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)
Otsuka Pharmaceutical Development & Commercialization, Inc.
Summary
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
Description
This study is a multinational, multicenter, open-label, non-randomized trial. The study consist of three periods: Screening Period, Treatment period and Follow-up period. Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 4), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV). Participants in this study will be assigned to tolvaptan and followed for 24 months over the course of the study. The overall trial duration is expected to be appr…
Eligibility
- Age range
- 0–18 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. 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. 2. 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. Exclusion Criteria: 1. Premature birth (≤ 32 weeks gestational age) for infants 28 days to…
Interventions
- DrugTolvaptan Suspension
Syrup
- DrugTolvaptan Tablets
Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.
Locations (23)
- Children's National Medical CenterWashington D.C., District of Columbia
- Emory University HospitalAtlanta, Georgia
- Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - NeonatologyChicago, Illinois
- Riley Hospital for ChildrenIndianapolis, Indiana
- Children's Hospital - New OrleansNew Orleans, Louisiana
- Johns Hopkins Pediatric Specialty ClinicBaltimore, Maryland