Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Information source: Novartis
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis
Intervention: Deferasirox (Drug)
Phase: Phase 3
Status: Active, not recruiting
Sponsored by: Novartis Official(s) and/or principal investigator(s):
Novartis, Study Chair, Affiliation: Novartis
Summary
This is an open-label, non-randomized, six month, 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 due to documented non-compliance, contraindications, unacceptable
toxicities and/or documented poor response.
Clinical Details
Official title: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Study design: Treatment, Non-Randomized, Open Label, Uncontrolled, Safety Study
Primary outcome: Provide expanded access of deferasirox (ICL670) to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusionsFurther evaluate the safety profile of ICL670
Secondary outcome: Evaluate the effect of treatment with ICL670 on changes in serum ferritin
Eligibility
Minimum age: 2 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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 within one week prior to baseline
- Patients with ALT ≥ 500 U/L within one week prior to baseline
- Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to
baseline
- Pregnancy (as indicated by serum β-HCG pregnancy test within 7 days of baseline 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.
Locations and Contacts
Arkansas Children's Hospital, UAMS College of Medicine, Little Rock, Arkansas 72202, United States
Alta Bates Comprehensive Cancer Center, Berkeley, California 94704, United States
Children's Hospital of Orange County, Orange, California 92868, United States
Stanford University, Stanford, California 94305, United States
Children's Hospital and Health Center of San Diego, San Diego, California 92123, United States
Alfred I. Dupong Hospital for Children, Wilmington, Delaware 19803, United States
Hematalogy Oncology Associates, Pensacola, Florida 32501, United States
Osler Medical, Inc., Melbourne, Florida 32901, United States
Tampa Children's Hospital at St. Joseph's Hospital, Tampa, Florida 33607, United States
James A. Haley Veterans Hospital, Tampa, Florida 33612, United States
Backus Children's Hospital, Memorial Health University Medical Center, Savannah, Georgia 31403, United States
Hematalogy Oncology Clinic, Baton Rouge, Louisiana 70808, United States
Borgess Hospital, Kalamazoo, Michigan 49048, United States
Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota 55405, United States
University of Mississippi Medical Center, Jackson, Mississippi 39762, United States
Children's Mercy Hospital, Kansas City, Missouri 64108, United States
The Cancer Institute of New Jersey, New Brunswick, New Jersey 08903, United States
Schneider Children's Hospital, New Hyde Park, New York 11040, United States
PCTI, Columbus, Ohio 43205, United States
The Children's Medical Center of Dayton, Dayton, Ohio 45404, United States
Oregon Health & Science University, Portland, Oregon 97239, United States
Children's Hospitals of Pittsburgh, Pittsburgh, Pennsylvania 15213, United States
Hershey Medical Center, Hershey, Pennsylvania 17033-0850, United States
Texas Children's Hospital, Houston, Texas 77030, United States
Children's Hospital of the Kings Daughters, Norfolk, Virginia 23507, United States
VCU Pediatric Hematology/Oncology, Richmond, Virginia 23219, United States
University of Washington Seattle Cancer Care Alliance, Seattle, Washington 98195, United States
Additional Information
Starting date: October 2005
Last updated: May 1, 2008
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