Conditions: Tuberous Sclerosis Complex (TSC) Lymphangioleiomyomatosis (LAM)
Keywords: Angiomyolipoma AML Tuberous Sclerosis Complex TSC mTOR RAD001 Mammalian Target of Rapamycin Everolimus Afinitor SEGA Subependymal Giant Cell Astrocytoma Seizures Tuberous sclerosis complex (TSC) Tuberous sclerosis benign tumors of brain kidney heart eyes lungs skinTSC1 TSC2 hamaratin tuberin, tumor growth suppressors gyri tubers Sporadic Lymphangioleiomyomatosis. Lymphangioleiomyomatosis (LAM) rare lung disease
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 118
Healthy volunteers: no

Brief summary:
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.

Detailed description:

Arms:
arm 1: Study drug was given by continuous oral daily dosing of two 5 mg tablets. arm 2: Placebo was given by continuous oral daily dosing of two 5 mg tablets.

Interventions: intervention 1: Everolimus (RAD001) intervention 2: Everolimus Placebo
Intervention descriptions: intervention 1: Everolimus is used in 5 mg strength tablets, blister-packed under aluminum foil in units of ten tablets and dosed on a daily basis. 10mg daily dosing throughout the trial. intervention 2: Matching placebo was provided as a matching tablet and was also blister-packed under aluminum foil in units of ten.

Protocol document: