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A study to assess the efficacy of 10 mg/kg/day hydroxyurea versus 20 mg/kg/day hydroxyurea in treatment of Indian sickle cell disease patients.

Phase 3
Not yet recruiting
Conditions
Health Condition 1: D578- Other sickle-cell disorders
Registration Number
CTRI/2022/04/042006
Lead Sponsor
ICMR Task Force
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Not Yet Recruiting
Sex
Not specified
Target Recruitment
0
Inclusion Criteria

1.Confirmed diagnosis of Sickle cell disease and not on hydroxyurea treatment for last 6 months.

2.Age 2-14 years of age.

3.Creatinine less than or equal to 1.4 mg/dl and/or estimated glomerular filtration rate greater than 45ml/min/1.73 m2

4.Liver function tests (especially ALT and conjugated bilirubin) less than or equal to 4 times upper limit of normal.

Exclusion Criteria

1)Red blood cell transfusion in the last 3 months.

2)Patient not willing to participate in the study.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Proportion of children with average hemoglobin rise of more than 2g/dl over 24 month period or average HbF rise by 4 points over 24 month period.Timepoint: 24 months
Secondary Outcome Measures
NameTimeMethod
SCA-related morbidity (over 24 month period on study drug) <br/ ><br>-Pain Event <br/ ><br>-Dactylitis <br/ ><br>-Acute Chest Syndrome <br/ ><br>-Hepatic Sequestration <br/ ><br>-Splenic Sequestration <br/ ><br>-Cerebrovascular events <br/ ><br>-Blood Transfusion requirements <br/ ><br> <br/ ><br>b)Incidence of severe adverse events (SAE) over 24 month period <br/ ><br>Death, Hospitalization more than 7 days, Life threatening events <br/ ><br>c)Incidence of hematologic toxicities over 24 month period on study drug. <br/ ><br>Hemoglobin (Hb) 4.0gm/dl <br/ ><br>Hb6.0 g/dl and absolute reticulocyte count (ARC) 100X109 /L. <br/ ><br>Hb 7.0g/dl and ARC 80 X109/L <br/ ><br>Platelets 80X 109/L <br/ ><br>Absolute neutrophil count (ANC) 1.5X109/L <br/ ><br>d)Change in creatinine levels over 24 month period as a measure of renal function. <br/ ><br> <br/ ><br>Timepoint: 24 months
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