Ignite Creation Date:
2024-05-05 @ 11:46 AM
Last Modification Date:
2024-10-26 @ 9:12 AM
Study NCT ID:
NCT00122980
Status:
TERMINATED
Last Update Posted:
2013-01-18
First Post:
2005-07-20
Brief Title:
Stroke With Transfusions Changing to Hydroxyurea
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Stroke With Transfusions Changing to Hydroxyurea
Status:
TERMINATED
Status Verified Date:
2012-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The study has been stopped due to safety and futility concerns
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 compare standard therapy transfusions and chelation with alternative therapy hydroxyurea and phlebotomy for the prevention of secondary stroke and management of iron overload in children with sickle cell anemia SCA
Detailed Description:
BACKGROUND
Stroke occurs in 10 of children with SCA and has a very high risk of recurrence without therapy Affected children receive chronic erythrocyte transfusions to prevent a secondary stroke which are effective but have limited long-term utility due to transmission of infectious agents erythrocyte alloantibody and autoantibody formation and iron overload Transfusion acquired iron overload can cause chronic organ damage with hepatic fibrosis and cirrhosis poor growth and development cardiac arrhythmias and early sudden death in young patients with SCA and stroke An alternative to transfusions for secondary stroke prevention that also addresses the issue of transfusion acquired iron overload is clearly needed Hydroxyurea can prevent acute vaso-occlusive events in SCA but its utility for cerebrovascular disease and for the prevention of secondary stroke in SCA is not proven Pilot data indicate hydroxyurea can prevent stroke recurrence in children with SCA after transfusions are discontinued serial phlebotomy reduces iron burden
DESIGN NARRATIVE
This is a Phase III randomized clinical trial for children with SCA The hypothesis is that hydroxyurea and phlebotomy can maintain an acceptable stroke recurrence rate and significantly reduce the hepatic iron burden The primary aim is to compare standard therapy transfusions and chelation with alternative therapy hydroxyurea and phlebotomy for the prevention of secondary stroke and management of iron overload Additional aims include comparisons of growth and development frequency of non-stroke neurological and other sickle-related events and quality of life The use of hydroxyurea for secondary stroke prevention coupled with removal of excess iron by phlebotomy would represent a significant improvement in the management of individuals with SCA and stroke If hydroxyurea is effective for the prevention of secondary stroke it may also be beneficial for other children with SCA and cerebrovascular disease including those at risk for primary stroke
The trial includes approximately 130 children 50-189 years of age with 65 subjects per treatment arm with SCA who have had symptomatic cerebral infarctions and have been treated with red cell transfusions for at least 18 months After completing baseline screening studies half the participants will be switched to a therapeutic program of hydroxyurea and phlebotomy Half of the participants will remain on transfusion and chelation The composite primary endpoint in this study is to compare two modalities of treatment for the prevention of secondary stroke and management of iron overload The impetus for this trial is the fact that long-term transfusion and chelation therapy in children is difficult is frequently unsuccessful and is often complicated by severe symptomatic iron overload particularly of the heart lungs and liver
Stroke occurs in 10 of children with SCA and has a very high risk of recurrence without therapy Affected children receive chronic erythrocyte transfusions to prevent a secondary stroke which are effective but have limited long-term utility due to transmission of infectious agents erythrocyte alloantibody and autoantibody formation and iron overload Transfusion acquired iron overload can cause chronic organ damage with hepatic fibrosis and cirrhosis poor growth and development cardiac arrhythmias and early sudden death in young patients with SCA and stroke An alternative to transfusions for secondary stroke prevention that also addresses the issue of transfusion acquired iron overload is clearly needed Hydroxyurea can prevent acute vaso-occlusive events in SCA but its utility for cerebrovascular disease and for the prevention of secondary stroke in SCA is not proven Pilot data indicate hydroxyurea can prevent stroke recurrence in children with SCA after transfusions are discontinued serial phlebotomy reduces iron burden
DESIGN NARRATIVE
This is a Phase III randomized clinical trial for children with SCA The hypothesis is that hydroxyurea and phlebotomy can maintain an acceptable stroke recurrence rate and significantly reduce the hepatic iron burden The primary aim is to compare standard therapy transfusions and chelation with alternative therapy hydroxyurea and phlebotomy for the prevention of secondary stroke and management of iron overload Additional aims include comparisons of growth and development frequency of non-stroke neurological and other sickle-related events and quality of life The use of hydroxyurea for secondary stroke prevention coupled with removal of excess iron by phlebotomy would represent a significant improvement in the management of individuals with SCA and stroke If hydroxyurea is effective for the prevention of secondary stroke it may also be beneficial for other children with SCA and cerebrovascular disease including those at risk for primary stroke
The trial includes approximately 130 children 50-189 years of age with 65 subjects per treatment arm with SCA who have had symptomatic cerebral infarctions and have been treated with red cell transfusions for at least 18 months After completing baseline screening studies half the participants will be switched to a therapeutic program of hydroxyurea and phlebotomy Half of the participants will remain on transfusion and chelation The composite primary endpoint in this study is to compare two modalities of treatment for the prevention of secondary stroke and management of iron overload The impetus for this trial is the fact that long-term transfusion and chelation therapy in children is difficult is frequently unsuccessful and is often complicated by severe symptomatic iron overload particularly of the heart lungs and liver
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 |
|---|---|---|---|
| 1U01HL078787-01A1 | NIH | None | httpsreporternihgovquickSearch1U01HL078787-01A1 |