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

Phase 3

Completed

Conditions: Diamond Blackfan Anemia
Thalassemia
Sickle Cell Disease
Myelofibrosis

Brief Summary: 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.

Recruitment & Eligibility

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.

Arm && Interventions

Group	Intervention	Description
Deferasirox	Deferasirox	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.

Primary Outcome Measures

Name	Time	Method
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events	Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)	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.

Secondary Outcome Measures

Name	Time	Method
The Change in Serum Ferritin Values From Baseline Through Completion of the Study	Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)	The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (\<1000), (1000-\<2500), (2500-\<4000), (4000-\<5500), (5500-\<7000) and (\>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.

Locations (138): Arkansas Children's Hospital, UAMS College of Medicine
🇺🇸
Little Rock, Arkansas, United States
Alta Bates Comprehensive Cancer Center
🇺🇸
Berkeley, California, United States
Children's Hospital of Orange County
🇺🇸
Orange, California, United States
Children's Hospital and Health Center of San Diego
🇺🇸
San Diego, California, United States
Stanford University
🇺🇸
Stanford, California, United States
Alfred I. Dupong Hospital for Children
🇺🇸
Wilmington, Delaware, United States
Osler Medical, Inc.
🇺🇸
Melbourne, Florida, United States
Hematalogy Oncology Associates
🇺🇸
Pensacola, Florida, United States
Tampa Children's Hospital at St. Joseph's Hospital
🇺🇸
Tampa, Florida, United States
James A. Haley Veterans Hospital
🇺🇸
Tampa, Florida, United States
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Arkansas Children's Hospital, UAMS College of Medicine
🇺🇸Little Rock, Arkansas, United States