Autosomal Recessive Polycystic Kidney Disease (ARPKD) MedDRA version: 20.0 Level: LLT Classification code 10036047 Term: Polycystic kidney, autosomal recessive System Organ Class: 100000004850
Conditions
Interventions
Product Name: Tolvaptan 50mg Granules
Product Code: OPC-41061
Pharmaceutical Form: Granules for oral suspension
INN or Proposed INN: Tolvaptan
CAS Number: 150683-30-0
Current Sponsor code: OPC-41061
Sponsors
Otsuka Pharmaceutical Development & Commercialization, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female subjects between 28 days and 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
Exclusion criteria
Exclusion criteria: 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 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the effect of tolvaptan on the need for RRT in pediatric subjects with ARPKD ;Secondary Objective: Evaluate changes in eGFR and palatability and acceptability of formulation ;Primary end point(s): Percentage of subjects having RRT by 1 year of age ;Timepoint(s) of evaluation of this end point: 12 Months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Rate of change of eGFR (eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) from pretreatment to post-treatment after 2 years of treatment - Age-appropriate assessment of acceptability and palatability of formulation ;Timepoint(s) of evaluation of this end point: pre-treatment to post treatment after 2 years of treatment | — |
Countries
Belgium, Canada, Czechia, France, Germany, Italy, Poland, Spain, United Kingdom, United States
Contacts
Public ContactMathew Taylor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Outcome results
None listed