Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01825512
• Date of registration: 03/04/2013
• Prospective Registration: Yes
• Primary sponsor: Consorzio per Valutazioni Biologiche e Farmacologiche
• Public title: Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
• Scientific title: Multicentre, Randomised, Open Label, Non-inferiority Trial to Evaluate the Efficacy and Safety of Deferiprone Compared to Deferasirox in Patients Aged From 1 Month to Less Than 18 Years Affected by Transfusion Dependent Haemoglobinopathies
• Date of first enrolment: March 17, 2014
• Target sample size: 393
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01825512
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Albania, Cyprus, Egypt, Greece, Italy, Tunisia, United Kingdom

Contacts

• Name: Aurelio Maggio, MD
• Affiliation: Ospedali Riuniti Villa Sofia-Cervello

• Name: Donato Bonifazi, Dr
• Affiliation: Consorzio per Valutazioni Biologiche e Farmacologiche

Key inclusion & exclusion 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 <100.000/mm3 during the run-in phase

- Absolute neutrophils count <1.500/mm3 during the run-in phase

- Hb levels lower than 8g/dL during the run-in phase

- Evidence of abnormal liver function

- Iron overload from causes other than transfusional haemosiderosis

- Severe heart dysfunction secondary to iron overload

- Serum creatinine level > 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 ß-HCG (Human chorionic gonadotropin) and lactating female patients



Age minimum: 1 Month
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Iron Overload

Intervention(s)

Drug: Deferasirox

Drug: Deferiprone

Primary Outcome(s)

Percentage of successfully chelated patients [Time Frame: twelve months]

Secondary Outcome(s)

Health-related quality of life [Time Frame: twelve months]

LIC (Liver Iron Concentration) [Time Frame: twelve months]

Steady state concentration (Css) of DFP and DFX [Time Frame: twelve months]

Safety and tolerability [Time Frame: monthly]

Secondary ID(s)

DEEP-2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

European Commission