Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

This study has been completed.
Information provided by:
Genzyme, a Sanofi Company Identifier:
First received: October 2, 2003
Last updated: June 19, 2008
Last verified: May 2007

A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.

Condition Intervention Phase
Drug: Deferitrin (GT56-252)
Drug: desferoxamine (DFO)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

Resource links provided by NLM:

Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • safety and tolerability
  • iron excretion in urine and stool
  • pharmacokinetic measurements

Estimated Enrollment: 25
Study Start Date: September 2003

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Beta-thalassemia patients, 18 years of age or older, currently undergoing chronic blood transfusion therapy and iron chelation therapy who weigh more than 40 kg.
  • No clinically significant findings on physical exam, medical history, or screening laboratories.
  • Serum ferritin greater than 500 ng/mL, serum creatine creatinine within the normal range and platelet count greater than 100,000/mm3.
  • Willing and able to discontinue DFO or L1 for the period of study.
  • Woman of child-bearing potential must have a negative serum pregnancy test at screening and use a medically acceptable form of birth control during the study and for 1 month afterward. Male patients must also use barrier contraceptives during the study and for 1 month afterward.
  • Have a level of understanding and willingness to cooperate with the confinement and all procedures. Able to provide voluntary signed/dated written informed consent.

Exclusion Criteria:

  • Serious medical condition unrelated to Beta-Thalassemia.
  • Participation in a previous investigational drug study within 30 days preceding screening.
  • Patients with a known allergy to DFO that prevents chronic administration.
  Contacts and Locations
Please refer to this study by its identifier: NCT00069862

United States, New York
The New York Presbyterian Hospital-Weill Medical College of Cornell University
New York, New York, United States, 10021
Sponsors and Collaborators
Genzyme, a Sanofi Company
  More Information

No publications provided Identifier: NCT00069862     History of Changes
Other Study ID Numbers: GTC-134-102
Study First Received: October 2, 2003
Last Updated: June 19, 2008
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme, a Sanofi Company:
Beta Thalassemia

Additional relevant MeSH terms:
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases processed this record on April 16, 2014