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

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ClinicalTrials.gov Identifier: NCT01783990
Recruitment Status : Active, not recruiting
First Posted : February 5, 2013
Last Update Posted : September 26, 2016
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
Brief Summary:
The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys. There was also a chance that treatment could prevent painful crises, lung disease, stroke, and blood infection.

Condition or disease Intervention/treatment
Sickle Cell Anemia Drug: Hydroxyurea

Detailed Description:
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.

Study Design

Study Type : Observational
Actual Enrollment : 158 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
Study Start Date : October 2012
Estimated Primary Completion Date : December 2016
Estimated Study Completion Date : December 2016

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea
U.S. FDA Resources

Groups and Cohorts

Group/Cohort Intervention/treatment
Active

Blood and urine specimens, and questionnaires related to the child's health status.

  • Liver Spleen Scan
  • Abdominal Ultrasound
  • Brain MRI/MRA
  • Cardiac Echocardiogram/Pulmonary Function Testing
  • Transcranial Doppler
  • Neuropsychology Testing (Vineland, WISC-IV, Connor CPT II and Peds QOL)
Drug: Hydroxyurea
Active and Passive groups both are on HU.
Other Name: HU
Passive
Information from usual clinical care of sickle cell disease. We will collect information from routine tests ordered by the child's clinical sickle cell doctors.
Drug: Hydroxyurea
Active and Passive groups both are on HU.
Other Name: HU


Outcome Measures

Primary Outcome Measures :
  1. The primary objective of Follow-Up Study II is to monitor the continued safety and potential efficacy of HU treatment. [ Time Frame: Every 6 months ]
    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.


Biospecimen Retention:   Samples With DNA
Stored blood and urine

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   24 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
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.
Criteria

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
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01783990


Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, District of Columbia
Children's National Medical Center Center for Cancer and Blood Disorders
Washington, District of Columbia, United States, 20010
Howard University College of Medicine
Washington, District of Columbia, United States, 20060
United States, Florida
University of Miami School of Medicine
Miami, Florida, United States, 33136
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30342
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center
Baltimore, Maryland, United States, 21215
United States, Michigan
Children's Hospital of Michigan/Wayne State University
Detroit, Michigan, United States, 48201
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, New York
Downstate Medical Center
Brooklyn, New York, United States, 11203
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
United States, Texas
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States, 75390
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
National Institutes of Health (NIH)
Investigators
Principal Investigator: Susan Assmann, PhD New England Research Institutes, Watertown, MA
More Information

Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT01783990     History of Changes
Other Study ID Numbers: HHSN268201200023C
First Posted: February 5, 2013    Key Record Dates
Last Update Posted: September 26, 2016
Last Verified: September 2016

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors