Cerebrovascular Accident Clinical Trial
Official title:
Stroke With Transfusions Changing to Hydroxyurea
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).
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 (5.0-18.9 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.
;
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention
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