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

Completed · Phase 3

Conditions studied: Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis

In brief

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.

Key facts

Study ID

NCT00235391

Run by

Novartis Pharmaceuticals

People needed

1683

Starts

2005-10-01

Expected to finish

2008-10-01

Last updated by the study team

2011-06-07

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• 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
• Backus Children's Hospital, Memorial Health University Medical Center — Savannah, Georgia, United States
• Hematalogy Oncology Clinic — Baton Rouge, Louisiana, United States
• Borgess Hospital — Kalamazoo, Michigan, United States
• Children's Hospitals and Clinics of Minnesota — Minneapolis, Minnesota, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• The Cancer Institute of New Jersey — New Brunswick, New Jersey, United States
• Schneider Children's Hospital — New Hyde Park, New York, United States
• PCTI — Columbus, Ohio, United States
• The Children's Medical Center of Dayton — Dayton, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Hershey Medical Center — Hershey, Pennsylvania, United States
• Children's Hospitals of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Texas Children's Hospital — Houston, Texas, United States
• Children's Hospital of the Kings Daughters — Norfolk, Virginia, United States
• Arkansas Children's Hospital, UAMS College of Medicine — Little Rock, Arkansas, United States

Full record on ClinicalTrials.gov

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