PETIT:Eltrombopag in PEdiatric patients with Thrombocytopenia from ITP

• Research type

  Research Study

• Full title

  A three part, staggered cohort, open-label and double blind, randomized, placebo controlled study to investigate the eficacy, safety, tolerability and pharmacokinetics of eltrombopag, a thrombopoietin receptor agonist, in previously treated pediatric patients with chronic immune thrombocytopenia (ITP).

• IRAS ID

  23440

• Eudract number

  2006-002946-13

• ISRCTN Number

  0

• Clinicaltrials.gov Identifier

  0

• Research summary

  The purpose of this study is to test the safety of a new medicine, eltrombopag, and to see how well it works in children.Chronic immune thrombocytopenia (ITP) is a blood disease characterised by a low platelet count. Chronic ITP is a term used when the platelet count remains low after 6 months. Platelets are small cells that help control bleeding. As there are fewer platelets in the blood, ITP presents itself as bruising or bleeding. Most children with ITP are not treated unless they have severe bleeding. Eltrombopag is approved in the US for the treatment of ITP in adults. In this study approximately 70 children with chronic ITP will be treated in 3 age based groups (cohorts). The cohorts will be recruited in the following order: Cohort 1 - 12-17yrs, Cohort 2 - 6-11yrs and Cohort 3 - 1-5yrs. In each cohort, 5 patients will be treated initially (part 1) and will receive eltrombopag for a total of 24 weeks, with a review of the data to confirm starting dose and safety information when the 5th patient has reached 12 weeks of treatment. If the review of that data allows progression into part 2, then 18 patients (in the same cohort) will enter a 7 week, double-blind placebo controlled treatment period (2 eltrombopag :1 placebo). The patients are then unblinded and will receive open-label eltrombopag for a total of 24 weeks. The process will start again for the next cohort. Information about eltrombopag will be collected through a number of tests and procedures.This study will be conducted in specialist centres in the UK, EU and USA.

• REC name

  North West - Haydock Research Ethics Committee

• REC reference

  09/H1010/45

• Date of REC Opinion

  18 Sep 2009

• REC opinion

  Further Information Favourable Opinion