Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01825512|
Recruitment Status : Completed
First Posted : April 5, 2013
Last Update Posted : October 24, 2017
Haemoglobinopathies are a group of inherited disorders characterized by structural variations of the haemoglobin molecule. Most of the patients affected require for survival chronic red blood cells transfusions to overcome ineffective erythropoiesis. Unfortunately, all chronically transfused patients become clinically iron overloaded as there is no physiological mechanism for the removal of iron from the body. The pathologic changes and clinical manifestations associated to chronic iron overload are common among all transfusional iron-overload patients, albeit best documented in patients with beta-thalassemia major. The recommended treatment consists in regular blood transfusions combined with chelating therapy to remove the harmful iron accumulation in the body.
Currently, in the clinical practice particularly in children and adolescents, the criteria leading to the choice of the chelating agent include also the adherence to therapy, thus favouring the use of oral chelators (Ceci A et al., 2011) DFP (Deferiprone) was the first oral chelator authorised in Europe in 1999 as second line treatment for the treatment of iron overload in patients with thalassaemia major when DFO (Deferoxamine) is contraindicated or inadequate. However, despite a wide experience of DFP with iron overloaded (specifically thalassaemic )patients, limited data are available for younger children. For this reason the need for additional data in younger children is expressively included in the 2009 PDCO (Paediatric Committee) Priority List.
The purpose of this study is to assess the non-inferiority of DFP compared to DFX (deferasirox)in paediatric patients affected by hereditary haemoglobinopathies requiring chronic transfusions and chelation. Non inferiority will be established in terms of percentage of patients successfully chelated, as assessed by serum ferritin levels (in all patients) and cardiac MRI T2* (in patients above 10 years of age able to have an MRI scan without sedation).
|Condition or disease||Intervention/treatment||Phase|
|Chronic Iron Overload||Drug: Deferiprone Drug: Deferasirox||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||393 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official 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|
|Actual Study Start Date :||March 17, 2014|
|Actual Primary Completion Date :||August 25, 2017|
|Actual Study Completion Date :||September 21, 2017|
75-100 mg/kg/day seven days per week
Deferiprone 80 mg/mL oral solution
Active Comparator: Deferasirox
20 to 40 mg/kg/day seven days per week
Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg
- Percentage of successfully chelated patients [ Time Frame: twelve months ]Percentage of successfully chelated patients is 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)
- LIC (Liver Iron Concentration) [ Time Frame: twelve months ]It is measured by MRI scan and will be assessed in all patients able to undergo MRI scan without sedation
- Safety and tolerability [ Time Frame: monthly ]Collection of adverse events (nature, severity, grade, duration), the monitoring of haematology, blood chemistry and urine values
- Health-related quality of life [ Time Frame: twelve months ]It is evaluated using the Child Health Questionnaire™ (CHQ) in the subgroup of patients for which the questionnaire is validated (5-18 years)
- Steady state concentration (Css) of DFP and DFX [ Time Frame: twelve months ]Assessment of drug concentrations at steady-state conditions. PK data will be analysed to confirm the influence of relevant covariates on the systemic exposure to Deferiprone.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01825512
|Study Director:||Donato Bonifazi, Dr||Consorzio per Valutazioni Biologiche e Farmacologiche|
|Principal Investigator:||Aurelio Maggio, MD||Ospedali Riuniti Villa Sofia-Cervello|