Thalassemia Intermedia Clinical Trial
Official title:
Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
The study hypothesis that treatment with Erythropoietin (EPO) combined with Human
Erythropoietin (HUO) therapy will result in hematologic improvement in thalassemia
intermedia patients.
Second is to determine whether any of the following correlate with improved hematologic
response:
A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline
levels,baseline Erythropoietin levels,baseline hemoglobin levels and baseline reticulocyte
counts (or % circulating nucleated erythroblasts/100 WBCs).
Goal:
The aim is to assess the possibility of steady increase of hemoglobin levels in thalassemia
intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea
and Erythropoietin, growth evaluation,quality of life (QoL) and decline transfusion
requirements during study period. Also to report and compare adverse events with other
published data regarding.
Status | Not yet recruiting |
Enrollment | 40 |
Est. completion date | December 2012 |
Est. primary completion date | December 2012 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 3 Years to 18 Years |
Eligibility |
Inclusion Criteria: - Patients with thalassemia intermedia. Diagnosis based on genetic mutations, hemoglobin electrophoresis and characteristic clinical data at presentation. - Require different transfusion requirements and not transfusion dependent. - Have a baseline hemoglobin of less than or equal to 6-8g/dl. - Patients with normal renal and liver function. Exclusion Criteria: - Evidence of active hepatitis (ALT > 5 times above ULN). - Evidence of renal impairment (serum creatinine > ULN). - Patients who are dependent on red blood cell transfusions. |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Egypt | hematology clinic ,pediatrics hospital, Ain Shams University hospital | Cairo |
Lead Sponsor | Collaborator |
---|---|
Ain Shams University |
Egypt,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin | Decrease in baseline transfusion frequency with increase of pre-transfusion hemoglobin by calculation of transfusion index and mean hemoglobin level | baseline and 6 month hemoglobin level and transfusion frequency | Yes |
Secondary | Change in baseline quality of life assessment. | Quality of life assessment using (QOL questionaire) at the begining and at 6 month | baseline and 6 month QOL questionaire | Yes |
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