NCT02338609

Long-term Monitoring of Growth and Development of Pediatric Patients Previously Treated With Everolimus

Long-term Follow-up Study to Monitor the Growth and Development of Pediatric Patients Previously Treated With Everolimus in Study CRAD001M2301 (EXIST-LT)

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 0 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of CRAD001M2305 was 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 monitored the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301 (NCT00789828) until they reach Tanner Stage V, or until age 16 for females or 17 for males whichever occurred first.

Detailed description

CRAD001M2305 was a prospective, multi-center phase IIIb/IV study. This study investigated if the physical and sexual development of pediatric patients was affected by previous or ongoing treatment with everolimus. The study monitored the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301(NCT00789828) until they reached Tanner Stage V, or until age 16 for females or 17 for males whichever occurred first. Continued treatment with everolimus was at investigator discretion and was 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 was followed at annual visits to the site until patients achieve Tanner Stage V or age 16 (females), age 17 (males) whichever occurred first. Adverse events, concomitant medication, appearance of menarche, were monitored and data collected every 3 months ("3-monthly"). Unless clinically indicated these 3-monthly visits were performed per telephone.

Conditions

• Growth and Development

Interventions

Type	Name	Description
DRUG	Everolimus	At the discretion of the investigator, pediatric patients could be treated with commercially available everolimus, as per local product information / standard of care. Treatment duration and dose modifications were at the investigator's discretion, as per the local product information.

Timeline

• Start date: 2014-12-17
• Primary completion: 2023-12-18
• Completion: 2023-12-18
• First posted: 2015-01-14
• Last updated: 2024-08-09
• Results posted: 2024-08-09

Locations

6 sites across 3 countries: United States, Belgium, Russia

Regulatory

• FDA-regulated drug study