Study of a new medicine for children who bruise and bleed easily
Research type
Research Study
Full title
A two part, doouble-blind,randomized, placebo-controlled and open-label study to investigate the efficacy, safety and tolerability of eltrombopag, a thrombopoietin receptor agonist, in paediatric patients with previously treated chronic immune (idiopathic) thrombocytopenic purpura (ITP).
IRAS ID
96106
Contact name
John David Grainger
Contact email
Sponsor organisation
GSK UK Plc
Eudract number
2011-002184-17
Research summary
The purpose of this study is to provide information on the safety, efficacy and optimum dosing regimens for eltrombopag in children with idiopathic thrombocytopenic purpura (ITP). Eltrombopag is already approved in a number of countries for the treatment of ITP in adults.ITP is a blood disease characterised by a low platelet count. Chronic ITP is a term used when the platelet count remains low after one year. Platelets are small cells that help control bleeding. As there are fewer platelets in the blood, ITP presents as bruising or bleeding. Most children with ITP are not treated unless they have severe bleeding. In this study approximately 75 children (including 12 from the UK) with previously treated chronic ITP will be treated in three age-based groups (cohorts): 1-5 years, 6-11 years and 12-17 years at enrollment. At least 25 children will be enrolled in each of the upper two cohorts and at least 12 in the youngest age cohort. In Part 1, children in each cohort will be randomised in a ratio of 2:1 to receive eltrombopag or placebo for a 13-week period. After verification and locking of the data for each child the investigator will be unblinded in order to ensure a safe starting dose is used in Part 2 - a 24-week open label period of treatment with eltrombopag. Throughout the study, the dose can be individualised for each patient based on their platelet response. Children receiving eltrombopag in Part 1 will receive a total of 37 weeks of treatment with eltrombopag. A six month follow up period completes the study. This study will be conducted in specialist centres in Europe (including UK) and the USA.
REC name
North West - Haydock Research Ethics Committee
REC reference
12/NW/0134
Date of REC Opinion
17 Feb 2012
REC opinion
Favourable Opinion