RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

• Status: Completed
• Trial end date: 2013-07-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborator: Novartis Pharmaceuticals
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Diagnosed with angiomyolipomas and have either Tuberous Sclerosis Complex or lymphangioleiomyomatosis

• Angiomyolipoma one centimeter or greater in largest diameter

• Between the ages of 18 and 65 years old.

• If female, documentation of negative pregnancy test prior to enrollment and, where applicable, use of appropriate non-estrogen containing birth control contraceptive regimen while on study.

• Adequate renal function (creatinine < 3 mg/dl)

Exclusion Criteria:

• Pregnant or lactating women

• Continuous requirement for supplemental oxygen

• Surgery within past 2 months

• Use of an investigational drug within last 30 days.