Clinical Trial: RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
Brief Summary:
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.
Detailed Summary:
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
Sponsor: Children's Hospital Medical Center, Cincinnati
Current Primary Outcome: Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline [ Time Frame: 12 months ]
Original Primary Outcome:
- The primary outcome measures will be the volumetrically determined angiomyolipoma burden
- Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline
- Patients will be considered complete responders if their angiomyolipomas decreases by 75% or more.
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Children's Hospital Medical Center, Cincinnati
Dates:
Date Received: April 6, 2007
Date Started: August 2005
Date Completion:
Last Updated: September 23, 2013
Last Verified: September 2013
