Phase I study to examine the effect of deferasirox on renal hemodynamics in β-thalassemia patients with transfusional iron overload - ND

• Conditions: thalassemia; MedDRA version: 6.1Level: HLGTClassification code 10022958

• Registration Number: EUCTR2006-006838-17-IT
• Lead Sponsor: OVARTIS FARMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-07-16
• Last Update Posted: 26 June 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 8

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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: 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).;Primary end point(s): Absolute change from baseline in GFR, RPF and FF;Main Objective: 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.