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

    matko.marlais@gosh.nhs.uk

  • Sponsor organisation

    Otsuka Pharmaceutical Development & Commercialization, Inc.

  • Eudract number

    2020-005992-10

  • ISRCTN Number

    ISRCTN00000000

  • Clinicaltrials.gov Identifier

    NCT04782258

  • 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