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

NCT ID: NCT01624038

Last Updated: 2012-06-20

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-06-30
• Study Completion Date: 2012-12-31

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.

Conditions

Thalassemia Intermedia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Hydroxyurea,blood transfusion

Hydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week.

Group Type: ACTIVE_COMPARATOR

hydroxyurea, blood transfusion

Intervention Type: DRUG

* 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.

Hydroxyurea, Epiao

• Hydroxyurea (Myers-Squibb, USA) 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. White cell count and platelet count were determined on a monthly basis. Side effects such as nausea, vomiting, diarrhea, rashes, and malaise, experienced during the first 6 h after taking the HU will be considered as clinical toxicity.
• Erythropiotien therapy (rHuEPO - Epiao) from 250 to 500 IU/kg rHuEPO subcutaneously three times a week.

Group Type: EXPERIMENTAL

Hydroxyurea ,Epiao

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

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.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ain Shams University

OTHER

Sponsor Role: lead

Responsible Party

Mohsen Saleh Elalfy

prof. Mohsen el alfy

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Mohsen S Elalfy, professor

Role: PRINCIPAL_INVESTIGATOR

Ain Shams University

Locations

hematology clinic ,pediatrics hospital, Ain Shams University hospital

Cairo, , Egypt

Countries

Egypt

Central Contacts

Amira A M Adly, Asst. prof.

Role: CONTACT

amiradiabetes@yahoo.com

0105245837

References

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.

Reference Type: DERIVED

PMID: 23927461 (View on PubMed)

Other Identifiers

huoepio

Identifier Type: -

Identifier Source: org_study_id