DEEP-2 Performing an efficacy/safety trial to compare deferiprone with deferasirox

The study – The DEEP-2 study is a Phase III multicentre, randomised, open label, non-inferiority active-controlled trial aiming at comparing for the first time the efficacy of deferiprone (DFP) versus deferasirox (DFX) in paediatric patients aged from 1 month to less than 18 years, affected by hereditary haemoglobinopathies.

Objectives – The main goal of the study is to provide evidence-based measurement of DFP efficacy in comparison with DFX, in terms of changes of serum ferritin levels and cardiac iron overload in paediatric patients affected by hereditary haemoglobinopaties requiring chronic transfusions and chelation.

The DEEP-2 primary outcome is to evaluate the percentage of successfully chelated patients (Time Frame: 12 months), assessed by serum ferritin levels (in all patients) and cardiac MRI T2* (in patients above 10 years of age able to perform an MRI scan without sedation). Secondary objectives of the DEEP-2 trial are the evaluation of the treatment efficacy in terms of hepatic iron concentrations measured by liver MRI (in all patients able to perform MRI scan without sedation), the assessment of the safety and tolerability profiles of the treatment, the healthcare resources utilisation and patient global assessment, including compliance and quality of life (QoL) evaluation, to confirm the influence of demographic covariates on the pharmacokinetic disposition of treatment across a subset of the study population.

Centres Involved – The DEEP-2 study has involved a total of 23 centres: Italy (12), Egypt (3), Greece (1), Albania (2), Cyprus (1), Tunisia (1), UK (3).

Study status –  The trial, was terminated with 393 patients randomized, 316 completed and 77 early terminations.  In particular, the last patient has performed the last visit (V16) on September 21st 2017 at the Nicosia site (ID:05).

At this phase of the study, all the experimental centers are addressed to complete all the following activities:

• completion of the Data Entry activities;
• resolution of the all queries arisen;
• finalization of the IMP’s disposal process;

All the CRAs are performing the last Monitoring and Close out Visits at sites. Moreover, the data cleaning activities are ongoing with the aim to ensure that all data are reliable and correctly processed.