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A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators

Phase 3
2 Years
Not Enrolling
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis

Thank you

Trial Information

A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators

Inclusion Criteria:

- Male or female patients greater than or equal to 2 years of age

- Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle
cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions

- Cannot be adequately treated with a locally approved iron chelator due to one of the
following reasons:

- Documented non-compliance, defined as having taken less than 50% of the
prescribed chelation therapy doses in the 12 months prior to study entry

- Contraindications, unacceptable toxicities and/or documented poor response to
locally approved iron chelators despite proper compliance

- History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red
blood cells (PRBC])

- Serum ferritin value greater than or equal to 1000 µg/L

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

Exclusion Criteria:

- Ongoing treatment with another iron chelator (Any other iron chelation therapy must
be discontinued at least 24 hours prior to study entry.)

- Patients who meet the eligibility criteria for any other ongoing Novartis sponsored
clinical study protocol with deferasirox and who have geographic access to these

- Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment
with deferasirox

- Serum creatinine above the upper limit of normal at screening.

- Patients with ALT ≥ 500 U/L at screening.

- Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to

- Pregnancy (as indicated by serum β-HCG pregnancy test at screening 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.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events

Outcome Description:

Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.

Outcome Time Frame:

Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 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:

October 2005

Completion Date:

October 2008

Related Keywords:

  • Thalassemia
  • Sickle Cell Disease
  • Diamond Blackfan Anemia
  • Myelofibrosis
  • Deferasirox
  • Congenital Anemias
  • Anemias
  • Red Blood Cell Disorders
  • Chronic Iron Overload
  • Transfusional Iron Overload
  • Iron Chelators
  • Oral Iron Chelators
  • Thalassemia
  • Sickle Cell Disease
  • Diamond Blackfan Anemia
  • Myelofibrosis
  • ICL670A
  • Primary Myelofibrosis
  • Anemia
  • Anemia, Sickle Cell
  • Thalassemia
  • Iron Overload
  • Anemia, Diamond-Blackfan



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