Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
Sickle Cell Disease, Iron Overload, Hemolytic Anemia
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring Sickle Cell Disease, Iron Overload from Repeated Blood Transfusions, Iron Overload, Blood Transfusions
Eligibility Criteria
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: 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 For patients ≤ 16 years old receiving simple transfusions: lifetime history of receipt of at least 120 ml/kg of packed red blood cells, OR 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 For all patients: liver iron content ≥ 7 mg Fe/g dry weight as measured by biopsy, Magnetic Resonance Imaging (MRI), or magnetic susceptibility performed within 3 months prior to entry into screening 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 Alanine aminotransferase (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 Myocardial Infarction, Congestive Heart Failure 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.
Sites / Locations
- University of Alabama Pediatric Hematology/Oncology
- University of Alabama Medical center
- University of South Alabama Medical Center
- University of South Alabama
- Loma Linda University Medical Center
- Children's Hospital Oakland
- Center for Cancer and Blood Disorders
- Children's National Medical Center
- Howard University Hospital
- Miami Children's Hospital
- Tampa Children's Hospital at St Joseph's
- Tampa Children's Hospital at St. Joseph's
- H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans Hospital
- Emory University School of Medicine
- Children's Healthcare of Atlanta at Scottish Rite
- Adult Sickle Cell Clinic
- Backus Children's Hospital
- University of Illinois at Chicago
- Children's Memorial Hospital
- Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for Children
- St. Jude Children's Hospital Affiliate
- Tulane University Sickle Cell Center
- Children's Hospital
- LSU Health Sciences Center/Carroll W. Feist Professor of Cancer Research
- Brigham and Woman's Hospital/Harvard Medical School
- Children's Hospital Boston
- Children's Hospital
- University of Michigan
- Karmanos Cancer Institute
- Washington University School of Medicine
- Sickle Cell Center, Montefiore Hospital
- SUNY Downstate Medical Center
- New York Methodist Hospital
- Weill Medical College of Cornell University
- Columbia University
- Carolinas Medical Transplant Center
- University of Cincinnati
- Children's Hospital Medical Center
- The University of Oklahoma
- Children's Hospital of Philadelphia
- Pennsylvania Oncology/Hematology
- Jefferson University
- Drexel University College of Medicine
- Children's Hospital of Pittsburgh
- Hillman Cancer Center
- Liberty Hematology Oncology Center
- Palmetto Health Clinical Trials
- Santee Hematology/Oncology
- St Jude's Children's Research Hospital
- St. Jude's Children Research Hospital
- Cooks Children's Hospital
- Texas Children's Hospital/Baylor College of Medicine
- Scott and White Memorial Hospital & Clinics
- Children's Hospital of the King's Daughter
- Medical College of Virginia
- Virginia Commonwealth University
- University of Alberta
- The Ottawa Hospital
- Hopital Ste-Justine
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Deferasirox (ICL670)
Deferoxamine (DFO) then ICL670
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.