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

Not Applicable

• Conditions: Iron Chelation Therapy; Beta-thalassemia; Serum Ferritin
• Interventions: Drug: desferal, ferriprox, blood transfusion

• Registration Number: NCT01996683
• Lead Sponsor: Ain Shams University

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.

Study Timeline

• Study First Submitted: 2013-11-22
• Study First Submitted QC: 2013-11-22
• Study First Posted: 2013-11-27
• Study Start: 2014-11
• Status Verified: 2015-01
• Last Update Submitted: 2015-01-13
• Last Update Posted: 2015-01-14
• Primary Completion: 2015-11
• Study Completion: 2015-11

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 50

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
iron chelation	desferal, ferriprox, blood transfusion	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.
blood transfusion only	desferal, ferriprox, blood transfusion	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.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.

Trial Locations

Locations (1)

Pediatric Hematology clinic, Ain Shams University; 🇪🇬 Cairo, Egypt