Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease (NCT01389024) | Clinical Trial Compass
CompletedPhase 2
Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
United States28 participantsStarted 2012-08-16
Plain-language summary
This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease. The investigators plan to identify children 12 to 48 months old without central nervous system complications and randomly assign 20 to treatment with hydroxyurea and 20 to treatment with placebo for 36 months. Neither the study doctors nor the participants will know which treatment they are receiving.
Who can participate
Age range12 Months – 54 Months
SexALL
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Inclusion criteria
✓. Participant must have sickle cell anemia (hemoglobin SS) or sickle Beta-zero (null) thalassemia (hemoglobin S-B0) as confirmed at the local institution by hemoglobin analysis after six months of age.
✓. Participant must be 9 to 48 months of age. All screening procedures except MRI can be completed between 9 and 12 months of age, with the exception of the MRI, for which the child must have reached the age of 12 months.
✓. Informed consent must be signed by the participant's legally authorized guardian acknowledging written consent to join the study.
Exclusion criteria
✕. History of a focal neurologic event lasting more than 24 hours with medical documentation or a history of prior overt stroke.
✕. Other neurological problems, such as neurofibromatosis, lead poisoning, non-febrile seizure disorder, or tuberous sclerosis.
✕. Known human immunodeficiency virus (HIV) infection.
✕. Treatment with anti-sickling drugs or hydroxyurea within 3 months or anticipated treatment during the course of the study.
✕. Chronic blood transfusion therapy, ongoing or planned.
✕. Poor adherence likely per his/her hematologist and study coordinator based on previous compliance in clinic appointments and following advice.
✕. Presence or planned permanent (or semi-permanent) metallic structures attached to their body. (e.g., braces on teeth), which their physicians believe will interfere with the MRI of the brain.
What they're measuring
1
Number of Randomized Participants With Central Nervous System Complications
✕. History of two or more TCD studies with a velocity ≥ 200 cm/sec by the non-imaging technique, or ≥185 cm/sec for the imaging technique or a indeterminate TCD.