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Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex

Phase 1/Phase 2
3 Years
Open (Enrolling)
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma

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Trial Information

Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex

Tuberous Sclerosis Complex (TSC)is a genetic disorder with a birth incidence of
approximately one in six thousand. Five to twenty percent of patients with TSC will develop
astrocytoma, a slowly progressive tumor. They grow and cause damage to surrounding brain
tissue, blockage of spinal fluid (hydrocephalus), blindness, trouble walking, seizures, and
brain damage. If untreated, they can be fatal. Standard treatment involves surgery to
remove the tumor; however surgery may itself cause brain damage, bleeding, or infection, as
well as other complications. Studies have shown that everolimus suppresses the chemicals
that cause tumors to grow in tuberous sclerosis, and may cause them to shrink.

The primary objective of this study is to find out the effects of everolimus on astrocytomas
in a six month trial in patients with Tuberous Sclerosis who have been diagnosed with an

Inclusion Criteria:

- Age three years and older

- If female and of child bearing potential, documentation of negative pregnancy test
prior to enrollment and, where applicable, use of appropriate, non- estrogen
containing birth control contraceptive regimen whle on study that is to be continued
3 months after discontinuation of everolimus. Use of barrier methods alone will not
constitute an acceptable contraceptive regimen.

- Clinically definite diagnosis of tuberous sclerosis (modified Gomez criteria or
positive genetic test)

- Presence of giant cell astrocytoma as defined by imaging characteristics and serial
increase in size of lesion on 2 or more MRI scans

- Adequate renal function (creatinine < 1.5 mg/dl)

Exclusion Criteria:

- Serious intercurrent medical illness or other uncontrolled medical disease which
could compromise participation in the study

- Significant hematologic or hepatic abnormality

- Continuous requirement for supplemental oxygen

- Intercurrent infection at initiation of oxygen

- Recent surgery (within 2 months of initiation of everolimus)

- Pregnant or lactating women

- Use of an investigational drug within the last 30 days

- Must be adequately recovered from the acute toxicities of any prior therapy

- Clinical evidence of impending herniation or focal neurologic deficit related to the
subject's astrocytoma

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Primary Outcome Measure will be incidence of reported and observed adverse side effects as a percentage of patients enrolled in the study and treated with everolimus. These will be analysed at 6 months after study initiation.

Outcome Time Frame:

During the entire study

Safety Issue:


Principal Investigator

David N Franz, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospital Medical Center, Cincinnati


United States: Food and Drug Administration

Study ID:

CCHMC IRB# 06-07-50



Start Date:

December 2006

Completion Date:

December 2015

Related Keywords:

  • Tuberous Sclerosis
  • Subependymal Giant Cell Astrocytoma
  • Tuberous Sclerosis Complex (TSC)
  • Subendymal Giant Cell Astrocytomas (SEGA'S)
  • Mammalian Target of Rapamycin (mTOR)
  • Everolimus (RAD001)
  • Tuberous Sclerosis with Subependymal Giant Cell Astrocytomas
  • Astrocytoma
  • Sclerosis
  • Tuberous Sclerosis



Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039