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

This study has been completed.
Sponsor:
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00069862
First received: October 2, 2003
Last updated: June 19, 2008
Last verified: May 2007
  Purpose

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
Beta-Thalassemia
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
  Eligibility

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

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
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: NCT00069862

Locations
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

ClinicalTrials.gov 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:
Beta-Thalassemia
Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on July 31, 2014