Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00789828
First received: November 12, 2008
Last updated: April 24, 2014
Last verified: April 2014
Results First Received: March 1, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Tuberous Sclerosis
Subependymal Giant Cell Astrocytoma
Interventions: Drug: Everolimus
Drug: Placebo

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients in the study were randomized in a 2:1 ratio (Everolimus: Placebo).

Reporting Groups
  Description
Everolimus 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.
Placebo Matching Placebo was administered orally.

Participant Flow:   Overall Study
    Everolimus     Placebo  
STARTED     78     39  
COMPLETED     2 [1]   8  
NOT COMPLETED     76     31  
Ongoing in Double Blind                 76                 31  
[1] Completed means discontinued the double-blind treatment period.



  Baseline Characteristics


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   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) ]

2.  Secondary:   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 ]

3.  Secondary:   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) ]

4.  Secondary:   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) ]

5.  Secondary:   Change From Baseline in Angiogenesis Biomarkers   [ Time Frame: Baseline, Week 4, Week 12, Week 24, Week 36 and Week 48 ]
Results not yet reported.   Anticipated Reporting Date:   09/2014   Safety Issue:   No

6.  Secondary:   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 ]
Results not yet reported.   Anticipated Reporting Date:   09/2014   Safety Issue:   Yes


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats


  More Information