Phase 3
Completed N=393
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Chronic Iron Overload
Source: ClinicalTrials.gov NCT01825512 ↗
Enrolled (actual)
393
Serious AEs
6.9%
Results posted
Apr 2021
Primary outcomePrimary: Percentage of Successfully Chelated Patients — 69; 80 Participants
◆ Published Evidence
Established
73citations · ~12 / year
Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial.
Summary
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40 mg/kg/day in paediatric patients (1 month < 18 years old) affected by hereditary haemoglobinopathies and requiring frequent transfusions and chelation.
Linked Publications (2)
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Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial.
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Ethical and procedural issues for applying researcher-driven multi-national paediatric clinical trials in and outside the European Union: the challenging experience of the DEEP project.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Successfully Chelated Patients |
69; 80 | — |
| SECONDARY Liver MRI |
-0.848; -2.975 | 0.074 |
| SECONDARY Cardiac MRI T2* |
0.488; 1.121 | 0.717 |
| SECONDARY Ferritin Level |
-397.583; -398.184 | 0.997 |
Eligibility Criteria
Inclusion Criteria
- Patients of both genders aged from 1 month up to less than 18 years at the time of enrolment
- Patients affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassemia syndromes and sickle cell disease
- Patients on current treatment with deferoxamine (DFO) or DFX or DFP in a chronic transfusion program receiving at least 150 mL/kg/year of packed red blood cells (corresponding approximately to 12 transfusions);
- For patients naïve to chelation treatment: patients that have received at least 150 mL/kg of packed red blood cells (corresponding to approximately 12 transfusions) in a chronic transfusion program and with serum ferritin levels ≥ 800 ng/mL;
- Until availability of results from the PK Study (Study DEEP-1, EudraCT n. 2012-000658-67) for patients aged from 1 month to less than 6 years: known intolerance or contraindication to DFO;
- Written informed consent and patient's informed assent, relating to his/her comprehension abilities and level of maturity
Exclusion Criteria
- Patients with intolerance or known contraindication to either DFP or DFX
- Patients receiving DFX at a dose > 40 mg/kg/day or DFP at a dose > 100 mg/kg/day at screening
- Platelet count ULN (Upper Limit of Normal) for age during the run-in phase
- History of significant medical or psychiatric disorder
- The patient has received another investigational drug within 30 days prior to this clinical trial
- Fever and other signs/symptoms of infection in the 10 days before baseline assessment
- Concomitant use of trivalent cation-dependent medicinal products such as aluminium-based antacids
- Positive test for β-HCG (Human chorionic gonadotropin) and lactating female patients
Data sourced from ClinicalTrials.gov (NCT01825512) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.