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

Novartis

Status and phase: Completed; Phase 3

Conditions: Subependymal Giant Cell Astrocytoma; Tuberous Sclerosis

Treatments: Drug: Everolimus; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00789828

2007-006997-27 (EudraCT Number)

CRAD001M2301

Details and patient eligibility

About

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

Enrollment: 117 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

117 participants in 2 patient groups, including a placebo group

Everolimus

Experimental group

Description:

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.

Treatment:

Drug: Everolimus

Placebo

Placebo Comparator group

Description:

Matching Placebo administered orally.

Treatment:

Drug: Placebo