Subependymal Giant Cell Astrocytoma (SEGA) associated with Tuberous Sclerosis Complex (TSC) MedDRA version: 21.0 Level: LLT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive nam
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Pediatric female patients who were on study treatment in study CRAD001M2301 within the past 3 months and have not reached Tanner Stage V or age 17 at the time of completion of study CRAD001M2301 or 2. Pediatric male patients who were on study treatment in study CRAD001M2301 within the past 3 months and have not reached Tanner Stage V or age 17 at the time of completion of study CRAD001M2301. 3. Written informed consent according to local guidelines. Are the trial subjects under 18? yes Number of subjects for this age range: 48 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Pediatric female patients who were on study treatment in study CRAD001M2301 and have not reached Tanner Stage V but are within 3 month of turning age 16 or 2. Pediatric male patients who were on study treatment in study CRAD001M2301 and have not reached Tanner Stage V but are within 3 months of turning age 17 3. Any patient who was pregnant prior to start of study CRAD001M2305
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To monitor the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in Study CRAD001M2301, who had received everolimus as part of study CRAD001M2301 and may or may not be continuing treatment with everolimus.;Secondary Objective: ? Long-term safety ? Age at menarche (females) ? Age at thelarche (females) ? Age at adrenarche (males) ? Age at Tanner Stages II-V ? Assess neuropsychological development by TAND Checklist ? Compare CRAD001M2305 and CRAD001MIC03 on height, BMI and sexual development (using Tanner Stages);Primary end point(s): Percentage of patients who achieved Tanner Stage V at or before age of 16 (females) or 17 (males). Height standard deviation score by year Mean endocrine laboratory values (FSH, LH, and estrogen or testosterone) by age;Timepoint(s) of evaluation of this end point: Annually | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Annually except for adverse events, concomitant medication and appearance of menarche, which will be monitored every 3 months;Secondary end point(s): Age at start of menses (menarche) – for females Age at onset of breast development (thelarche) – for females Age at pubic hair development (adrenarche) – for males Age at Tanner Stage II-V - all patients TAND Checklist individual responses over time since the enrollment date Indirect comparison between CRAD001M2305 and CRAD001MIC03 will be performed on the following endpoints: Percentage of patients who achieved Tanner Stage V at or before age of 16 (females) or 17 (males). Height/BMI standard deviation score by year since baseline. Safety will be assessed by the National Cancer Institute’s (NCI) Common Toxicity Criteria for Adverse Events (CTCAE), version 3.0 Notes: ? Safety assessments will consist of monitoring and recording adverse and serious adverse events ? Safety follow-up as per product information | — |
Countries
Belgium, Canada, Poland, Russian Federation, United States
Contacts
Public ContactClinical Trial Information desk
Novartis Pharma AG
Outcome results
None listed