Status:

COMPLETED

Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis

Lead Sponsor:

Novartis Pharmaceuticals

Conditions:

Chronic Anemia

Transfusional Hemosiderosis

Eligibility:

All Genders

2+ years

Phase:

PHASE2

Brief Summary

The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anem...

Eligibility Criteria

Inclusion

  • Inclusion criteria (Core):
  • Patients with transfusional iron overload due to:
  • low or intermediate (INT-1) risk Myelodysplastic Syndrome (MDS)determined via International Prognosis Scoring System (IPSS) criteria
  • other congenital or acquired anemias excluding B-thalassemia and sickle cell disease
  • Lifetime transfusion history of ≥20 unit (approximately 100 mL/kg) of packed red blood cells or showing evidence of iron overload (serum ferritin \>1000 µg/L).
  • Able to provide written informed consent
  • Life expectancy ≥ 12 months If patient was previously treated with deferiprone, a washout period of one month should occur before the first dose of deferasirox
  • Inclusion criteria (Extension):
  • Patients completing the planned 12-month core study (CICL670A2204).
  • Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with national legislation.
  • Exclusion criteria (Core and Extension):
  • Patients with β-thalassemia, sickle cell disease or myelodysplastic syndrome with an IPSS score being Intermediate-2 or High.
  • Patients with serum creatinine \> ULN
  • Patients with ALT(SGPT) levels \> 5 x ULN
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio \>0.5 mg/mg in a non-first void urine sample on two assessments during the screening period.
  • History of HIV positive test result , or of clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal range)
  • Patients on investigational MDS therapies, including lenalidomide, thalidomide, azacitidine and arsenic trioxide, must have a ≥ 4 week washout period prior to the first dose of study drug.
  • Patients with systemic uncontrolled hypertension
  • Patients with unstable cardiac disease not controlled by standard medical therapy
  • Systemic disease (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
  • Pregnancy (as documented in required screening laboratory test) or breast feeding.
  • Patients treated with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
  • Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
  • Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the study protocol
  • History of hypersensitivity to any of the study drug or excipients
  • Sexually active pre-menopausal female patients without adequate contraception. Female patients must use effective contraception or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
  • Other protocol defined inclusion/exclusion criteria may apply.

Exclusion

    Key Trial Info

    Start Date :

    October 1 2007

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    February 1 2012

    Estimated Enrollment :

    102 Patients enrolled

    Trial Details

    Trial ID

    NCT00631163

    Start Date

    October 1 2007

    End Date

    February 1 2012

    Last Update

    June 29 2021

    Active Locations (31)

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    Page 1 of 8 (31 locations)

    1

    Novartis Investigative Site

    Nagoya, Aichi-ken, Japan, 453-8511

    2

    Novartis Investigative Site

    Nagoya, Aichi-ken, Japan, 466-8560

    3

    Novartis Investigative Site

    Fukuoka, Fukuoka, Japan, 814-0180

    4

    Novartis Investigative Site

    Sapporo, Hokkaido, Japan, 060-8543