Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients

NCT ID: NCT00110617

Last Updated: 2011-05-26

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

212 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-05-31

Study Completion Date

2008-04-30

Brief Summary

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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.

Detailed Description

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Conditions

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Sickle Cell Disease Iron Overload Hemolytic Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Deferasirox (ICL670)

Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.

Group Type EXPERIMENTAL

Deferasirox (ICL670)

Intervention Type DRUG

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 (DFO) then ICL670

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.

Group Type EXPERIMENTAL

Deferasirox (ICL670)

Intervention Type DRUG

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 (DFO)

Intervention Type DRUG

Deferoxamine was supplied in vials of 500 mg and 2000 mg administered subcutaneously for a weekly dose of 175 mg/kg.

Interventions

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Deferasirox (ICL670)

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.

Intervention Type DRUG

Deferoxamine (DFO)

Deferoxamine was supplied in vials of 500 mg and 2000 mg administered subcutaneously for a weekly dose of 175 mg/kg.

Intervention Type DRUG

Eligibility Criteria

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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 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
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Novartis Pharmaceuticals

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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University of Alabama Pediatric Hematology/Oncology

Birmingham, Alabama, United States

Site Status

University of Alabama Medical center

Birmingham, Alabama, United States

Site Status

University of South Alabama Medical Center

Mobile, Alabama, United States

Site Status

University of South Alabama

Mobile, Alabama, United States

Site Status

Loma Linda University Medical Center

Loma Linda, California, United States

Site Status

Children's Hospital Oakland

Oakland, California, United States

Site Status

Center for Cancer and Blood Disorders

Washington D.C., District of Columbia, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Howard University Hospital

Washington D.C., District of Columbia, United States

Site Status

Miami Children's Hospital

Miami, Florida, United States

Site Status

Tampa Children's Hospital at St Joseph's

Tampa, Florida, United States

Site Status

Tampa Children's Hospital at St. Joseph's

Tampa, Florida, United States

Site Status

H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans Hospital

Tampa, Florida, United States

Site Status

Emory University School of Medicine

Atlanta, Georgia, United States

Site Status

Children's Healthcare of Atlanta at Scottish Rite

Atlanta, Georgia, United States

Site Status

Adult Sickle Cell Clinic

Augusta, Georgia, United States

Site Status

Backus Children's Hospital

Savannah, Georgia, United States

Site Status

University of Illinois at Chicago

Chicago, Illinois, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

St. Jude Children's Hospital Affiliate

Baton Rouge, Louisiana, United States

Site Status

Tulane University Sickle Cell Center

New Orleans, Louisiana, United States

Site Status

Children's Hospital

New Orleans, Louisiana, United States

Site Status

LSU Health Sciences Center/Carroll W. Feist Professor of Cancer Research

Shreveport, Louisiana, United States

Site Status

Brigham and Woman's Hospital/Harvard Medical School

Boston, Massachusetts, United States

Site Status

Children's Hospital Boston

Boston, Massachusetts, United States

Site Status

Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

SUNY Downstate Medical Center

Brooklyn, New York, United States

Site Status

New York Methodist Hospital

Brooklyn, New York, United States

Site Status

Weill Medical College of Cornell University

New York, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

Sickle Cell Center, Montefiore Hospital

The Bronx, New York, United States

Site Status

Carolinas Medical Transplant Center

Charlotte, North Carolina, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

The University of Oklahoma

Oklahoma City, Oklahoma, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Pennsylvania Oncology/Hematology

Philadelphia, Pennsylvania, United States

Site Status

Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Drexel University College of Medicine

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Hillman Cancer Center

Pittsburgh, Pennsylvania, United States

Site Status

Liberty Hematology Oncology Center

Columbia, South Carolina, United States

Site Status

Palmetto Health Clinical Trials

Columbia, South Carolina, United States

Site Status

Santee Hematology/Oncology

Sumter, South Carolina, United States

Site Status

St Jude's Children's Research Hospital

Memphis, Tennessee, United States

Site Status

St. Jude's Children Research Hospital

Memphis, Tennessee, United States

Site Status

Cooks Children's Hospital

Fort Worth, Texas, United States

Site Status

Texas Children's Hospital/Baylor College of Medicine

Houston, Texas, United States

Site Status

Scott and White Memorial Hospital & Clinics

Temple, Texas, United States

Site Status

Children's Hospital of the King's Daughter

Norfolk, Virginia, United States

Site Status

Medical College of Virginia

Richmond, Virginia, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

University of Alberta

Edmonton, Alberta, Canada

Site Status

The Ottawa Hospital

Ottawa, Ontario, Canada

Site Status

Hopital Ste-Justine

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

Related Links

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http://www.novartisclinicaltrials.com

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Other Identifiers

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CICL670A2201

Identifier Type: -

Identifier Source: org_study_id

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