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Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

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ClinicalTrials.gov Identifier: NCT00890396
Recruitment Status : Completed
First Posted : April 29, 2009
Results First Posted : August 20, 2020
Last Update Posted : August 20, 2020
Sponsor:
Information provided by (Responsible Party):
National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary:
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA. The purpose of this follow-up study is to evaluate the long-term effects of hydroxyurea in children who have participated in the BABY HUG study.

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

Detailed Description:

SCA is an inherited blood disorder in which the body makes sickle-shaped red blood cells that contain abnormal hemoglobin. The sickled cells block blood flow in the vessels that lead to limbs and organs. This can cause pain, serious infections, and organ damage. The BABY HUG study (NCT00006400) is examining whether the medication hydroxyurea can prevent organ damage, especially in the spleen and kidneys, in children with SCA. This study is a follow-up study to the BABY HUG study and will enroll children who have participated in the BABY HUG study. The purpose of this study is to examine the long-term effects of using hydroxyurea as a treatment for SCA, including both the risks and benefits. Study researchers will also investigate the optimal age to begin treatment with hydroxyurea in children with SCA.

This study will enroll children between 2 and 7 years old who participated in the BABY HUG study. Hydroxyurea will not be provided to participants as part of this study, but participants may receive the medication from their own doctors. Parents of participants can choose for their child to participate in this study in one of two ways-by enrolling in either a passive follow-up group or an active follow-up group. For participants in the passive follow-up group, study researchers will review participants' medical records every 6 months, in addition to reviewing brain ultrasound tests and computed tomography (CT) or magnetic resonance imaging (MRI) scans, if completed. Participants will have a blood and urine collection at baseline and Year 4 (or at the end of the study, whichever comes first). Participants in the active follow-up group will take part in the same study procedures as participants in the passive follow-up group. In addition, at Year 2, participants in this group will undergo an additional blood and urine collection, a scanning procedure to obtain images of the liver and spleen, a kidney test, neuropsychological testing, and an ultrasound imaging test to evaluate liver and spleen size.

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Study Type : Observational
Actual Enrollment : 163 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Study
Study Start Date : September 2008
Actual Primary Completion Date : December 2011
Actual Study Completion Date : December 2011

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia
Drug Information available for: Hydroxyurea

Group/Cohort Intervention/treatment
Active Follow-up
An active follow-up involved the performance of many of the laboratory tests and procedures done during BABY HUG clinical trials study. These included, but not limited to, serial laboratory parameters that were not part of routine clinical care such as Hgb F levels, pitted cell count, Howell-Jolly Body determination, a liver-spleen scan, diethylenetriaminepentaacetic acid (DTPA) glomerular filtration rate (GFR) measurement, creatinine clearance, Cystatin C, urine concentrating ability, transcranial Doppler, and neuropsychological testing.
Drug: Hydroxyurea
Parents and child's doctor may plan to use or not to use hydroxyurea.

Passive Follow-up
A passive follow-up involved the abstraction of clinical data from the medical record. Results of physical examinations and laboratory tests performed as part of routine clinical care were recorded.
Drug: Hydroxyurea
Parents and child's doctor may plan to use or not to use hydroxyurea.




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: 48 Months from the date of randomization ]
    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 to 48 months 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: 48 Months from the date of randomization ]
    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 to 48 months 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: 48 Months from the date of randomization ]
    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 Randomized to Hydroxyurea vs Placebo [ Time Frame: 72 Months from the date of randomization ]
    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).

  5. 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: 48 Months from the date of randomization ]
    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.

  6. 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: 72 Months from the date of randomization ]
    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.

  7. Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo [ Time Frame: 48 Months from the date of randomization ]
    The change in Howell-Jolly Bodies from randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between the randomized treatment groups (hydroxyurea vs placebo).

  8. Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo [ Time Frame: 72 Months from the date of randomization ]
    The change in Howell-Jolly Bodies from randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between the randomized treatment groups (hydroxyurea vs placebo).

  9. Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit [ Time Frame: 48 Months from the date of randomization ]
    The change in Howell-Jolly Bodies 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.

  10. Change in Howell-Jolly Bodies From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit [ Time Frame: 72 Months from the date of randomization ]
    The change in Howell-Jolly Bodies (HJB) 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


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   2 Years to 7 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children from the initial BABY HUG study who agree to participate in this follow-up study.
Criteria

Inclusion Criteria:

  • All children who completed at least 18 months of follow-up visits in the initial BABY HUG study
  • Children from the initial BABY HUG study who are on a chronic transfusion program or who are recipients of a bone marrow transplant

Exclusion Criteria:

  • Any child who was not enrolled in the initial BABY HUG study for at least 18 months

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): NCT00890396


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
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
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, Pennsylvania
Drexel University
Philadelphia, Pennsylvania, United States, 19134
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)
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):
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Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT00890396    
Other Study ID Numbers: 647
N01 HB07160 ( Other Grant/Funding Number: NHLBI )
First Posted: April 29, 2009    Key Record Dates
Results First Posted: August 20, 2020
Last Update Posted: August 20, 2020
Last Verified: July 2009
Keywords provided by National Heart, Lung, and Blood Institute (NHLBI):
Sickle Cell Anemia
Hydroxyurea
Additional relevant MeSH terms:
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Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors