DEferiprone Evaluation in Paediatrics
Research type
Research Study
Full title
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate the efficacy and safety of deferiprone compared to deferasirox in paediatric patients aged from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies
IRAS ID
148257
Contact name
Paul Telfer
Contact email
Eudract number
2012-000353-31
Clinicaltrials.gov Identifier
Research summary
DEEP-2 trial is aimed to evaluate the efficacy and safety of deferiprone compared to deferasirox in paediatric patients aged from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies. Deferiprone is intended to be administered as a new oral formulation specifically developed for children.
The primary objective is to assess the non-inferiority of deferiprone compared to Deferasirox ìn terms of changes in ferritin levels and cardiac iron concentration in paediatric patients affected by hereditary haemoglobinopaties requiring chronic transfusions and chelation.
Accordingly, the primary endpoint is the percentage of successfully chelated patients assessed by serum ferritin levels (all patients) and cardiac MRI T2* (patients above 10 years of age able to have an MRI scan without sedation).
In addition, Quality of Life will be also assessed during the trial.
344 paediatric patients from 7 different countries (both European and non-European) are planned to be recruited.
The overall duration of the study is 13-14 months: run-in phase (28 days) including a wash-out period (6 days); treatment phase (12 months); follow-up phase (1 month).REC name
London - West London & GTAC Research Ethics Committee
REC reference
14/LO/1403
Date of REC Opinion
26 Jan 2015
REC opinion
Further Information Favourable Opinion