Clinical Trial: Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Brief Summary:

This is an open label long term follow up study, open to those subjects who were previously enrolled in"RAD001 Therapy of Angiomyolipomata in Patients with Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis", CCHMC IRB #2008-0812 and who meet the criteria for this long-term follow-up study.

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 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: RAD001 tolerance [ Time Frame: Every three months while on study drug ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Angiomyolipoma volume reduction [ Time Frame: Every year while on study drug ]

Original Secondary Outcome: Same as current

Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: November 17, 2008
Date Started: December 2008
Date Completion:
Last Updated: September 17, 2013
Last Verified: September 2013