This study will examine the long-term safety and efficacy of Deferasirox in patients with
sickle cell disease and iron overload from repeated blood transfusions.
Inclusion Criteria
- Age greater than or equal to 2 years
- Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)
- Iron overload from repeated blood transfusion, as defined by one of the following:
1. For patients > 16 years old receiving simple transfusions: lifetime history of
receipt of at least 120 ml/kg or 30 adult units of packed red blood cells, OR
2. For patients ≤ 16 years old receiving simple transfusions: lifetime history of
receipt of at least 120 ml/kg of packed red blood cells, OR
3. For all patients receiving exchange transfusions in the absence of a previous
attempt to achieve negative iron balance: lifetime performance of at least 20
procedures, OR
4. For all patients: liver iron content ≥ 7 mg Fe/g
- For entry into the screening period: serum ferritin of ≥ 1000 µg/mL on at least two
occasions during the prior year obtained in the absence of concomitant infection.
- Body weight > 10 kg
- No known allergy or contraindication to the administration of deferoxamine
- Ability to comply with all study-related procedures, medications, and evaluations
- Sexually active pre-menopausal female patients must use double-barrier contraception,
oral contraceptive plus barrier contraceptive, or must have undergone clinically
documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal
defined by amenorrhea for at least 12 months.
- Written informed consent by the patient or for pediatric patient's consent of the
patient's legal guardian. The definition of the term 'pediatric' for enrollment and
study conduct will be in accordance with the local legislation.
Exclusion Criteria
- Serum creatinine above the upper limit of normal
- Significant proteinuria
- History of nephrotic syndrome
- ALT ≥ 250 U/L at screening
- Clinical evidence of active hepatitis B or hepatitis C
- History of HIV
- Fever or other signs/symptoms of infection within 10 days prior to the screening
visit
- Uncontrolled systemic hypertension
- History of MI, CHF or unstable cardiac disease not controlled by standard medical
therapy
- Clinically relevant cataract or a previous history of clinically relevant ocular
toxicity related to iron chelation
- Presence of a surgical or medical condition that might significantly alter the
absorption, distribution, metabolism or excretion of any study drug
- History of drug or alcohol abuse within the 12 months prior to enrollment
- Pregnant or breast feeding patients
- Patients treated with systemic investigational drug within 4 weeks prior or with
topical investigational drug 7 days prior to the screening visit
- Randomization in a previous clinical trial involving ICL670
Other protocol-related inclusion / exclusion criteria may apply.
University of Alabama Pediatric Hematology/Oncology, Birmingham, Alabama 35233, United States
University of South Alabama Medical Center, Mobile, Alabama 36604, United States
University of South Alabama, Mobile, Alabama 36617, United States
Loma Linda University Medical Center, Loma Linda, California 92354, United States
Children's Hospital Oakland, Oakland, California 94609, United States
Howard University Hospital, Washington, District of Columbia 20059, United States
Center for Cancer and Blood Disorders, Washington, District of Columbia 20010-2970, United States
Tampa Children's Hospital at St. Joseph's, Tampa, Florida 33607-6387, United States
Miami Children's Hospital, Miami, Florida 33155, United States
James A. Haley Veterans Hospital, Tampa, Florida 33612, United States
Tampa Children's Hospital at St Joseph's, Tampa, Florida 33607-6387, United States
Miami Children's Hospital, Miami, Florida 33155, United States
Adult Sickle Cell Clinic, Augusta, Georgia 30912-3128, United States
Backus Children's Hospital, Savannah, Georgia 31403, United States
Children's Healthcare of Atlanta at Scottish Rite, Atlanta, Georgia 30342, United States
Emory University School of Medicine, Atlanta, Georgia 30303, United States
Children's Memorial Hospital, Chicago, Illinois 60614-3394, United States
University of Illinois at Chicago, Chicago, Illinois 60612, United States
Pediatric Sickle Cell Program, Indianapolis, Indiana 46202, United States
Tulane University Sickle Cell Center, New Orleans, Louisiana 70112, United States
Children's Hospital, New Orleans, Louisiana 70118, United States
Carroll W. Feist Professor of Cancer Research, Shreveport, Louisiana 71130, United States
St. Jude Children's Hospital Affiliate, Baton Rouge, Louisiana 70808, United States
Children's Hospital, Boston, Massachusetts 02115, United States
Lecturer in Medicine, Harvard Medical School, Boston, Massachusetts 02115, United States
Children's Hospital Boston, Boston, Massachusetts 02115, United States
Karmanos Cancer Institute, Detroit, Michigan 48201, United States
University of Michigan, Ann Arbor, Michigan 48109-0238, United States
Washington University School of Medicine, St. Louis, Missouri 63110, United States
Washington University School of Medicine, St. Louis, Missouri 63110, United States
Columbia University, New York, New York 10032, United States
SUNY Downstate Medical Center, Brooklyn, New York 11203, United States
Sickle Cell Center, Montefiore Hospital, Bronx, New York 10467-2490, United States
Weill Medical College of Cornell University, New York, New York 10021, United States
New York Methodist Hospital, Brooklyn, New York 11215, United States
Carolinas Medical Center, Charlotte, North Carolina 28232, United States
University of Cincinnati, Cincinnati, Ohio 45219, United States
Children's Hospital Medical Center, Cincinnati, Ohio 72764, United States
The University of Oklahoma, Oklahoma City, Oklahoma 73104, United States
Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania 15213, United States
Pennsylvania Oncology/Hematology, Philadelphia, Pennsylvania 19106, United States
Hillman Cancer Center, Pittsburgh, Pennsylvania 15232, United States
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104-4399, United States
Drexel University College of Medicine, Philadelphia, Pennsylvania 19134-1095, United States
Jefferson University, Philadelphia, Pennsylvania 19107, United States
Liberty Hematology Oncology Center, Columbia, South Carolina 29203, United States
Palmetto Health Clinical Trials, Columbia, South Carolina 29203, United States
Santee Hematology/Oncology, Sumter, South Carolina 29150, United States
St Jude's Children's Research Hospital, Memphis, Tennessee 38105-2794, United States
Cooks Children's Hospital, Fort Worth, Texas 76104-2724, United States
Scott and White Memorial Hospital & Clinics, Temple, Texas 76508, United States
Texas Children's Hospital/Baylor College of Medicine, Houston, Texas 77030-2399, United States
Virginia Commonwealth University, Richmond, Virginia 23298-0646, United States
Children's Hospital of the King's Daughter, Norfolk, Virginia 23507, United States
Medical College of Virginia, Richmond, Virginia 23298-0306, United States
Medical College of Virginia, Richmond, Virginia 23298-0306, United States
