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Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol


N/A
24 Months
18 Years
Open (Enrolling by invite only)
Both
Sickle Cell Anemia

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Trial Information

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.


Inclusion Criteria:



- All subjects enrolled in the BABY HUG Follow-Up I Study who participated for at least
24 months are eligible for the Follow-Up Study II

Exclusion Criteria:

- Subjects that have received a Stem Cell Transplant are not eligible for enrollment

Type of Study:

Observational

Study Design:

Observational Model: Cohort, Time Perspective: Retrospective

Outcome Measure:

The primary objective of Follow-Up Study II is to monitor the continued safety and potential efficacy of HU treatment.

Outcome Description:

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.

Outcome Time Frame:

Every 6 months

Safety Issue:

Yes

Principal Investigator

Bruce Thompson, Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Clinical Trials and Surveys

Authority:

United States: Food and Drug Administration

Study ID:

HHSN268201200023C

NCT ID:

NCT01783990

Start Date:

October 2012

Completion Date:

December 2016

Related Keywords:

  • Sickle Cell Anemia
  • Anemia
  • Anemia, Sickle Cell

Name

Location

University of Mississippi Medical CenterJackson, Mississippi  39216-4505
Medical University of South CarolinaCharleston, South Carolina  29425-0721
St. Jude Children's Research HospitalMemphis, Tennessee  38105-2794
Emory University School of MedicineAtlanta, Georgia  30322
University of Texas Southwestern Medical Center at DallasDallas, Texas  75235-8897
University of Alabama at BirminghamBirmingham, Alabama  35294-3300
Johns Hopkins University School of MedicineBaltimore, Maryland  21205
Duke University Medical CenterDurham, North Carolina  27710
Howard University College of MedicineWashington, District of Columbia  20059
University of Miami School of MedicineMiami, Florida  33136
Downstate Medical CenterBrooklyn, New York  11203
Children's National Medical Center Center for Cancer and Blood DisordersWashington, District of Columbia  20010
Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient CenterBaltimore, Maryland  21215
Children's Hospital of Michigan/Wayne State UniversityDetroit, Michigan  48201