NCT00006400

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 193 (actual)
• Sponsor: National Heart, Lung, and Blood Institute (NHLBI) · NIH
• Sex: All
• Age: 9 Months – 18 Months
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Hematologic Diseases
• Anemia, Sickle Cell

Interventions

Type	Name	Description
DRUG	Hydroxyurea	Participants will receive hydroxyurea.
DRUG	Placebo	Participants will receive placebo.

Timeline

• Start date: 2000-08-01
• Primary completion: 2009-09-01
• Completion: 2009-09-01
• First posted: 2000-10-13
• Last updated: 2020-08-19
• Results posted: 2020-08-19

Locations

14 sites across 1 country: United States