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

Beta-thalassemia Clinical Trial

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)

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT01996683
• Other study ID #: iron chelation in thalassemia
• Status: Not yet recruiting
• Phase: N/A
• First received: November 22, 2013
• Last updated: January 13, 2015
• Start date: November 2014
• Est. completion date: November 2015

Study information

• Verified date: January 2015
• Source: Ain Shams University
• Contact: Amira A M Adly, Asst. prof
• Phone: 01005245837
• Email: amiradiabetes[@]yahoo.com
• Is FDA regulated: No
• Health authority: Egypt: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

safety and efficacy of different iron chelation therapy in transfusion dependent beta-thalaasemia patients with low serum ferritin and continued regular transfusion regimen.

Description:

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 50
• Est. completion date: November 2015
• Est. primary completion date: November 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 18 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta-thalassemia
• Iron Chelation Therapy
• Serum Ferritin
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
Egypt	Pediatric Hematology clinic, Ain Shams University	Cairo

Sponsors (1)

Lead Sponsor	Collaborator
Ain Shams University

Country where clinical trial is conducted

Egypt, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	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	Yes