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

Ain Shams University

Status: Unknown

Conditions: Iron Chelation Therapy; Serum Ferritin; Beta-thalassemia

Treatments: Drug: desferal, ferriprox, blood transfusion

Study type: Interventional

Funder types: Other

Identifiers

NCT01996683

iron chelation in thalassemia

Details and patient eligibility

About

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

Full description

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

Enrollment: 50 estimated patients

Sex: All

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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50 participants in 2 patient groups, including a placebo group

iron chelation

Active Comparator group

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.

Treatment:

Drug: desferal, ferriprox, blood transfusion

blood transfusion only

Placebo Comparator group

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.

Treatment:

Drug: desferal, ferriprox, blood transfusion