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

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ClinicalTrials.gov Identifier: NCT01624038
Recruitment Status : Unknown
Verified June 2012 by Mohsen Saleh Elalfy, Ain Shams University.
Recruitment status was:  Not yet recruiting
First Posted : June 20, 2012
Last Update Posted : June 20, 2012
Sponsor:
Information provided by (Responsible Party):
Mohsen Saleh Elalfy, Ain Shams University

Tracking Information
First Submitted Date  ICMJE April 13, 2012
First Posted Date  ICMJE June 20, 2012
Last Update Posted Date June 20, 2012
Study Start Date  ICMJE June 2012
Estimated Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 18, 2012)
Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin [ Time Frame: baseline and 6 month hemoglobin level and transfusion frequency ]
Decrease in baseline transfusion frequency with increase of pre-transfusion hemoglobin by calculation of transfusion index and mean hemoglobin level
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: June 18, 2012)
Change in baseline quality of life assessment. [ Time Frame: baseline and 6 month QOL questionaire ]
Quality of life assessment using (QOL questionaire) at the begining and at 6 month
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Official Title  ICMJE Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Thalassemia Intermedia
Intervention  ICMJE
  • 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.

  • 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.
Study Arms  ICMJE
  • 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.
    Intervention: Drug: hydroxyurea, blood transfusion
  • 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.
    Intervention: Drug: Hydroxyurea ,Epiao
Publications * 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 β-thalassemia intermedia patients. Eur J Haematol. 2013 Dec;91(6):522-33. doi: 10.1111/ejh.12182. Epub 2013 Oct 5.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: June 18, 2012)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2012
Estimated Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Egypt
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01624038
Other Study ID Numbers  ICMJE huoepio
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Mohsen Saleh Elalfy, Ain Shams University
Study Sponsor  ICMJE Ain Shams University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Mohsen S Elalfy, professor Ain Shams University
PRS Account Ain Shams University
Verification Date June 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP