Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
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 use of RAD001 to treat angiomyolipomas in
tuberous sclerosis or sporadic LAM is considered experimental.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
RAD001 tolerance
Every three months while on study drug
Yes
John Bissler, MD
Principal Investigator
Children's Hospital Medical Center, Cincinnati
United States: Food and Drug Administration
CCHMC IRB# 2008-0333
NCT00792766
December 2008
December 2013
Name | Location |
---|---|
Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio 45229-3039 |