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

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00061763
First received: June 3, 2003
Last updated: March 2, 2011
Last verified: March 2011
  Purpose

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 Intervention Phase
Beta-thalassemia
Myelodysplastic Syndromes
Fanconi Syndrome
Anemia, Diamond-Blackfan
Anemia, Aplastic
Drug: Deferasirox
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

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

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

Enrollment: 175
Study Start Date: May 2003
Primary Completion Date: November 2004 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00061763

Locations
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
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00061763     History of Changes
Other Study ID Numbers: CICL670A0108
Study First Received: June 3, 2003
Last Updated: March 2, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
beta-thalassemia
iron overload
deferoxamine
Myelodysplastic Syndromes
Fanconi Syndrome
Anemia, Diamond-Blackfan
Anemia, Aplastic

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Anemia, Diamond-Blackfan
Beta-Thalassemia
Fanconi Anemia
Fanconi Syndrome
Myelodysplastic Syndromes
Preleukemia
Syndrome
Thalassemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Anemia, Hypoplastic, Congenital
Bone Marrow Diseases
Disease
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Kidney Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Neoplasms
Pathologic Processes
Precancerous Conditions
Red-Cell Aplasia, Pure
Renal Tubular Transport, Inborn Errors
Urologic Diseases
Deferasirox
Deferoxamine

ClinicalTrials.gov processed this record on October 29, 2014