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

August 17, 2017 updated by: Novartis Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

175

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Oakland, California, United States, 94609
        • Children's Hospital Oakland
      • Stanford, California, United States, 94305-5208
        • Stanford hospital
    • Illinois
      • Arlington Heights, Illinois, United States, 60004
        • Northwest Medical Specialists
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Children's Hospital Boston
    • New York
      • New York, New York, United States, 10021
        • Weill Medical College of Cornell University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-4318
        • Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
To evaluate the effects of treatment on the liver iron content(LIC)

Secondary Outcome Measures

Outcome Measure
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2003

Primary Completion (Actual)

November 1, 2004

Study Registration Dates

First Submitted

June 3, 2003

First Submitted That Met QC Criteria

June 3, 2003

First Posted (Estimate)

June 4, 2003

Study Record Updates

Last Update Posted (Actual)

August 22, 2017

Last Update Submitted That Met QC Criteria

August 17, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670A0108

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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