Overview

This trial is active, not recruiting.

Conditions tuberous sclerosis, subependymal giant cell astrocytoma
Treatments everolimus, placebo
Phase phase 3
Targets mTOR, FKBP-12
Sponsor Novartis Pharmaceuticals
Start date August 2009
End date March 2011
Trial size 117 participants
Trial identifier NCT00789828, 2007-006997-27, CRAD001M2301

Summary

This study evaluated the efficacy and safety of Everolimus in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Everolimus was administered orally at a starting dose of 4.5mg/m^2 daily and subsequently titrated to attain whole blood trough concentration of 5 to 15 ng/mL. Dose adjustments were permitted based on safety and whole blood trough concentrations.
everolimus RAD001
Everolimus was formulated as tablets of 1.0-mg strength and was blisterpacked under aluminum foil in units of 10 tablets.
(Placebo Comparator)
Matching Placebo administered orally.
placebo
Placebo was provided as a matching tablet and was blisterpacked under aluminum foil in units of 10.

Primary Outcomes

Measure
Subependymal Giant Cell Astrocytomas (SEGA) Response Rate
time frame: From date of randomization until the earliest date of first documented SEGA progression, date of further anti-SEGA medication (including open-label Everolimus)/surgery or analysis cut-off date (02-Mar-2011)

Secondary Outcomes

Measure
Change From Baseline to Week 24 in Total Seizure Frequency Per 24 Hours From Video EEG as Per Central Reader.
time frame: Baseline, Week 24
Time to SEGA Progression Based on Independent Central Radiology Review
time frame: From date of randomization until the earliest date of first documented SEGA progression, date of further anti-SEGA medication (including open-label Everolimus)/surgery or analysis cut-off date (02-Mar-2011)
Skin Lesion Response Rate as Per Investigator Assessment Using the Physician's Global Assessment of Clinical Condition (PGA)
time frame: From date of randomization until the earliest date of first skin lesion progression, date of start of further therapy against skin lesions (including open-label Everolimus) or analysis cut-off date (02-Mar-2011)
Change From Baseline in Angiogenesis Biomarkers
time frame: Baseline, Week 4, Week 12, Week 24, Week 36 and Week 48
Changes in Renal Function Assessed Using Creatinine Clearance/Globerular Filtration Rate.
time frame: From start of treatment and assessed on a continuous basis through the duration of the study

Eligibility Criteria

Male or female participants of any age.

Inclusion Criteria: - All Ages - Definite diagnosis of Tuberous Sclerosis according to the modified Gomez criteria - At least one Subependymal Giant Cell Astrocytoma of at least 1 cm in diameter - Evidence of SEGA worsening as compared to prior MRI scans - Females of child bearing potential must use birth control - Written informed consent Exclusion Criteria: - SEGA related surgery is likely to be required in the opinion of the investigator - Recent heart attack, cardiac related chest pain or stroke - Severely impaired lung function - Severe liver dysfunction - Severe kidney dysfunction - Pregnancy or breast feeding - Current infection - History of organ transplant - Surgery within two months prior to study enrollment - Prior therapy with a medication in the same class as Everolimus - Uncontrolled high cholesterol - Uncontrolled diabetes - HIV - Patients with metal implants thus prohibiting MRI evaluations Other protocol-defined inclusion/exclusion criteria may apply

Additional Information

Official title A Randomized, Double-blind, Placebo-controlled Study of Everolimus in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)
Trial information was received from ClinicalTrials.gov and was last updated in April 2014.
Information provided to ClinicalTrials.gov by Novartis.