing
17. Platelet count <50,000 µL
18. Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia).
19. Bladder dysfunction and/or difficulty voiding
20. Taking a vasopressin agonist (eg, desmopressin)
21. 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)
22. History of cholangitis
23. Received or are scheduled to receive a liver transplant
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method