E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10022958 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To estimate the effects of deferasirox on changes from baseline in GFR and RPF using 51Cr-EDTA and 123I-Hippuran (I-hip) respectively and the corresponding filtration fraction (FF) in deferasirox naïve patients with β- thalassemia and transfusional iron overload. |
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E.2.2 | Secondary objectives of the trial |
To explore the relationship between absolute changes to baseline in serum creatinine and absolute changes to baseline in GFR, RPF and FF, respectively To explore the relationship between absolute changes to baseline in serum ferritin and absolute changes to baseline in markers of renal function (GFR, RPF, FF and serum creatinine). |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Male or female patients >= 18 years of age without prior history of deferasirox treatment β-thalassemia patients receiving regular transfusions every 2-5 weeks leading to iron intake equal or greater than 0.25 mg/kg/day Transfusion history of >= 20 units of packed red blood cells Serum ferritin values >= 500 µg/L or LIC >= 2 mg/kg of dry weight as measured by SQUID at baseline |
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E.4 | Principal exclusion criteria |
Creatinine above the upper limit of normal range at screening Urine protein/creatinine ratio of > 0.5 mg/mg at screening ALT greater than 5 x ULN at screening History of nephrotic syndrome History of relevant ocular or auditory toxicity related to iron chelation History of HIV positivity History of hypertension, diabetes, hypoproteinemia, obesity Patients with a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of deferasirox or study agents used to measure GFR and RFP at screening Allergy or contraindication to the administration of deferasirox, 51CR-EDTA or 123I-Hippuran Any disease that would prevent the patient from undergoing study treatment and study procedures (including providing informed consent) History of drug or alcohol abuse within the 12 months prior to enrollment Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug within 7 days prior to the screening visit Patients with underlying cardiac disease requiring continuous iron chelation therapy Treatment with drugs that may affect renal parameters (i.e, ACE inhibitors, cyclosporine, etc) Pregnant or breast feeding patients Sexually active pre-menopausal female patients without adequate contraception. Female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months. Inability to comply with all study related procedures, medications and evaluations |
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E.5 End points |
E.5.1 | Primary end point(s) |
Absolute change from baseline in GFR, RPF and FF |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 6 |