Conditions: Tuberous Sclerosis Subependymal Giant Cell Astrocytoma
Keywords: SEGA Tuberous Sclerosis Subependymal Giant Cell Astrocytoma mTOR RAD001 Mamalian Target Rapamycin Everolimus TSC
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 117
Healthy volunteers: no

Brief summary:
This study evaluated the efficacy and safety of Everolimus in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.

Detailed description:

Arms:
arm 1: Everolimus was administered orally at a starting dose of 4.5mg/m\^2 daily and subsequently titrated to attain whole blood trough concentration of 5 to 15 ng/mL. Dose adjustments were permitted based on safety and whole blood trough concentrations. arm 2: Matching Placebo administered orally.

Interventions: intervention 1: Everolimus intervention 2: Placebo
Intervention descriptions: intervention 1: Everolimus was formulated as tablets of 1.0-mg strength and was blisterpacked under aluminum foil in units of 10 tablets. intervention 2: Placebo was provided as a matching tablet and was blisterpacked under aluminum foil in units of 10.

Protocol document: