Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
Hematologic Diseases, Anemia, Sickle Cell
About this trial
This is an interventional prevention trial for Hematologic Diseases focused on measuring Blood Diseases, Sickle Cell Anemia
Eligibility Criteria
Inclusion Criteria: Majority fetal and sickle (FS or SF) hemoglobin pattern confirmed centrally by electrophoresis (screening may begin at 7 months of age) Exclusion Criteria: Chronic transfusion therapy Cancer Less than 5th percentile (10th percentile for the pilot study) height, weight, or head circumference for age Severe developmental delay (e.g., cerebral palsy or other mental retardation, Grade III/IV intraventricular hemorrhage) Stroke with neurological deficit Surgical splenectomy Participating in other clinical intervention trials Probable or known diagnosis of Hemoglobin S-Hereditary Persistence of Fetal Hemoglobin Known hemoglobin S-beta plus thalassemia (hemoglobin A present) Any condition or chronic illness, which in the opinion of the principal investigator, makes participation unadvised or unsafe Inability or unwillingness to complete baseline (pre-enrollment) studies, including blood or urine specimen collection, liver-spleen scan, abdominal sonogram, neurological examination, neuropsychological testing, or transcranial Doppler ultrasound (interpretable study not required, but confirmed velocity greater than 200 cm/sec results in ineligibility) Previous or current treatment with hydroxyurea (HU) or another anti-sickling drug The following exclusion criteria are transient; patients can be re-evaluated for eligibility: Hemoglobin less than 6.0 gm/dL Reticulocyte count less than 80,000/cu mm if hemoglobin is less than 9 gm/dL Neutrophil count less than 2,000/cu mm Platelet count less than 130,000/cu mm Blood transfusion in the 2 months prior to study entry unless HbA is less than 10% ALT greater than twice the upper limit of normal Ferritin less than 10 ng/ml Serum creatinine greater than twice the upper limit of normal for age Bayley standardized mental score below 70
Sites / Locations
- University of Alabama at Birmingham
- Children's National Medical Center
- Howard University
- University of Miami
- Emory University School of Medicine
- Johns Hopkins University
- Children's Hospital of Michigan/Wayne State Univ.
- University of Mississippi Medical Center
- SUNY Health Science Center, Brooklyn
- Duke University Medical Center
- Drexel University
- Medical University of South Carolina
- St. Jude Children's Research Hospital
- University of Texas SW Medical Center
Arms of the Study
Arm 1
Arm 2
Active Comparator
Placebo Comparator
Hydroxyurea
Placebo
Participants will receive hydroxyurea.
Participants will receive placebo.