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A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions

Phase 2
2 Years
Not Enrolling
Sickle Cell Disease, Iron Overload, Hemolytic Anemia

Thank you

Trial Information

A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions

Inclusion Criteria:

- Age greater than or equal to 2 years

- Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)

- Iron overload from repeated blood transfusion, as defined by one of the following:

1. For patients > 16 years old receiving simple transfusions: lifetime history of
receipt of at least 120 ml/kg or 30 adult units of packed red blood cells, OR

2. For patients ≤ 16 years old receiving simple transfusions: lifetime history of
receipt of at least 120 ml/kg of packed red blood cells, OR

3. For all patients receiving exchange transfusions in the absence of a previous
attempt to achieve negative iron balance: lifetime performance of at least 20
procedures, OR

4. For all patients: liver iron content ≥ 7 mg Fe/g dry weight as measured by
biopsy, Magnetic Resonance Imaging (MRI), or magnetic susceptibility performed
within 3 months prior to entry into screening

- For entry into the screening period: serum ferritin of ≥ 1000 µg/mL on at least two
occasions during the prior year obtained in the absence of concomitant infection.

- Body weight > 10 kg

- No known allergy or contraindication to the administration of deferoxamine

- Ability to comply with all study-related procedures, medications, and evaluations

- Sexually active pre-menopausal 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 by the patient or for pediatric patient's consent of the
patient's legal guardian. The definition of the term 'pediatric' for enrollment and
study conduct will be in accordance with the local legislation.

Exclusion Criteria:

- Serum creatinine above the upper limit of normal

- Significant proteinuria

- History of nephrotic syndrome

- Alanine aminotransferase (ALT) ≥ 250 U/L at screening

- Clinical evidence of active hepatitis B or hepatitis C

- History of HIV

- Fever or other signs/symptoms of infection within 10 days prior to the screening

- Uncontrolled systemic hypertension

- History of Myocardial Infarction, Congestive Heart Failure or unstable cardiac
disease not controlled by standard medical therapy

- Clinically relevant cataract or a previous history of clinically relevant ocular
toxicity related to iron chelation

- Presence of a surgical or medical condition that might significantly alter the
absorption, distribution, metabolism or excretion of any study drug

- History of drug or alcohol abuse within the 12 months prior to enrollment

- Pregnant or breast feeding patients

- Patients treated with systemic investigational drug within 4 weeks prior or with
topical investigational drug 7 days prior to the screening visit

- Randomization in a previous clinical trial involving ICL670

Other protocol-related inclusion / exclusion criteria may apply.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment

Outcome Description:

The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.

Outcome Time Frame:

24 Weeks

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

May 2005

Completion Date:

April 2008

Related Keywords:

  • Sickle Cell Disease
  • Iron Overload
  • Hemolytic Anemia
  • Sickle Cell Disease
  • Iron Overload from Repeated Blood Transfusions
  • Iron Overload
  • Blood Transfusions
  • Anemia
  • Anemia, Hemolytic
  • Anemia, Sickle Cell
  • Iron Overload
  • Hemolysis



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