Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
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
1 other identifier
interventional
212
2 countries
59
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started May 2005
Typical duration for phase_2
59 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2005
CompletedFirst Submitted
Initial submission to the registry
May 10, 2005
CompletedFirst Posted
Study publicly available on registry
May 11, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2008
CompletedResults Posted
Study results publicly available
May 2, 2011
CompletedMay 26, 2011
May 1, 2011
2.9 years
May 10, 2005
January 11, 2011
May 23, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
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.
24 Weeks
Secondary Outcomes (3)
Absolute Change in Serum Ferritin From Baseline to Week 24
Baseline, 24 Weeks
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52
Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104
Start of Deferasirox (ICL670) treatment, 104 Weeks
Study Arms (2)
Deferasirox (ICL670)
EXPERIMENTALDeferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
Deferoxamine (DFO) then ICL670
EXPERIMENTALDeferoxamine (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.
Interventions
Deferasirox was provided in 125 mg, 250 mg, and 500 mg dispersible tablets and was administered orally at an initial dose of 20 mg/kg/day.
Deferoxamine was supplied in vials of 500 mg and 2000 mg administered subcutaneously for a weekly dose of 175 mg/kg.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (59)
University of Alabama Pediatric Hematology/Oncology
Birmingham, Alabama, 35233, United States
University of Alabama Medical center
Birmingham, Alabama, 35294, 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
Center for Cancer and Blood Disorders
Washington D.C., District of Columbia, 20010-2970, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Howard University Hospital
Washington D.C., District of Columbia, 20059, United States
Miami Children's Hospital
Miami, Florida, 33155, United States
Tampa Children's Hospital at St Joseph's
Tampa, Florida, 33607-6387, United States
Tampa Children's Hospital at St. Joseph's
Tampa, Florida, 33607-6387, United States
H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans Hospital
Tampa, Florida, 33612, United States
Emory University School of Medicine
Atlanta, Georgia, 30303, United States
Children's Healthcare of Atlanta at Scottish Rite
Atlanta, Georgia, 30342, United States
Adult Sickle Cell Clinic
Augusta, Georgia, 30912-3128, United States
Backus Children's Hospital
Savannah, Georgia, 31403, United States
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
Children's Memorial Hospital
Chicago, Illinois, 60614-3394, United States
Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
St. Jude Children's Hospital Affiliate
Baton Rouge, Louisiana, 70808, United States
Tulane University Sickle Cell Center
New Orleans, Louisiana, 70112, United States
Children's Hospital
New Orleans, Louisiana, 70118, United States
LSU Health Sciences Center/Carroll W. Feist Professor of Cancer Research
Shreveport, Louisiana, 71130, United States
Brigham and Woman's Hospital/Harvard Medical School
Boston, Massachusetts, 02115, United States
Children's Hospital Boston
Boston, Massachusetts, 02115, United States
Children's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109-0238, United States
Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
SUNY Downstate Medical Center
Brooklyn, New York, 11203, United States
New York Methodist Hospital
Brooklyn, New York, 11215, United States
Weill Medical College of Cornell University
New York, New York, 10021, United States
Columbia University
New York, New York, 10032, United States
Sickle Cell Center, Montefiore Hospital
The Bronx, New York, 10467-2490, United States
Carolinas Medical Transplant 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 Philadelphia
Philadelphia, Pennsylvania, 19104-4399, United States
Pennsylvania Oncology/Hematology
Philadelphia, Pennsylvania, 19106, United States
Jefferson University
Philadelphia, Pennsylvania, 19107, United States
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19134-1095, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232, 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
St. Jude's Children Research Hospital
Memphis, Tennessee, 38105, United States
Cooks Children's Hospital
Fort Worth, Texas, 76104-2724, United States
Texas Children's Hospital/Baylor College of Medicine
Houston, Texas, 77030-2399, United States
Scott and White Memorial Hospital & Clinics
Temple, Texas, 76508, 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
Virginia Commonwealth University
Richmond, Virginia, 23298-0646, United States
University of Alberta
Edmonton, Alberta, T6G 2H7, Canada
The Ottawa Hospital
Ottawa, Ontario, K1H 8L6, Canada
Hopital Ste-Justine
Montreal, Quebec, H3T 1C5, Canada
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Due to severe good clinical practice violations, data from 9 participants recruited for Center 512 was excluded from the main analysis of the study.
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
May 10, 2005
First Posted
May 11, 2005
Study Start
May 1, 2005
Primary Completion
April 1, 2008
Study Completion
April 1, 2008
Last Updated
May 26, 2011
Results First Posted
May 2, 2011
Record last verified: 2011-05