BABY HUG FU II: PEDIATRIC HYDROXYUREA PHASE III CLINICAL TRIAL FOLLOW-UP OBSERVATIONAL STUDY II
The purpose of this observational study is to perform clinical follow-up of subjects enrolled in the original Pediatric Hydroxyurea Phase III (BABY HUG) Clinical Trial through at least the first decade of life following completion of their period of randomized study treatment. This long-term follow-up study was envisioned at the conception of the BABY HUG Treatment Study, and parents/guardians as well as local Institutional Review Boards (IRB) were made aware of the Investigators[AND]amp;apos; intent to follow the subjects in the consent forms obtained during the BABY HUG Treatment Study enrollment period. BABY HUG Follow-Up Study II includes enhanced neuropsychological, brain, cardiac, and pulmonary evaluations for this very well characterized cohort of subjects. This unique population facilitates a follow up study of this magnitude. Subjects may elect to participate in one of the two study arms. The Passive arm consists of lab work whereas the Active arm incorporates additional studies including echocardiography, pulmonary function testing and MRI/MRA. Assessment of other target organs in sickle cell disease including pulmonary and cardiac function will be performed in addition to evaluation of developmental aspects of SCD and potential HU toxicity. The overall goals of the BABY HUG Follow-Up Study II are to define more accurately the long-term risks and benefits of early HU treatment and to evaluate organ function, growth and psychosocial development, and the predictive value of biomarkers. Follow-Up Study II will have one primary endpoint analysis: comparison of the proportion of children with decreased or absent spleen function. All of this information is needed to determine the potential benefits or possible risks of HU intervention early in the life of subjects with SCD and provide information pertaining to possible practice guidelines for the first decade of life.
All subjects who have completed at least 24 months of on the BABY HUG Follow-Up Study I (STU# 102010-056) are eligible for this follow-up study. (In general, that is otherwise healthy children with majority fetal and sickle hemoglobin patterns who entered the BABY HUG study between the ages of 9 and 17 months with no other major complications.)