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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2012 by Ain Shams University.
Recruitment status was  Not yet recruiting
Sponsor:
Information provided by (Responsible Party):
Mohsen Saleh Elalfy, Ain Shams University
ClinicalTrials.gov Identifier:
NCT01624038
First received: April 13, 2012
Last updated: June 18, 2012
Last verified: June 2012
  Purpose

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.


Condition Intervention Phase
Thalassemia Intermedia
Drug: Hydroxyurea ,Epiao
Drug: hydroxyurea, blood transfusion
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.

Resource links provided by NLM:


Further study details as provided by Ain Shams University:

Primary Outcome Measures:
  • Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin [ Time Frame: baseline and 6 month hemoglobin level and transfusion frequency ] [ Designated as safety issue: Yes ]
    Decrease in baseline transfusion frequency with increase of pre-transfusion hemoglobin by calculation of transfusion index and mean hemoglobin level


Secondary Outcome Measures:
  • Change in baseline quality of life assessment. [ Time Frame: baseline and 6 month QOL questionaire ] [ Designated as safety issue: Yes ]
    Quality of life assessment using (QOL questionaire) at the begining and at 6 month


Estimated Enrollment: 40
Study Start Date: June 2012
Estimated Study Completion Date: December 2012
Estimated Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 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.
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.
Experimental: 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.
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.


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

Ages Eligible for Study:   3 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01624038

Contacts
Contact: Amira A M Adly, Asst. prof. 0105245837 amiradiabetes@yahoo.com

Locations
Egypt
hematology clinic ,pediatrics hospital, Ain Shams University hospital Not yet recruiting
Cairo, Egypt
Principal Investigator: Mohsen Saleh El-Alfy, professor of pediatrics         
Sponsors and Collaborators
Ain Shams University
Investigators
Principal Investigator: Mohsen S Elalfy, professor Ain Shams University
  More Information

No publications provided

Responsible Party: Mohsen Saleh Elalfy, prof. Mohsen el alfy, Ain Shams University
ClinicalTrials.gov Identifier: NCT01624038     History of Changes
Other Study ID Numbers: huoepio
Study First Received: April 13, 2012
Last Updated: June 18, 2012
Health Authority: Egypt: Institutional Review Board

Keywords provided by Ain Shams University:
Hydroxyurea
Erythropoitin therapy
Thalassemia intermedia

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Epoetin alfa
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Hematinics
Hematologic Agents
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014