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A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions

Phase 2
2 Years
Not Enrolling
Sickle Cell Disease, Iron Overload, Hemolytic Anemia

Thank you

Trial Information

A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With 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 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

- 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.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment

Outcome Description:

The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.

Outcome Time Frame:

24 Weeks

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

May 2005

Completion Date:

April 2008

Related Keywords:

  • Sickle Cell Disease
  • Iron Overload
  • Hemolytic Anemia
  • Sickle Cell Disease
  • Iron Overload from Repeated Blood Transfusions
  • Iron Overload
  • Blood Transfusions
  • Anemia
  • Anemia, Hemolytic
  • Anemia, Sickle Cell
  • Iron Overload
  • Hemolysis



Howard University Hospital Washington, District of Columbia  20060
University of Michigan Ann Arbor, Michigan  48109-0624
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Washington University School of Medicine Saint Louis, Missouri  63110
Loma Linda University Medical Center Loma Linda, California  92354
New York Methodist Hospital Brooklyn, New York  11215-3609
Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania  15213
University of South Alabama Medical Center Mobile, Alabama  36604
Miami Children's Hospital Miami, Florida  33155-4069
Weill Medical College of Cornell University New York, New York  10021
University of Illinois at Chicago Chicago, Illinois  60612
SUNY Downstate Medical Center Brooklyn, New York  11203
Emory University School of Medicine Atlanta, Georgia  30322
Columbia University New York, New York  10032-3784
Children's Hospital Boston Boston, Massachusetts  02115
Medical College of Virginia Richmond, Virginia  23298-0341
Children's Memorial Hospital Chicago, Illinois  60614
Santee Hematology/Oncology Sumter, South Carolina  29150
Virginia Commonwealth University Richmond, Virginia  
Karmanos Cancer Institute Detroit, Michigan  48201
Children's Hospital Oakland Oakland, California  94609
Drexel University College of Medicine Philadelphia, Pennsylvania  19129
University of Cincinnati Cincinnati, Ohio  45267-0502
Tampa Children's Hospital at St Joseph's Tampa, Florida  33607
Tulane University Sickle Cell Center New Orleans, Louisiana  70112
Sickle Cell Center, Montefiore Hospital Bronx, New York  10467
Children's Hospital Medical Center Cincinnati, Ohio  45229
Liberty Hematology Oncology Center Columbia, South Carolina  29203
Palmetto Health Clinical Trials Columbia, South Carolina  29203
Texas Children's Hospital/Baylor College of Medicine Houston, Texas  77030
Scott and White Memorial Hospital & Clinics Temple, Texas  76508
Children's Hospital of the King's Daughter Norfolk, Virginia  23507
Hillman Cancer Center Pittsburg, Pennsylvania  15232
University of South Alabama Mobile, Alabama  36693
University of Alabama Pediatric Hematology/Oncology Birmingham, Alabama  35233
University of Alabama Medical center Birmingham, Alabama  35294
Center for Cancer and Blood Disorders Washington, District of Columbia  20010-2970
Tampa Children's Hospital at St. Joseph's Tampa, Florida  33607-6387
H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans Hospital Tampa, Florida  33612
Children's Healthcare of Atlanta at Scottish Rite Atlanta, Georgia  30342
Adult Sickle Cell Clinic Augusta, Georgia  30912-3128
Backus Children's Hospital Savannah, Georgia  31403
Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for Children Indianapolis, Indiana  46202
St. Jude Children's Hospital Affiliate Baton Rouge, Louisiana  70808
Children's Hospital New Orleans, Louisiana  70118
LSU Health Sciences Center/Carroll W. Feist Professor of Cancer Research Shreveport, Louisiana  71130
Children's Hospital Boston, Massachusetts  02115
Brigham and Woman's Hospital/Harvard Medical School Boston, Massachusetts  02115
Carolinas Medical Transplant Center Charlotte, North Carolina  28232
The University of Oklahoma Oklahoma City, Oklahoma  73104
Jefferson University Philadelphia, Pennsylvania  19107
Pennsylvania Oncology/Hematology Philadelphia, Pennsylvania  19106
St Jude's Children's Research Hospital Memphis, Tennessee  38105-2794
St. Jude's Children Research Hospital Memphis, Tennessee  38105
Cooks Children's Hospital Fort Worth, Texas  76104-2724