Study to evaluate the safety of crushed defasirox in paediatrics V00
Research type
Research Study
Full title
A single-arm interventional Phase IV, post-authorisation study evaluating the safety of pediatric patients with\ntransfusional hemosiderosis treated with deferasirox crushed film coated tablets
IRAS ID
237540
Contact name
Sara Trompeter
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2016-003482-25
Duration of Study in the UK
1 years, 4 months, 21 days
Research summary
\nDeferasirox film-coated tablet is an iron chelator (i.e. it traps iron) which is a medicine used to remove the excess iron from the body (also called iron overload).\nDeferasirox film-coated tablet was approved in March 2015 by the US Food & Drug Administration and by the European Medicines Agency in March 2016.\nDuring the European Health Authorities evaluation of the deferasirox film-coated tablet application, they expressed the need for further evaluation, especially when the deferasirox film-coated tablets are crushed. This study is being conducted to find out if crushed deferasirox film-coated tablet is safe and has beneficial effects in children aged from 2 years to less than 6 years who have transfusional hemosiderosis. Hemosiderosis is a form of Iron overload caused by multiple blood transfusions.\nThe parent of the children will be asked to crush the tablets and mix with a soft food. The tablets will be given once a day and the study will last for 6 months. \nPatients may have already been on deferasirox or another Iron chelator and can change treatment.\nPatient will be asked to attend the clinic approximately 11 times during the 6 months. The patients will be required to provide blood and urine samples and receive physical examinations. The study will also use questionnaires to assess the experience of taking the drug the taste and any gastrointestinal side effects.\n
REC name
Wales REC 3
REC reference
17/WA/0417
Date of REC Opinion
13 Apr 2018
REC opinion
Further Information Favourable Opinion