Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
Condition: Sickle Cell Disease
MeSH terms: Anemia, Sickle Cell
Intervention: ICL670
Intervention description: Daily doses of ICL670 were taken orally 30 minutes before breakfast. The doses range from 5-40 mg/kg and were determined based on the patient's trend in serum ferritin over time during the core study (0109) and on the frequency of blood transfusions the patient received. The treatment duration was up to 4 years.
Intervention MeSH terms: Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 185.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Deferasirox; ICL670A; Iron chelators; Sickle Cell Disease; Transfusional Hemosiderosis

Summary:

      The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670
      is studied in sickle cell disease patients with transfusional hemosiderosis.
    

Eligibility criteria:

        Inclusion Criteria:

        Patients were included who met the following criteria:

          -  Completion of the core [Study 0109]

          -  Serum ferritin greater than or equal to 500 µg/L

          -  Ability to comply with all study-related procedures, medications, and evaluations

          -  Sexually active post-menarche 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.

          -  Written informed consent and assent by the patient and or their parents or legal
             guardian.

        Additional inclusion criteria for pediatric patients The definition of the term 'pediatric'
        for enrollment and study conduct was in accordance with local law. Parents or the legal
        guardians were fully informed by the investigator as to the requirements of the study. The
        pediatric patients themselves were informed according to their capabilities in a language
        and terms that they were able to understand. Written informed consent was obtained from
        their legal guardian on the patient's behalf in accordance with national legislation. If
        capable, all patients had to also personally sign their written informed consent.

        Exclusion Criteria:

        Patients who met the following criteria were to be excluded:

          -  History of non-compliance to medical regimens and patients who are considered
             potentially unreliable and/or not cooperative

          -  Serum creatinine above the age-appropriate upper limit of normal within one week prior
             to entry

          -  Patients with ALT ≥ 500 U/L within one week prior to entry

          -  Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to
             baseline

          -  Pregnancy (as indicated by serum β-HCG pregnancy test for all female patients with the
             potential to become pregnant) and patients who are breastfeeding

          -  Patients treated with systemic investigational drug within 4 weeks prior to or with
             topical investigational drug within 7 days prior to the baseline visit

        Other protocol-defined inclusion/exclusion criteria may apply