Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

Completed · Phase 3 · Has a placebo group

Conditions studied: Hematologic Diseases, Anemia, Sickle Cell

In brief

The purpose of this study is to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia.

Key facts

Study ID

NCT00006400

Run by

National Heart, Lung, and Blood Institute (NHLBI)

People needed

193

Starts

2000-08-01

Expected to finish

2009-09-01

Last updated by the study team

2020-08-19

Who can join

Age: 1 and older, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Majority fetal and sickle (FS or SF) hemoglobin pattern confirmed centrally by electrophoresis (screening may begin at 7 months of age)

You may not qualify if…

• 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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Howard University — Washington D.C., District of Columbia, United States
• University of Miami — Miami, Florida, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Children's Hospital of Michigan/Wayne State Univ. — Detroit, Michigan, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• SUNY Health Science Center, Brooklyn — Brooklyn, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Drexel University — Philadelphia, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• University of Texas SW Medical Center — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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