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Hydroxyurea in Young Children With Sickle Cell Anemia

Not Applicable
Completed
Conditions
Sickle Cell Anemia
Interventions
Registration Number
NCT00519701
Lead Sponsor
Duke University
Brief Summary

The purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage.

Detailed Description

Previous studies have shown that hydroxyurea therapy in adults and older children with SCA improves laboratory parameters and ameliorates the clinical severity of disease. Little is known, however, about the effects of hydroxyurea on the chronic organ damage that occurs in patients with SCA and leads to significant morbidity and mortality in young adults. The objectives of this study are to assess the safety and efficacy of HU in young children with SCA and to determine whether HU preserves renal function, reduces transcranial doppler ultrasound (TCD) values, and prevents development of brain ischemia as evidenced by MRI/MRA imaging. In addition, we will evaluate the effects of hydroxyurea on quality of life

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
14
Inclusion Criteria
  • Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)
Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
1hydroxyureahydroxyurea
Primary Outcome Measures
NameTimeMethod
Transcranial doppler ultrasound velocity2 years
Magnetic resonance imaging/angiography2 years
Glomerular Filtration Rate2 years
Quality of Life2 years
Neurocognitive outcomes2 years
Secondary Outcome Measures
NameTimeMethod
Growth parameters2 years
Hematological parameters2 years

Trial Locations

Locations (1)

Duke University Medican Center

🇺🇸

Durham, North Carolina, United States

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