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

Beta-Thalassemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00069862
• Other study ID #: GTC-134-102
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: October 2, 2003
• Last updated: March 4, 2015
• Start date: September 2003
• Est. completion date: November 2005

Study information

• Verified date: March 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: November 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta-Thalassemia
• Iron Overload
• Thalassemia

Intervention

Drug:
• Deferitrin (GT56-252)

• desferoxamine (DFO)

Locations

Country	Name	City	State
United States	The New York Presbyterian Hospital-Weill Medical College of Cornell University	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	safety and tolerability
Primary	iron excretion in urine and stool
Primary	pharmacokinetic measurements