Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
1 other identifier
interventional
40
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2012
Shorter than P25 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 13, 2012
CompletedStudy Start
First participant enrolled
June 1, 2012
CompletedFirst Posted
Study publicly available on registry
June 20, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2012
CompletedJune 20, 2012
June 1, 2012
6 months
April 13, 2012
June 18, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (1)
Change in baseline quality of life assessment.
baseline and 6 month QOL questionaire
Study Arms (2)
Hydroxyurea,blood transfusion
ACTIVE COMPARATORHydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week.
Hydroxyurea, Epiao
EXPERIMENTAL* 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.
Interventions
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 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.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
hematology clinic ,pediatrics hospital, Ain Shams University hospital
Cairo, Egypt
Related Publications (1)
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.
PMID: 23927461DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Mohsen S Elalfy, professor
Ain Shams University
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- prof. Mohsen el alfy
Study Record Dates
First Submitted
April 13, 2012
First Posted
June 20, 2012
Study Start
June 1, 2012
Primary Completion
December 1, 2012
Study Completion
December 1, 2012
Last Updated
June 20, 2012
Record last verified: 2012-06