Beta-Thalassemia
Conditions
Keywords
Beta Thalassemia
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.
Interventions
DRUGDeferitrin (GT56-252)
DRUGdesferoxamine (DFO)
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| safety and tolerability | — |
| iron excretion in urine and stool | — |
| pharmacokinetic measurements | — |
Countries
United States
Outcome results
None listed