A one-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral ICL670 (20 mg/kg/day) in patients diagnosed with transfusion-dependent iron overloadEstudio multicéntrico, abierto, de un solo grupo y de un año de duración, para evaluar la eficacia y seguridad de ICL670 (20 mg/kg/día) administrado vía oral en pacientes diagnosticados de sobrecarga férrica asociada a transfusiones

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 1541

Inclusion Criteria

• transfusion-dependent anemias (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of = 1000 ng/ml
•age = 2 years
• Adult patients: written informed consent
• Pediatric patients: written informed consent by their parents or legal guardians on the patient’s behalf in accordance with the national legislation. If capable, all patients should also personally sign their written informed assent.

Are the trial subjects under 18? yes
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

• non-transfusional hemosiderosis
• clinical evidence supporting the need of intensive chelation
• mean levels of alanine aminotransferase (ALT) > 300 U/l
• uncontrolled systemic hypertension
• serum creatinine above the upper limit of normal (ULN)
• significant proteinuria
• history of nephrotic syndrome
• third degree atrioventricular block, clinically relevant Q-T interval prolongation, patients requiring treatment with digoxin and similar compounds or drugs which may induce prolongation of the Q-T interval
• history of clinically relevant ocular toxicity related to iron chelation
• systemic diseases which would prevent the patient from undergoing study treatment
• psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
• pregnant or breastfeeding patients
• treatment with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
• any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug
• history or likelihood of non-compliance to medical regimens
• history of drug or alcohol abuse within the past 12 months or evidence of such abuse during the run-in period
• positive test to HIV
•life expectancy of < 1year
• pediatric patients only: body weight which prevents the use of the smallest tablet strength (125 mg) for proper dosing

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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