Tuberous Sclerosis Clinical Trial
Official title:
RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
The purpose of this research study is to find out what effects RAD001 has on angiomyolipomas
of a person with Tuberous Sclerosis Complex and to determine the safe dose of RAD001 without
toxicity.
The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly
even cause regression.
Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one
in six thousand. Angiomyolipomas occur in eighty percent of persons with TS, and in up to
60% of persons with LAM. They are fatty tumors that often occur in kidney or liver as well
as other parts of the body. They can grow and cause damage to surrounding kidney tissue and
even renal failure. They may also leak blood causing potentially life-threatening
hemorrhage.
Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow
in tuberous sclerosis and sporadic LAM.
The primary goal of this study is to evaluate the clinical effectiveness of RAD001 in a 24
month trial. Although the primary goal is to determine if the drug RAD001 has effects on
angiomyolipomas, other diseases associated with tuberous sclerosis will be monitored too,
specifically any change in involvement of your brain or lungs with tuberous sclerosis. The
use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered
experimental
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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