Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00006400
Status:
COMPLETED
Last Update Posted:
2020-08-19
First Post:
2000-10-12
Brief Title:
Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Heart Lung and Blood Institute NHLBI
Study Overview
Official Title:
Pediatric Hydroxyurea Phase III Clinical Trial BABY HUG
Status:
COMPLETED
Status Verified Date:
2011-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| N01 HB07160 | None | None | None |
| N01 HB07150 | None | None | None |
| N01 HB07151 | None | None | None |
| N01 HB07152 | None | None | None |
| N01 HB07153 | None | None | None |
| N01 HB07154 | None | None | None |
| N01 HB07155 | None | None | None |
| N01 HB07156 | None | None | None |
| N01 HB07157 | None | None | None |
| N01 HB07158 | None | None | None |
| N01 HB07159 | None | None | None |