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Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00061763
Recruitment Status : Completed
First Posted : June 4, 2003
Last Update Posted : August 22, 2017
Information provided by:

Brief Summary:
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.

Condition or disease Intervention/treatment Phase
Beta-thalassemia Myelodysplastic Syndromes Fanconi Syndrome Anemia, Diamond-Blackfan Anemia, Aplastic Drug: Deferasirox Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 175 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine
Study Start Date : May 2003
Actual Primary Completion Date : November 2004

Primary Outcome Measures :
  1. To evaluate the effects of treatment on the liver iron content(LIC)

Secondary Outcome Measures :
  1. Evaluate tolerability profile
  2. Estimate the absolute and relative change of LIC and total body iron excretion (TBIE) rate
  3. Evaluate the relationship between LIC and potential surrogate markers
  4. Evaluate the relationship between PD and safety variables

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Beta-thalassemia patients with documented non-compliance to deferoxamine, defined as taking less than 50% of prescribed doses in year prior to study, and having a liver iron content at least 14 mg iron/gm dry weight liver tissue
  • Beta-thalassemia patients unable to take deferoxamine because of documented side effects or contra-indication, or documented poor response despite proper compliance, with liver iron content at least 2 mg iron/gm dry weight liver tissue
  • Patients with chronic anemias with a liver iron content at least 2 mg/gm dry weight liver tissue.
  • Beta-thalassemia or other chronic anemia patients having previously taken deferiprone, provided that they stop the deferiprone at least 28 days before the study and have a liver iron content at least 2 mg/gm dry weight liver tissue.
  • All patients: Regular transfusions indicated by a requirement of at least 8 blood transfusions per year.
  • Life expectancy of at least one year.

Exclusion Criteria:

  • Beta-thalassemia able to be treated with deferoxamine, Sickle Cell Disease or non-transfusional iron overload
  • Elevated liver enzymes in the year preceding 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 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.
  • Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from undergoing any of the treatment options.
  • Psychiatric or additive 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.
  • Patients treated with systemic investigational drugs within 4 weeks or topical investigational drugs within 7 days before the start of teh study.
  • Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as gastrointestinal disease or major surgery, renal disease, difficulty voiding or urinary obstruction, or impaired pancreatic function.
  • Non-compliant or unreliable patients
  • Patients unable to undergo any study procedures such as the hearing or eye tests, or the liver echocardiography.
  • Patients that would need a dose of Deferasirox less than 125 mg per day.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00061763

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United States, California
Children's Hospital Oakland
Oakland, California, United States, 94609
Stanford Hospital
Stanford, California, United States, 94305-5208
United States, Illinois
Northwest Medical Specialists
Arlington Heights, Illinois, United States, 60004
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, New York
Weill Medical College of Cornell University
New York, New York, United States, 10021
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104-4318
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00061763    
Other Study ID Numbers: CICL670A0108
First Posted: June 4, 2003    Key Record Dates
Last Update Posted: August 22, 2017
Last Verified: August 2017
Keywords provided by Novartis:
iron overload
Myelodysplastic Syndromes
Fanconi Syndrome
Anemia, Diamond-Blackfan
Anemia, Aplastic
Additional relevant MeSH terms:
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Fanconi Syndrome
Myelodysplastic Syndromes
Anemia, Aplastic
Fanconi Anemia
Anemia, Diamond-Blackfan
Iron Overload
Pathologic Processes
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Bone Marrow Failure Disorders
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases