Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Novartis

Status and phase: Completed; Phase 3

Conditions: Myelofibrosis; Thalassemia; Diamond Blackfan Anemia; Sickle Cell Disease

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00235391

CICL670A2203

Details and patient eligibility

About

This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.

Enrollment: 1,683 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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 sites
• 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 baseline
• 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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1,683 participants in 1 patient group

Deferasirox

Experimental group

Description:

Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.

Treatment:

Drug: Deferasirox