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)
Trial Snapshot
- Phase
- Not Applicable
- Sponsor
- Ain Shams University
- Enrollment
- 50
- Locations
- 1
- Primary Endpoint
- downward-trend of SF(serum ferritin) over previous 6 months after reduction of chelation dose with continuation of regular transfusion regimen.
Study Overview
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 6 Years to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
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.
Intervention: desferal, ferriprox, blood transfusion (Drug)
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.
Intervention: desferal, ferriprox, blood transfusion (Drug)
Outcomes
Primary Outcomes
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
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 Outcomes
- safety and occurrence of AEs in both studied groups (with and without chelation) in relation to different chelation therapy.(12 month)
Investigators
Mohsen Saleh Elalfy
professor of pediatrics
Ain Shams University