Overview

This trial is active, not recruiting.

Condition growth and development
Treatments everolimus, envirolimus drug class as prescribed by physician
Phase phase 4
Targets mTOR, FKBP-12
Sponsor Novartis Pharmaceuticals
Start date December 2014
End date February 2026
Trial size 15 participants
Trial identifier NCT02338609, 2013-003795-13, CRAD001M2305

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety study
Intervention model single group assignment
Masking open label
Arm
(Experimental)
All patients will have been previously treated with everolimus as part of CRAD001M2301. Continued treatment with everolimus is allowed but not required for participation in this study. However, the physician may choose to place the patient on another treatment.
everolimus
Blood sample and Tanner Stage assessment
(Experimental)
envirolimus drug class as prescribed by physician
At the discretion of the investigator, patients may be treated with commercially available everolimus, as per local product information/ standard of care.

Primary Outcomes

Measure
Percentage of patients who achieved Tanner Stage V at or before age 16 (females) or 17 (males)
time frame: Annually up to 12 years
Height/Body Mass Index (BMI) standard deviation score since baseline
time frame: Annually up to 12 years
Mean Endocrine laboratory values
time frame: Annually up to 12 years

Secondary Outcomes

Measure
Long-term safety as assessed by National Cancer Institute (NCI) Common Toxicity Criteria or Adverse Events (CTCAE)
time frame: Annually up to 12 years
Age at menarche/thelarche (females) or adrenarche (males)
time frame: Annually up to 12 years
Age at Tanner Stage II, III, IV, V
time frame: Annually up to 12 years
Neuropsychological development
time frame: Annually up to 12 years
Height/BMI standard deviation score by year since baseline
time frame: Annually up to 12 years
Percentage of patients who achieved Tanner Stage V at or before age 16 (females) or 17 (males)
time frame: Annually up to 12 years

Eligibility Criteria

Male or female participants up to 17 years old.

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

Additional Information

Official title Long-term Follow-up Study to Monitor the Growth and Development of Pediatric Patients Previously Treated With Everolimus in Study CRAD001M2301
Description 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.
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Novartis.