Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
The current observational trial, Follow-Up Study II (designated FUS II) includes enhanced
neuropsychological, brain, cardiac, and pulmonary evaluations for this very well
characterized cohort of subjects. Measures of spleen and renal function and markers of DNA
damage will continue to be collected. 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.
Observational Model: Cohort, Time Perspective: Retrospective
The primary objective of Follow-Up Study II is to monitor the continued safety and potential efficacy of HU treatment.
Safety of HU will be assessed by ongoing clinical monitoring of growth and development, age-appropriate neuropsychological evaluation, serial hematologic and chemistry parameters, and the frequency of expected and unexpected clinical events related to sickle cell disease. Disease and treatment-related effects on the spleen will be measured by a liver-spleen scan, pitted cell counts and HJB enumeration. Kidney function will be measured by GFR estimation from the Schwartz formula or cystatin C measurements and the urine osmolality and urine microalbumin:creatinine ratio. The BH FU II investigators will also seek to study how early treatment with HU changes the child's disease trajectory on open-label HU.
Every 6 months
Bruce Thompson, Ph.D.
Clinical Trials and Surveys
United States: Food and Drug Administration
|University of Mississippi Medical Center||Jackson, Mississippi 39216-4505|
|Medical University of South Carolina||Charleston, South Carolina 29425-0721|
|St. Jude Children's Research Hospital||Memphis, Tennessee 38105-2794|
|Emory University School of Medicine||Atlanta, Georgia 30322|
|University of Texas Southwestern Medical Center at Dallas||Dallas, Texas 75235-8897|
|University of Alabama at Birmingham||Birmingham, Alabama 35294-3300|
|Johns Hopkins University School of Medicine||Baltimore, Maryland 21205|
|Duke University Medical Center||Durham, North Carolina 27710|
|Howard University College of Medicine||Washington, District of Columbia 20059|
|University of Miami School of Medicine||Miami, Florida 33136|
|Downstate Medical Center||Brooklyn, New York 11203|
|Children's National Medical Center Center for Cancer and Blood Disorders||Washington, District of Columbia 20010|
|Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center||Baltimore, Maryland 21215|
|Children's Hospital of Michigan/Wayne State University||Detroit, Michigan 48201|