Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.

Thalassemia Intermedia Clinical Trial

Official title:

Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT01624038
• Other study ID #: huoepio
• Status: Not yet recruiting
• Phase: Phase 2/Phase 3
• First received: April 13, 2012
• Last updated: June 18, 2012
• Start date: June 2012
• Est. completion date: December 2012

Study information

• Verified date: June 2012
• Source: Ain Shams University
• Contact: Amira A M Adly, Asst. prof.
• Phone: 0105245837
• Email: amiradiabetes[@]yahoo.com
• Is FDA regulated: No
• Health authority: Egypt: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• 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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta-Thalassemia
• Thalassemia
• Thalassemia Intermedia

Intervention

Drug:
• Hydroxyurea ,Epiao
Hydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week. Erythropiotien therapy (rHuEPO - Epiao) from 250 to 500 IU/kg rHuEPO subcutaneously three times a week.
• hydroxyurea, blood transfusion
Hydroxyurea was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week. Hydroxyurea toxicity was defined as a white cell count of less than 2500/µL or a platelet count of less than 100,000/µL, in which case the drug was discontinued.

Locations

Country	Name	City	State
Egypt	hematology clinic ,pediatrics hospital, Ain Shams University hospital	Cairo

Sponsors (1)

Lead Sponsor	Collaborator
Ain Shams University

Country where clinical trial is conducted

Egypt, 

Outcome

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