This subproject is one of many research subprojects utilizing theresources provided by a Center grant funded by NIH/NCRR. The subproject andinvestigator (PI) may have received primary funding from another NIH source,and thus could be represented in other CRISP entries. The institution listed isfor the Center, which is not necessarily the institution for the investigator.Pediatric Hydroxyurea Phase III Clinical Trial; a Randomized Double-blind Placebo Controlled Trial of Hydroxyurea Therapy in Very Young Children. The study will be preceded by a two-year Internal Pilot Study.All the children enrolled in the Internal Pilot Study and the main study will receive study treatment for the duration of 104 weeks. All children in the Internal Pilot Study will be followed until the last child's study treatment has ended with observation for untoward effects of discontinuing study treatment. After study treatment ends, renewed consent will be requested for continued follow-up clinic visits at 3, 6, 9, 12, 16, 20, 24, and 30 months after the end of study treatment, and every six months thereafter for up to five years.OBJECTIVES: Objectives of the Internal Pilot Study: 1. To determine the feasibility of BABY HUG in terms of recruitment, follow-up, adherence to study treatment, and compliance with the study schedule of procedures. 2. To assess Hydroyurea toxicity, effect on growth and development, and occurrence of severe/unexpected adverse events. 3. To establish the distribution and inter-observer and intra-observer variability of spleen function based on dual, independent readings of liver-spleen scans. 4. To evaluate the validity and variability of glomerular filtration rate (GFR) as estimated by serum creatinine and height (the Schwartz formula) compared with a 'gold standard' such as DTPA clearance. 5. to assess some pharmacokinetics parametes of Hydroxyurea can reduce in the BABY HUG age group. Objective of the Main Study: 1. To determine whether daily oral Hydroxyurea can reduce by /=50% chronic organ damage that develops in young children with sickle cell anemia. 2. To determine the relationship between fetal hemoglobin (HbF) levels and chronic organ damage in young children with sickle cell anemia. 3. To investigate the safety of Hydroxyurea for young children with sickle cell anemia regarding a. physcial growth and development, b. neuropsychological development, c. immunological responses, and d. mutagenic effects on DNA.
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