Chronic iron overload MedDRA version: 14.1 Level: LLT Classification code 10065974 Term: Chronic iron overload System Organ Class: 100000004861
Conditions
Interventions
Product Name: DEFERIPRONE
Product Code: NA
Pharmaceutical Form: Oral solution
INN or Proposed INN: DEFERIPRONE
CAS Number: 30652-11-0
Concentration unit: mg/ml milligram(s)/millilitre
Concentration ty
Sponsors
CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients on current treatment with 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); 2. 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; 3. For patients aged from 1 month to less than 6 years: known intolerance or contraindication to deferoxamine; 4. Written informed consent and patient's informed assent to patient’s maturity and understanding Are the trial subjects under 18? yes Number of subjects for this age range: 344 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients with known intolerance or contraindication to either DFP or DFX 2. Patients receiving DFX at a dose > 40 mg/kg/day or DFP at a dose > 100 mg/kg/day at screening 3. Platelet count ULN for age during the run-in phase 10. History of significant medical or psychiatric disorder 11. The patient has received another investigational drug within 30 days prior to this study 12. Fever and other signs/symptoms of infection in the 10 days before baseline assessment 13. Concomitant use of trivalent cation-dependent medicinal products such as aluminium-based antacids 14. Positive test for ß-HCG
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: 1. to assess treatment efficacy in terms of ferritin levels 2. to assess treatment efficacy in terms of cardiac MRI T2* in patients over 10 years of age able to perform MRI scan without sedation 3. to estimate the efficacy of treatments in terms of liver iron concentration (LIC) measured by MRI in all paediatric subjects able to have MRI scan without sedation 4. to evaluate the safety and tolerability profile of treatments 5. to assess healthcare resources utilisation and patient global assessment, including compliance and quality of life evaluation 6. to confirm the relationship of demographic covariates and the disposition of DFP across the study population.;Timepoint(s) of evaluation of this end point: Serum ferritine will be measured every 3 months Cardiac MRI (Magnetic Resonance Imaging) T2* will be measured at month 1, 6 and 12 of treatment. Primary endpoint in terms of percentage of successfully chelated patients will be assessed as difference between basal and final (12 months) levels;Main Objective: To assess the non-inferiority of Deferiprone compared to Deferasirox ìn terms of changes in ferritin levels and cardiac iron concentration;Primary end point(s): 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). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Liver iron concentration (LlC) as measured by MRI (Magnetic Resonance Imaging) in patients able to undergo MRI scan without sedation. 2. Safety and tolerability assessments 3. Quality of Life;Timepoint(s) of evaluation of this end point: 1. LCI will be assessed at baseline and at the end of treatment (12 months); 2. Adverse events (nature, severity, grade, duration) will be recorded monthly 3. Quality of Life will be assessed at month 1 and 12 of treatment. | — |
Countries
Albania, Egypt, Greece, Italy, Tunisia, United Kingdom
Contacts
Public ContactDirezione Scientifica
Consorzio per le Valutazioni Biologiche e Farmacologiche
Outcome results
None listed