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)
N/A
N/A
Both
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
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
- HIV
- Patients with metal implants thus prohibiting MRI evaluations
Other protocol-defined inclusion/exclusion criteria may apply
Type of Study:
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
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:
No
Principal Investigator
Novartis Pharmaceuticlas
Investigator Role:
Study Director
Investigator Affiliation:
Novartis Pharmaceuticals
Authority:
United States: Food and Drug Administration
Study ID:
CRAD001M2301
NCT ID:
NCT00789828
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
Name | Location |
|---|---|
| University of Alabama at Birmingham | Birmingham, Alabama 35294-3300 |
| University of Chicago Comer Children's Hospital | Chicago, Illinois 60637 |
| Children's Healthcare of Atlanta | Atlanta, Georgia 30342 |
| University of California at Los Angeles | Los Angeles, California 90095 |
| Barrow Tuberous Sclerosis Center | Phoenix, Arizona 85013 |
| Texas Scottish Rite Hospital for Children | Dallas, Texas 75219 |
| Cincinnati Children's Hospital Medical Center Cincinnati Children's Hosp | Cincinnati, Ohio 45229-3039 |
| Massachusetts General Hospital Mass General | Boston, Massachusetts 02114 |
| Children's Hospital Oakland Hematology/Oncology Dept | Oakland, California 94609-1809 |
| Children's Hospital Boston SC-1 | Boston, Massachusetts 02115 |
| Minnesota Epilepsy Group - PA | St. Paul, Minnesota 55102-2383 |
| Children's Regional Outpatients Center SC | Fairfax, Virginia 22031 |
