Oral Tolvaptan extension study in children with Hyponatremia

  • Research type

    Research Study

  • Full title

    A Phase 3b, Multicenter, Extension Follow-up Trial to Evaluate the Long-term Safety of Children and Adolescent Subjects With Euvolemic or Hypervolemic Hyponatremia Who Have Previously Participated in a Trial of Titrated Oral SAMSCA® (Tolvaptan)

  • IRAS ID

    166695

  • Contact name

    Detlef Bockenhauer

  • Contact email

    d.bockenhauer@ucl.ac.uk

  • Sponsor organisation

    Otsuka Pharmaceutical Development & Commercialization, Inc.

  • Eudract number

    2013-002810-11

  • Clinicaltrials.gov Identifier

    NCT02020278

  • Duration of Study in the UK

    2 years, 4 months, 24 days

  • Research summary

    Hyponatraemia is a disease seen in all age groups, hospitalised children in particular are at risk, and across a wide range of patients. It is characterised by a subnormal concentration of sodium in the blood and is manifest by a range of neurological symptoms which, left untreated, can be life threatening. Hyponatraemia is associated with significant additional hospital morbidity and cost.\nIn the adult population, hyponatraemia occurs in 15% to 20% of hospitalised patients. A similar situation exists for children, in whom approximately 25% of hospitalised children are found to have mild hyponatraemia and approximately 1% were found to have moderate hyponatraemia. Hyponatraemia can be caused by either a deficiency of salt or, more commonly, by an excess of water. The latter is called dilutional hyponatraemia.\nThe study drug, Tolvaptan, is an experimental medicine which means it has not yet been approved by a drug approval agency for use in children and adolescents. This study is a follow-up study for children and adolescents who have previously participated in a trial of oral Tolvaptan patients for the chronic treatment of dilutional hyponatraemia. This study is designed to investigate the post-treatment safety follow-up, and to demonstrate that Tolvaptan safely and effectively achieves and maintains increased sodium concentrations in children and adolescent participants with dilutional hyponatraemia when used for both multiple short-term treatments and/or longer chronic treatments.\nAbout 100 participants aged 4 years to 18 years will be in the study across North America, Canada, and Europe. \nThe core of this study is the follow-up portion, which has a duration of safety follow-up of 6 months.\nThe treatment portion of this study is open-label where children and adolescent participants who have previously participated in a Tolvaptan hyponatraemia trial may be eligible for one or more additional cycles of Tolvaptan treatment.

  • REC name

    London - Bloomsbury Research Ethics Committee

  • REC reference

    16/LO/0724

  • Date of REC Opinion

    13 May 2016

  • REC opinion

    Favourable Opinion