ong-term monitoring of growth and development of pediatric patients previously treated with everolimus

Phase 1

• Conditions: Subependymal Giant Cell Astrocytoma (SEGA) associated with Tuberous Sclerosis Complex (TSC); MedDRA version: 21.0Level: LLTClassification code 10045138Term: Tuberous sclerosisSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-003795-13-BE
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-11-17
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 48

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>? Age at menarche (females)<br>? Age at thelarche (females)<br>? Age at adrenarche (males)<br>? Age at Tanner Stages II-V<br>? Assess neuropsychological development by TAND Checklist<br>? Compare CRAD001M2305 and CRAD001MIC03 on height, BMI and<br>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).<br>Height standard deviation score by year <br>Mean endocrine laboratory values (FSH, LH, and estrogen or testosterone) by age;Timepoint(s) of evaluation of this end point: Annually