Clinical trial NCT01624038: Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Official title: Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Thalassemia Intermedia
Keywords: Hydroxyurea
Erythropoitin therapy
Thalassemia intermedia
Lead sponsor: Ain Shams University
Sponsor class: OTHER
First posted: 2012-06-20
Enrollment: 40
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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 level and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs).

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.

THE following criteria are used when including the patient in the study:

Patients with thalassemia intermedia.Diagnosis based on genetic mutations, hemoglobin electrophoresis and characteristic clinical data at presentation. Patients requiring different transfusion requirements and not transfusion dependent.Patients having a baseline hemoglobin of less than or equal to 6-8g/dl.Patients with normal renal and liver function.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin
timeFrame: baseline and 6 month hemoglobin level and transfusion frequency
Secondary outcomes:
measure: Change in baseline quality of life assessment.
timeFrame: baseline and 6 month QOL questionaire
Other outcomes:
Locations:
city: Cairo
country: Egypt
facility: hematology clinic ,pediatrics hospital, Ain Shams University hospital
Study officials:
affiliation: Ain Shams University
name: Mohsen S Elalfy, professor
role: PRINCIPAL_INVESTIGATOR
References:
citation: Elalfy MS, Adly AA, Ismail EA, Elhenawy YI, Elghamry IR. Therapeutic superiority and safety of combined hydroxyurea with recombinant human erythropoietin over hydroxyurea in young beta-thalassemia intermedia patients. Eur J Haematol. 2013 Dec;91(6):522-33. doi: 10.1111/ejh.12182. Epub 2013 Oct 5.