Beta-thalassemia, Iron Chelation Therapy, Serum Ferritin
Conditions
Keywords
B-thalassemia, ferritin
Brief summary
safety and efficacy of different iron chelation therapy in transfusion dependent beta-thalaasemia patients with low serum ferritin and continued regular transfusion regimen.
Detailed description
Treatment of transfusional iron overload in TM patients with low serum ferritin (continued decrease in serum ferritin ) even after reduction of chelation dose.
Interventions
DRUGdesferal, ferriprox, blood transfusion
Deferiprone (75-100 mg/kg/d) and deferoxamine (20-60 mg/kg/d) aimed at achieving normal body iron stores in poly-transfued arm1. Arm 2 will only recieve blood transfusion with no chelation
Sponsors
Ain Shams University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* • Subjects with low transfusional iron overload secondary to thalassemia major showing downward-trend in SF over last 6 months ( 2 readings are needed). * Thalassemia major patients with SF equal or less than 500 after reduction of chelation dose. Patients on different types of chelation monotherapy will be included * The subjects must also have a level of understanding and willingness to cooperate with the confinement and procedures described in the consent form and scheduled by the study site. In addition, he/she must be able to provide voluntary written informed assent or consent forms.
Exclusion criteria
* subjects with HIV positive or have active HCV. * A history of serious immunologic hypersensitivity to any medication such as anaphylaxis or angioedema. * Participation in a previous investigational drug study within the 30 days preceding screening. * A woman of childbearing potential must have a negative serum pregnancy test at screening. She must use a medically acceptable form of birth control during the study and for a month afterwards who are pregnant or breast-feeding. * An inability to adhere to the designated procedures and restrictions of this protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| downward-trend of SF(serum ferritin) over previous 6 months after reduction of chelation dose with continuation of regular transfusion regimen. | 6 month | non inferiority of discounting chelation compared with continuing chelation in thalassemia patients with low serum ferritin and/or showing a downward-trend of SF(serum ferritin) over previous 6 months after reduction of chelation dose with continuation of regular transfusion regimen. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy. | 12 month | determine the safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy. |
Countries
Egypt
Contacts
Primary ContactAmira A M Adly, Asst. prof
Backup ContactYasmine I Elhenawy, lecturer
Outcome results
None listed