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

Primary Purpose

Beta-Thalassemia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Deferitrin (GT56-252)
desferoxamine (DFO)
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Beta-Thalassemia focused on measuring Beta Thalassemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • The New York Presbyterian Hospital-Weill Medical College of Cornell University

Outcomes

Primary Outcome Measures

safety and tolerability
iron excretion in urine and stool
pharmacokinetic measurements

Secondary Outcome Measures

Full Information

First Posted
October 2, 2003
Last Updated
March 4, 2015
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT00069862
Brief Title
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
Official Title
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
Study Type
Interventional

2. Study Status

Record Verification Date
March 2015
Overall Recruitment Status
Completed
Study Start Date
September 2003 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
November 2005 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-Thalassemia
Keywords
Beta Thalassemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
25 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Deferitrin (GT56-252)
Intervention Type
Drug
Intervention Name(s)
desferoxamine (DFO)
Primary Outcome Measure Information:
Title
safety and tolerability
Title
iron excretion in urine and stool
Title
pharmacokinetic measurements

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
The New York Presbyterian Hospital-Weill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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