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

NCT01996683 | Unknown DMC

• 1 other identifier: iron chelation in thalassemia
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Beta-thalassemia; Serum Ferritin; Iron Chelation Therapy

Keywords

B-thalassemia; ferritin

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy.

  12 month

Study Arms (2)

iron chelation

ACTIVE COMPARATOR

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

blood transfusion only

PLACEBO COMPARATOR

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

Interventions

desferal, ferriprox, blood transfusion DRUG

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

Also known as: deferiprone, deferoxamine

blood transfusion only; iron chelation

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ain Shams University: lead

Study Sites (1)

Pediatric Hematology clinic, Ain Shams University

Cairo, Egypt

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Deferoxamine; Deferiprone; Blood Transfusion

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Hydroxamic Acids; Hydroxylamines; Amines; Organic Chemicals; Hydroxy Acids; Carboxylic Acids; Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Biological Therapy; Therapeutics

Study Officials

• Mohsen S. Elalfy, professour

  Ain Shams University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Amira A M Adly, Asst. prof

CONTACT

01005245837; amiradiabetes@yahoo.com

Yasmine I Elhenawy, lecturer

CONTACT

01004084038; dr_yasmi@yahoo.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: professor of pediatrics

Study Record Dates

• First Submitted: November 22, 2013
• First Posted: November 27, 2013
• Study Start: November 1, 2014
• Primary Completion: November 1, 2015
• Study Completion: November 1, 2015
• Last Updated: January 14, 2015

Record last verified: 2015-01

Locations

EG (1)