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

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)

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

Study Overview

• Status: Unknown
• Conditions: Beta-thalassemia; Serum Ferritin; Iron Chelation Therapy
• Intervention / Treatment: Drug: desferal, ferriprox, blood transfusion

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 Type: Interventional
• Enrollment (Anticipated): 50
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Egypt
  • Cairo, Egypt
    • Pediatric Hematology clinic, Ain Shams University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / 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: deferoxamine; deferiprone
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: deferoxamine; deferiprone

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Ain Shams University

Study record dates

Study Major Dates

• Study Start: November 1, 2014
• Primary Completion (ANTICIPATED): November 1, 2015
• Study Completion (ANTICIPATED): November 1, 2015

Study Registration Dates

• First Submitted: November 22, 2013
• First Submitted That Met QC Criteria: November 22, 2013
• First Posted (ESTIMATE): November 27, 2013

Study Record Updates

• Last Update Posted (ESTIMATE): January 14, 2015
• Last Update Submitted That Met QC Criteria: January 13, 2015
• Last Verified: January 1, 2015

More Information

Terms related to this study

• Keywords: ferritin; B-thalassemia
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Molecular Mechanisms of Pharmacological Action; Chelating Agents; Sequestering Agents; Iron Chelating Agents; Siderophores; Deferoxamine; Deferiprone
• Other Study ID Numbers: iron chelation in thalassemia