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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 : Completed
First Posted : February 5, 2013
Results First Posted : August 20, 2020
Last Update Posted : August 20, 2020
Sponsor:
Collaborator:
National Institutes of Health (NIH)
Information provided by (Responsible Party):
National Heart, Lung, and Blood Institute (NHLBI)

Study Description
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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 ((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 sickle cell disease (SCD) and potential HU toxicity.
Study Design
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Study Type : Observational
Actual Enrollment : 150 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
Actual Primary Completion Date : December 31, 2016
Actual Study Completion Date : December 31, 2016

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Groups and Cohorts
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Group/Cohort Intervention/treatment
Active
Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), and urine microalbumin:creatinine ratio were collected at study entry, annually, and exit to Follow-Up Study II. Variable-diversity-joining (VDJ) and a stored blood sample were collected at study entry and study exit. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, magnetic resonance imaging (MRI) / magnetic resonance angiography (MRA), cardiac echocardiogram, or neuropsychology testing were collected once during the study when the child was 10 years old.
 Drug: Hydroxyurea
Parents and child's doctor may plan to use or not to use hydroxyurea.
Passive
Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), variable-diversity-joining (VDJ), urine microalbumin:creatinine ratio and a stored blood sample were collected at study entry and exit to Follow-Up Study II. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, MRI/MRA, cardiac echocardiogram, or neuropsychology testing were collected as part of clinical care.
 Drug: Hydroxyurea
Parents and child's doctor may plan to use or not to use hydroxyurea.


Outcome Measures
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Primary Outcome Measures :
  1. Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo [ Time Frame: baseline and when child turned 10 years old ]
    The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between the randomized treatment groups (hydroxyurea vs placebo). The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, decreased to normal, absent to absent, or absent to decreased.

  2. Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit [ Time Frame: baseline and when child turned 10 years old ]
    The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, decreased to normal, absent to absent, or absent to decreased.

  3. Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo [ Time Frame: Baseline and End of follow-up II study (up to 13 years from randomization date) ]
    The change in the percentage of pitted cell from randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between the randomized treatment groups (hydroxyurea vs placebo).

  4. Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit [ Time Frame: Baseline and End of follow-up II study (up to 13 years from randomization date) ]
    The change in the percentage of pitted cell from the randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit.

  5. Change in Howell Jolly Body (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo [ Time Frame: Baseline and End of follow-up II study (up to 13 years from randomization date) ]
    The change in Howell Jolly Body from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell Jolly Body was compared between the randomized treatment groups (hydroxyurea vs placebo).

  6. Change in Howell Jolly Body (HJB) Count From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea [ Time Frame: Baseline and End of follow-up II study (up to 13 years from randomization date) ]
    The change in Howell Jolly Body count from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit.


Biospecimen Retention:   Samples With DNA
Stored blood and urine

Eligibility Criteria
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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:   Non-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
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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
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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
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Principal Investigator: Susan Assmann, PhD New England Research Institutes, Watertown, MA
  Study Documents (Full-Text)

Documents provided by National Heart, Lung, and Blood Institute (NHLBI):
Informed Consent Form  [PDF] October 16, 2006
Study Protocol and Statistical Analysis Plan  [PDF] August 9, 2009

More Information
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Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT01783990    
Other Study ID Numbers: HHSN268201200023C
First Posted: February 5, 2013    Key Record Dates
Results First Posted: August 20, 2020
Last Update Posted: August 20, 2020
Last Verified: September 2016
Additional relevant MeSH terms:
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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