Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis
1 other identifier
interventional
195
1 country
34
Brief Summary
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing 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
34 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, 2003
CompletedFirst Submitted
Initial submission to the registry
August 11, 2003
CompletedFirst Posted
Study publicly available on registry
August 13, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2007
CompletedAugust 22, 2017
August 1, 2017
4.2 years
August 11, 2003
August 18, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Evaluate the safety and tolerability of multiple doses of ICL670
1 year
Secondary Outcomes (4)
Estimate the absolute and relative change of liver iron content (LIC) and total body iron excretion (TBIE)
at baseline, after 24 weeks and at 1year (end of study)
Evaluate the pharmacokinetics
24 hours post-dose @ 4, 12, 24 and 52 weeks
Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variables
at 24 and 52 weks pre-dose
Evaluate the relationship between hepatic iron and potential surrogate markers
at screen, at washout, then every 2 weeks for the first 12 weeks followed by every 4 weeks
Study Arms (1)
ICL670 + deferoxamine
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- Age greater than or equal to 2 years
- Sickle cell disease patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- Serum ferritin greater than 1000 mg/ml
- Liver iron content greater than 2 mg iron/g dw assessed by means of superconducting quantum interference device (SQUID) for patients who receive simple transfusions and greater than 5 mg iron/ g dw for patients who receive exchange transfusions or who have a history of intermittent blood transfusion.
- Regular transfusion aimed at maintaining % Hb A above 50% or a previous history of simple transfusion being the recipient of at least 20 units of packed red blood cells.
You may not qualify if:
- Chronic anemias other than sickle cell disease
- Documented toxicity to deferoxamine
- Elevated liver enzymes in the year preceeding enrollment
- Active hepatitis B or hepatitis C
- HIV seropositivity
- Elevated serum creatinine or significant proteinuria
- History of nephrotic syndrome
- Uncontrolled systemic hypertension
- Fever and other signs/symptoms of infection within 10 days prior to the start of the study
- Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation
- Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval (other than beta-adrenergic receptor blocking agents).
- Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from undergoing any of the treatment options
- Psychiatric or addictive disorders that would prevent the patient from giving informed consent
- History of drug or alcohol abuse within the 12 months prior to the study
- Pregnant or breast feeding patients
- +6 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (34)
U. of S. Alabama Medical Center
Mobile, Alabama, 36604, United States
Loma Linda University Medical Center
Loma Linda, California, 92354, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital & Research Center
Oakland, California, 94609, United States
Colorado Sickle Cell Treatment and Research Center
Denver, Colorado, 80262, United States
Howard University Hospital
Washington D.C., District of Columbia, 20059, United States
Tampa Children's Hospital at St Joseph's
Tampa, Florida, 33607, United States
Georgia Comprehensive Sickle cell Center, Grady Hospital
Atlanta, Georgia, 30335, United States
Adult Sickle Cell Clinic, Medical College of Georgia
Augusta, Georgia, 30912, United States
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
Tulane University Sickle Cell Center
New Orleans, Louisiana, 70112, United States
Children's Hospital, Department of Hematology/Oncology
New Orleans, Louisiana, 70118, United States
Children's Hospital Boston, Division of Hematology/Oncology
Boston, Massachusetts, 02115, United States
Boston Medical Center
Boston, Massachusetts, 02118, United States
Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
NY Methodist Hospital
Brooklyn, New York, 11215, United States
Weill Medical College of Cornell University
New York, New York, 10021, United States
U. Of Rochester Medical Center
Rochester, New York, 14642, United States
Sickle Cell Center, Montefiore Hospital
The Bronx, New York, 10467, United States
Wake Forest University School of Medicine
Winston-Salem, North Carolina, 27106, United States
Barrett Center, University of Cincinnati
Cincinnati, Ohio, 45219, United States
Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
James Cancer Hospital
Columbus, Ohio, 43210, United States
Penn State Milton S Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213, 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
Baylor College of Medicine
Houston, Texas, 77030, United States
Texas Children's Hospital/Baylor College of Medicine
Houston, Texas, 77030, 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
Related Publications (1)
Vichinsky E, Onyekwere O, Porter J, Swerdlow P, Eckman J, Lane P, Files B, Hassell K, Kelly P, Wilson F, Bernaudin F, Forni GL, Okpala I, Ressayre-Djaffer C, Alberti D, Holland J, Marks P, Fung E, Fischer R, Mueller BU, Coates T; Deferasirox in Sickle Cell Investigators. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol. 2007 Feb;136(3):501-8. doi: 10.1111/j.1365-2141.2006.06455.x.
PMID: 17233848RESULT
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
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
August 11, 2003
First Posted
August 13, 2003
Study Start
May 1, 2003
Primary Completion
July 1, 2007
Last Updated
August 22, 2017
Record last verified: 2017-08