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Sponsored by: |
National Heart, Lung, and Blood Institute (NHLBI) |
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Information provided by: | National Heart, Lung, and Blood Institute (NHLBI) |
ClinicalTrials.gov Identifier: | NCT00006400 |
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 | Intervention | Phase |
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Hematologic Diseases Anemia, Sickle Cell |
Drug: Hydroxyurea Drug: Placebo |
Phase III |
Study Type: | Interventional |
Study Design: | Prevention, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment, Efficacy Study |
Official Title: | Pediatric Hydroxyurea in Sickle Cell Anemia (BABY HUG) |
Estimated Enrollment: | 200 |
Study Start Date: | August 2000 |
Estimated Study Completion Date: | September 2009 |
Estimated Primary Completion Date: | September 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Active Comparator
Participants will receive hydroxyurea.
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Drug: Hydroxyurea
Participants will receive hydroxyurea.
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2: Placebo Comparator
Participants will receive placebo.
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Drug: Placebo
Participants will receive placebo.
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BACKGROUND:
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.
DESIGN NARRATIVE:
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.
Ages Eligible for Study: | 9 Months to 18 Months |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
The following exclusion criteria are transient; patients can be re-evaluated for eligibility:
United States, District of Columbia | |
Children's Research Institute | |
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, Maryland | |
Johns Hopkins University | |
Baltimore, Maryland, United States, 21287 | |
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, 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 |
Study Chair: | Julio Barredo, MD | Medical University of South Carolina |
Study Chair: | James F. Casella, MD | Johns Hopkins University |
Study Chair: | Caterina Minnetti, MD | Children's Research Institute |
Study Chair: | Rathi V. Iyer, MD | University of Mississippi Medical Center |
Study Chair: | Scott T. Miller, MD | SUNY Health Science Center, Brooklyn |
Study Chair: | Sohail R. Rana, MD | Howard University |
Study Chair: | Zora R. Rogers, MD | University of Texas SW Medical Center |
Study Chair: | Bruce Thompson | Clinical Trials and Surveys Corporation |
Study Chair: | Stuart Toledano, MD | University of Miami |
Study Chair: | Winfred C. Wang, MD | St. Jude Children's Research Hospital |
Study Chair: | Sherri A. Zimmerman, MD | Duke University |
Responsible Party: | Howard University ( Sohail R. Rana, MD ) |
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 |
Study First Received: | October 12, 2000 |
Last Updated: | October 14, 2008 |
ClinicalTrials.gov Identifier: | NCT00006400 |
Health Authority: | United States: Federal Government |
Blood Diseases Sickle Cell Anemia |
Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hydroxyurea Hematologic Diseases Hemoglobinopathies |
Anemia Anemia, Hemolytic Hemoglobinopathy Anemia, Sickle Cell Sickle cell anemia |
Antisickling Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses |
Hematologic Agents Enzyme Inhibitors Nucleic Acid Synthesis Inhibitors Pharmacologic Actions |