To determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia.

Condition	Treatment or Intervention	Phase
Blood Disease Anemia, Sickle Cell	Drug: Hydroxyurea	Phase III

Further Study Details: 

BACKGROUND: In 1995, the Multicenter Study of Hydroxyurea (MSH Trial) demonstrated that hydroxyurea is effective in decreasing the frequency of painful crises, hospitalizations for crises, acute chest syndrome, and blood transfusions by 50 percent. 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 infants, 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 the NHLBI undertake the BABY HUG trial.

DESIGN NARRATIVE: BABY HUG is a randomized, double blind, placebo controlled trial 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 willl be recruited to receive either hydroxyurea or placebo. The children will be screened at trial start-up for signs of abnormal brain, renal, pulmonary, and splenic function, and for developmental milestones. They will then be randomly assigned to receive either hydroxyurea or placebo and followed with yearly studies of chronic end organ damage of the major organ systems. The primary endpoints will be a 50 percent reduction in rates of damage to the major organs with surrogate markers of organ function to be used during follow-up in Phase II of the trial.

Ages Eligible for Study:  up to  2 Years,  Genders Eligible for Study:  Both

Criteria

No eligibility criteria

District of Columbia
      Children's Research Institute, Washington,  District of Columbia,  20010,  United States; Recruiting
      Howard University, Washington,  District of Columbia,  20060,  United States; Recruiting
Florida
      University of Miami, Miami,  Florida,  33136,  United States; Recruiting
Maryland
      Johns Hopkins University, Baltimore,  Maryland,  21287,  United States; Recruiting
Mississippi
      University of Mississippi Medical Center, Jackson,  Mississippi,  39216,  United States; Recruiting
New York
      SUNY Health Science Center, Brooklyn, Brooklyn,  New York,  11203,  United States; Recruiting
North Carolina
      Duke University Medical Center, Durham,  North Carolina,  27710,  United States; Recruiting
South Carolina
      Medical University of South Carolina, Charleston,  South Carolina,  29425,  United States; Recruiting
Tennessee
      St. Jude Children's Research Hospital, Memphis,  Tennessee,  38105,  United States; Recruiting
Texas
      University of Texas SW Medical Center, Dallas,  Texas,  75390,  United States; Recruiting

Study chairs or principal investigators

Julio Barredo,  Medical University of South Carolina   
James Casella,  Johns Hopkins University   
Catherine Driscoll,  Children's Research Institute   
Rathi Iyer,  University of Mississippi Medical Center   
Scott Miller,  SUNY Health Science Center, Brooklyn   
Sohail Rana,  Howard University   
Zora Rogers,  University of Texas SW Medical Center   
Bruce Thompson,  Clinical Trials and Surveys Corp.   
Stuart Toledano,  University of Miami   
Winfred Wang,  St. Jude Children's Research Hospital   
Sherri Zimmerman,  Duke University   

Study ID Numbers:  89
Record last reviewed:  March 2005
Last Updated:  April 1, 2005
Record first received:  October 12, 2000
ClinicalTrials.gov Identifier:  NCT00006400
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08