CRAD001M2305 is a prospective, multi-center phase IIIb/IV study. This study will investigate if the physical and sexual development of pediatric patients is affected by previous or ongoing treatment with everolimus. The study will monitor the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301 until they reach Tanner Stage V, or until age 16 for females or 17 for males whichever occurs first. Continued treatment with everolimus is at investigator discretion and is not required for participation in CRAD001M2305. Growth (height, weight), and sexual development (Tanner Stages, sex hormone levels, age at menarche, thelarche (females) and adrenarche (males)), and brain development (assessed by TAND Checklist, dates of brain MRI) of patients participating in this long-term follow-up study will be followed at annual visits to the site until patients achieve Tanner Stage V or age 16 (females), age 17 (males) whichever occurs first. Adverse events, concomitant medication, appearance of menarche, will be monitored and data collected every 3 months (3-monthly). Unless clinically indicated these 3-monthly visits can be performed per telephone.
The primary objective of CRAD001M2305 is to report the long-term effects of everolimus treatment on height, weight and sexual development (using Tanner Stages) in children and adolescents with Tuberous Sclerosis Complex (TSC)-associated with Subependymal Giant Cell Astrocytoma (SEGA). The study will monitor the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301 until they reach Tanner Stage V, or until age 16 for females or 17 for males whichever occurs first.