Back to resultsEfficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
Primary Purpose
Beta-thalassemia, Serum Ferritin, Iron Chelation Therapy
Status
Unknown status
Phase
Not Applicable
Locations
Egypt
Study Type
Interventional
Intervention
desferal, ferriprox, blood transfusion
Sponsored by
About this trial
This is an interventional treatment trial for Beta-thalassemia focused on measuring B-thalassemia, ferritin
Eligibility Criteria
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.
Sites / Locations
- Pediatric Hematology clinic, Ain Shams University
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
iron chelation
blood transfusion only
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT01996683
First Posted
November 22, 2013
Last Updated
January 13, 2015
Sponsor
Ain Shams University
1. Study Identification
Unique Protocol Identification Number
NCT01996683
Brief Title
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
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 Type
Interventional
2. Study Status
Record Verification Date
January 2015
Overall Recruitment Status
Unknown status
Study Start Date
November 2014 (undefined)
Primary Completion Date
November 2015 (Anticipated)
Study Completion Date
November 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Ain Shams University
4. Oversight
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-thalassemia, Serum Ferritin, Iron Chelation Therapy
Keywords
B-thalassemia, ferritin
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
iron chelation
Arm Type
Active Comparator
Arm Description
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.
Arm Title
blood transfusion only
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
desferal, ferriprox, blood transfusion
Other Intervention Name(s)
deferiprone, deferoxamine
Intervention Description
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
Primary Outcome Measure Information:
Title
downward-trend of SF(serum ferritin) over previous 6 months after reduction of chelation dose with continuation of regular transfusion regimen.
Description
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.
Time Frame
6 month
Secondary Outcome Measure Information:
Title
safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy.
Description
determine the safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy.
Time Frame
12 month
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Amira A M Adly, Asst. prof
Phone
01005245837
Email
amiradiabetes@yahoo.com
First Name & Middle Initial & Last Name or Official Title & Degree
Yasmine I Elhenawy, lecturer
Phone
01004084038
Email
dr_yasmi@yahoo.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mohsen S. Elalfy, professour
Organizational Affiliation
Ain Shams University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Pediatric Hematology clinic, Ain Shams University
City
Cairo
Country
Egypt
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mohsen S Elalfy, professor
First Name & Middle Initial & Last Name & Degree
Amira A Adly, Assist. prof.
First Name & Middle Initial & Last Name & Degree
Yasmine I Elhenawy, lecturer
12. IPD Sharing Statement
Learn more about this trial
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
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