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Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00006400
Recruitment Status : Completed
First Posted : October 13, 2000
Last Update Posted : April 27, 2011
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary:
The purpose of this study is to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia.

Condition or disease Intervention/treatment Phase
Hematologic Diseases Anemia, Sickle Cell Drug: Hydroxyurea Drug: Placebo Phase 3

Detailed Description:


In 1995, the Multicenter Study of Hydroxyurea (MSH) demonstrated that hydroxyurea is effective in decreasing the frequency of painful crises, hospitalizations for crises, acute chest syndrome, and blood transfusions by 50%. The recently completed phase II study of hydroxyurea in children (PED HUG) demonstrated that children have a response to hydroxyurea similar to that seen in adults in terms of increasing fetal hemoglobin levels and total hemoglobin, and decreasing complications associated with sickle cell anemia. In addition, this study demonstrated that the drug does not adversely affect growth and development between the ages of 5 and 15. A recently completed pilot study of hydroxyurea given to children between the ages of 6 months and 24 months demonstrated that the drug is tolerated well by small infant, and that the fetal hemoglobin switch can be forced to remain in the "on position" by hydroxyurea administration.

A Special Emphasis Panel (SEP) met on April 12, 1996 to review the results of the MSH trial and the progress to date of the PED HUG study. The SEP recommended that NHLBI undertake the BABY HUG trial.


BABY HUG is a randomized, double-blind, placebo-controlled study to determine if hydroxyurea can prevent the onset of chronic end organ damage in young children with sickle cell anemia. Approximately 200 children with sickle cell disease will be recruited to receive either hydroxyurea or placebo. The children will be screened at study entry for signs of abnormal brain, kidney, pulmonary, and splenic function, and developmental milestones. They will then be randomly assigned to receive either hydroxyurea or placebo and followed yearly to assess chronic end organ damage of the major organ systems. The primary endpoint will be a 50% reduction in rates of damage to the major organs with surrogate markers of organ function during follow-up in Phase II of the trial.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 191 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Prevention
Official Title: Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG)
Study Start Date : August 2000
Actual Primary Completion Date : September 2009
Actual Study Completion Date : September 2009

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Active Comparator: 1
Participants will receive hydroxyurea.
Drug: Hydroxyurea
Participants will receive hydroxyurea.

Placebo Comparator: 2
Participants will receive placebo.
Drug: Placebo
Participants will receive placebo.

Primary Outcome Measures :
  1. 50% reduction in rates of damage to the major organs with surrogate markers of organ function [ Time Frame: Measured during follow-up evaluations ]

Information from the National Library of Medicine

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Ages Eligible for Study:   9 Months to 18 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Majority fetal and sickle (FS or SF) hemoglobin pattern confirmed centrally by electrophoresis (screening may begin at 7 months of age)

Exclusion Criteria:

  • Chronic transfusion therapy
  • Cancer
  • Less than 5th percentile (10th percentile for the pilot study) height, weight, or head circumference for age
  • Severe developmental delay (e.g., cerebral palsy or other mental retardation, Grade III/IV intraventricular hemorrhage)
  • Stroke with neurological deficit
  • Surgical splenectomy
  • Participating in other clinical intervention trials
  • Probable or known diagnosis of Hemoglobin S-Hereditary Persistence of Fetal Hemoglobin
  • Known hemoglobin S-beta plus thalassemia (hemoglobin A present)
  • Any condition or chronic illness, which in the opinion of the principal investigator, makes participation unadvised or unsafe
  • Inability or unwillingness to complete baseline (pre-enrollment) studies, including blood or urine specimen collection, liver-spleen scan, abdominal sonogram, neurological examination, neuropsychological testing, or transcranial Doppler ultrasound (interpretable study not required, but confirmed velocity greater than 200 cm/sec results in ineligibility)
  • Previous or current treatment with hydroxyurea (HU) or another anti-sickling drug
  • The following exclusion criteria are transient; patients can be re-evaluated for eligibility:

    1. Hemoglobin less than 6.0 gm/dL
    2. Reticulocyte count less than 80,000/cu mm if hemoglobin is less than 9 gm/dL
    3. Neutrophil count less than 2,000/cu mm
    4. Platelet count less than 130,000/cu mm
    5. Blood transfusion in the 2 months prior to study entry unless HbA is less than 10%
    6. ALT greater than twice the upper limit of normal
    7. Ferritin less than 10 ng/ml
    8. Serum creatinine greater than twice the upper limit of normal for age
    9. Bayley standardized mental score below 70

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 identifier (NCT number): NCT00006400

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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
Washington, District of Columbia, United States, 20060
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30342
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Michigan
Children's Hospital of Michigan/Wayne State Univ.
Detroit, Michigan, United States, 48201
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, New York
SUNY Health Science Center, Brooklyn
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 SW Medical Center
Dallas, Texas, United States, 75390
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
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Principal Investigator: Sherron Jackson, MD Medical University of South Carolina
Principal Investigator: James F. Casella, MD Johns Hopkins University
Principal Investigator: Lori Luchtman-Jones, MD Children's National Research Institute
Principal Investigator: Rathi V. Iyer, MD University of Mississippi Medical Center
Principal Investigator: Scott T. Miller, MD SUNY Health Science Center, Brooklyn
Principal Investigator: Sohail R. Rana, MD Howard University
Principal Investigator: Zora R. Rogers, MD University of Texas SW Medical Center
Principal Investigator: Bruce W Thompson, Ph.D. Clinical Trials and Surveys Corp
Principal Investigator: Julio Barredo, MD University of Miami Medical Center
Study Chair: Winfred C. Wang, MD St. Jude Children's Research Hospital
Principal Investigator: Courtney Thornburg, MD Duke University
Principal Investigator: Thomas Howard, MD University of Alabama at Birmingham
Principal Investigator: Lori Luck, MD Drexel University
Principal Investigator: R. Clark Brown, MD, PhD Emory University
Principal Investigator: Sharada Sarnaik, MD Wayne State University
Publications of Results:

Other Publications:
Publications automatically indexed to this study by Identifier (NCT Number):

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Responsible Party: Jonathan Goldsmith, MD, NHLBI Identifier: NCT00006400    
Other Study ID Numbers: 89
N01 HB07150
N01 HB07151
N01 HB07152
N01 HB07153
N01 HB07154
N01 HB07155
N01 HB07156
N01 HB07157
N01 HB07158
N01 HB07159
N01 HB07160
First Posted: October 13, 2000    Key Record Dates
Last Update Posted: April 27, 2011
Last Verified: April 2011
Keywords provided by National Heart, Lung, and Blood Institute (NHLBI):
Blood Diseases
Sickle Cell Anemia
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors