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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)

Phase 3
Open (Enrolling)
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma

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Trial Information

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)

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


- Patients with metal implants thus prohibiting MRI evaluations

Other protocol-defined inclusion/exclusion criteria may apply

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Subependymal Giant Cell Astrocytomas (SEGA) Response Rate

Outcome Description:

Sega response Rate is defined as the percentage of patients whose best overall status is response as determined by Independent Central Radiology Review. SEGA response was defined as: (1) a ≥ 50% reduction in SEGA volume relative to baseline (where SEGA volume was the sum of all target SEGA lesion volumes identified at baseline); and (2) no unequivocal worsening of non-target SEGA lesions, no new SEGA lesions (≥ 1 cm in longest diameter), and no new or worsening hydrocephalus.

Outcome 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)

Safety Issue:


Principal Investigator

Novartis Pharmaceuticlas

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

August 2009

Completion Date:

November 2014

Related Keywords:

  • Tuberous Sclerosis
  • Subependymal Giant Cell Astrocytoma
  • SEGA
  • Tuberous Sclerosis
  • Subependymal Giant Cell Astrocytoma
  • mTOR
  • RAD001
  • Mamalian Target Rapamycin
  • Everolimus
  • TSC
  • Astrocytoma
  • Sclerosis
  • Tuberous Sclerosis



University of Alabama at BirminghamBirmingham, Alabama  35294-3300
University of Chicago Comer Children's HospitalChicago, Illinois  60637
Children's Healthcare of AtlantaAtlanta, Georgia  30342
University of California at Los AngelesLos Angeles, California  90095
Barrow Tuberous Sclerosis CenterPhoenix, Arizona  85013
Texas Scottish Rite Hospital for ChildrenDallas, Texas  75219
Cincinnati Children's Hospital Medical Center Cincinnati Children's HospCincinnati, Ohio  45229-3039
Massachusetts General Hospital Mass GeneralBoston, Massachusetts  02114
Children's Hospital Oakland Hematology/Oncology DeptOakland, California  94609-1809
Children's Hospital Boston SC-1Boston, Massachusetts  02115
Minnesota Epilepsy Group - PASt. Paul, Minnesota  55102-2383
Children's Regional Outpatients Center SCFairfax, Virginia  22031