Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)

Study Overview
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Brief Summary
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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
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Treatment of transfusional iron overload in TM patients with low serum ferritin (continued decrease in serum ferritin ) even after reduction of chelation dose.

Official Title
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A Randomized Prospective Comparative Study of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)

Conditions
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Beta-thalassemia, Serum Ferritin, Iron Chelation Therapy

Intervention / Treatment
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* Drug: desferal, ferriprox, blood transfusion


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: iron chelation<br>Included 25 thalassemia patients with low serum ferritin (< 500) or downward ferritin trend inspite of reduction of chelation dose over the last 6 months. They will continue their chelation therapy. | Drug: desferal, ferriprox, blood transfusion<br>* 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<br>* Other names: deferoxamine;|
| Placebo Comparator: blood transfusion only<br>Included 25 thalassemia patients with low serum ferritin (< 500) or downward ferritin trend inspite of reduction of chelation dose over the last 6 months. They will be subjected to discontinuation of their chelation therapy. | Drug: desferal, ferriprox, blood transfusion<br>* 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<br>* Other names: deferoxamine;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| downward-trend of SF(serum ferritin) over previous 6 months after reduction of chelation dose with continuation of regular transfusion regimen. | 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. | 6 month | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy. | determine the safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy. | 12 month | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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B-thalassemia, ferritin