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

Phase 1

Completed

• Conditions: Beta-Thalassemia

• Registration Number: NCT00069862
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2003-09
• Study First Submitted: 2003-10-02
• Study First Submitted QC: 2003-10-02
• Study First Posted: 2003-10-03
• Study Completion: 2005-11
• Status Verified: 2015-03
• Last Update Submitted: 2015-03-04
• Last Update Posted: 2015-03-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Primary Outcome Measures

Name	Time	Method
safety and tolerability
iron excretion in urine and stool
pharmacokinetic measurements

Trial Locations

Locations (1)

The New York Presbyterian Hospital-Weill Medical College of Cornell University; 🇺🇸 New York, New York, United States