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
Pharmaceutical Form: Oral solution
Sponsors
Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: a) Patients in a chronic transfusional program who have received at least 150 ml/kg/year of packed red blood cells (corresponding approximately to 12 transfusions) and on current treatment with DFO, DFX, DFP; aged from 1 month to less than 6 years. b) Patients naïve to any chelation treatment who have received not less than 150 ml/kg of packed red blood cells (corresponding to approximately 12 transfusions) and have ferritin levels > 800 ng/mL, aged from 1 month to less than 6 years. c) Patients who meet the transfusion criteria (150 ml/kg/year corresponding approximately to 12 transfusions) and have known intolerance or contraindication to DFO And if all of the following criteria apply: d) Patients affected by any hereditary haemoglobinopathies requiring chronic transfusion therapy including but not limited to thalassaemia and sickle cell disease e) Written informed consent obtained from their legal guardian on the patient's behalf in accordance with the national legislations. According to his/her capability, patient's informed assent will be collected Are the trial subjects under 18? yes Number of subjects for this age range: 30 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: a) Patient with known intolerance or contraindication to the trial treatment b) Patient with Hb levels less than 8g/dl (entry may be delayed until values return to normal) c) Patient with platelet count 5 times the upper normal limit during six months preceding enrolment; entry may be delayed until values return to normal) e) iron overload from causes other than trasfusional haemosiderosis f) severe heart dysfunction secondary to iron overload defined as the occurrence of heart failure or severe arrhythmia or as indicated by cardiac T2* lower than 10 ms, if recent MRI data is available, g) Patient with serum creatinine level above the upper normal limit at screening; entry may be delayed until values return to normal. h) Serological evidence of chronic hepatitis B (presence of HBe Ag, HBsAg, HBcAb-IgM, in the absence of HBsAb). i) History of significant medical or psychiatric disorder that may impair compliance with the requirements of the protocol. j) The patient has received another investigational drug within 30 days prior to this study. k) Patient with a pre-existing condition or any other surgical or medical condition which might significantly interfere with normal gastrointestinal and hepatic function that could alter the absorption, metabolism, and/or excretion of the study drug. l) Patient with a known history of HIV seropositivity. m) Fever and other signs/symptoms of infection in the 10 days before drug administration(treatment day) n) Concomitant use of other iron chelators or trivalent cation-dependent medicinal products such as aluminium-based antacids. o) Patient with a chronic condition that does not allow suspension of related treatment from starting of washout until drug is administered.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the pharmacokinetics of deferiprone in paediatric patients aged from 1 month to less than 6 years;Secondary Objective: 1) To identify dose levels yielding deferiprone exposures comparable to adults and define the dose rationale in children aged from 1 month to less than 6 years. 2) To evaluate safety and tolerability of deferiprone after single dose administration in children aged from 1 month to less than 6 years. 3) To evaluate the effect of demographic covariates on DFP disposition and estimate the clearance distribution across the population.;Primary end point(s): Pharmacokinetic endpoints derived from the PK study will include: • Primary PK parameters: CL/F, Vd/F, Ka • Secondary PK parameters: AUC, Cmax,Tmax, Css and Cmin;Timepoint(s) of evaluation of this end point: Samples will be collected from pre-dose up to 8 hours post-dose on a single day after drug administration | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary pharmacodynamic/biomarker endpoints will include: Clinical safety and tolerability data including ferritin levels, spontaneous AE reporting, ECGs, vital signs, nursing/physician observation and clinical laboratory values (haematology and biochemistry).;Timepoint(s) of evaluation of this end point: Blood samples for clinical chemistry and haematology will be collected at screening and during follow up and early termination visit if a patient withdraws or is withdrawn from the study. Ferritin levels will be collected only at screening. Adverse events, vital signs and ECG will also be monitored throughout the study. | — |
Countries
Egypt
Contacts
Public ContactDirezione Scientifica
Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF)
Outcome results
None listed