Long-term Follow-up for Growth and Development of Pediatric Patients From CRAD001M2301
Long-term Follow-up for Growth and Development of Pediatric Patients From CRAD001M2301
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.
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.
Inclusion Criteria:

 -  Pediatric female patients who were on study treatment in study [CRAD001M2301] within
 the past 63 months and have not reached Tanner Stage V or age 16 at the time of
 completion of [CRAD001M2301] or

 -  Pediatric male patients who were on study treatment in study [CRAD001M2301] within the
 past 6 months and have not reached Tanner Stage V or age 17 at the time of completion
 of [CRAD001M2301]

 -  Written informed consent according to local guidelines

 Exclusion Criteria:

 -  Pediatric female patients who were on study treatment in CRAD001M2301 and have not
 reached Tanner Stage V but are within 3 month of turning age 16 or

 -  Pediatric male patients who were on study treatment in CRAD001M2301 and have not
 reached Tanner Stage V but are within 3 months of turning age 17

 -  Any patient who was pregnant prior to start of CRAD001M2305

 Other protocol-defined Inclusion/Exclusion criteria