Phase 3b trial in Children with ARPKD from 28 days to less than 18 yea
Research type
Research Study
Full title
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)
IRAS ID
300087
Contact name
Matko Marlais
Contact email
Sponsor organisation
Otsuka Pharmaceutical Development & Commercialization, Inc.
Eudract number
2020-005992-10
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
Tolvaptan is being studied as a possible treatment for autosomal recessive polycystic kidney disease (ARPKD). ARPKD is a genetic disease that causes enlarged kidneys due to cysts (cyst are like fluid-filled balloons), breathing problems due to lack of space caused by the enlarged kidneys, excessive urine production, thirst, high blood
pressure, loss of kidney function, growth problems, congenital hepatic fibrosis (a liver abnormality), and enlarged
spleen after birth.
The kidneys of patients with ARPKD respond abnormally to a hormone in the bloodstream called vasopressin. This abnormal response can cause the cysts in the kidneys, associated with the disease, to grow more quickly. Tolvaptan works by blocking the effect of this hormone in the kidneys which may slow the growth of the kidney cysts.
Tolvaptan is currently approved only in adults. The trial will be the first trial of tolvaptan in a pediatric ARPKD population.
Participants in this study will be assigned to tolvaptan for 19 months and closely monitored over the course of the study. The primary objective of this study is to evaluate the safety, efficacy, and tolerability of tolvaptan in pediatric
subjects with autosomal recessive polycystic kidney disease (ARPKD).REC name
London - Riverside Research Ethics Committee
REC reference
21/LO/0511
Date of REC Opinion
24 Sep 2021
REC opinion
Further Information Favourable Opinion