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

Sponsor

Ain Shams University (Other)

Overall Status

Unknown status

CT.gov ID

NCT01996683

Collaborator

(none)

Enrollment

Location

Arms

Duration (Months)

4.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Beta-thalassemia Serum Ferritin Iron Chelation Therapy	Drug: desferal, ferriprox, blood transfusion	N/A

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

50 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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)

Study Start Date :

Nov 1, 2014

Anticipated Primary Completion Date :

Nov 1, 2015

Anticipated Study Completion Date :

Nov 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: iron chelation 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 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 Other Names: deferiprone deferoxamine
Placebo Comparator: blood transfusion only 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 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 Other Names: deferiprone deferoxamine

Arm

Intervention/Treatment

Active Comparator: iron chelation

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

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

Other Names:

deferiprone

deferoxamine

Placebo Comparator: blood transfusion only

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

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

Other Names:

deferiprone

deferoxamine

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.