Phase III trial of Novartis drug Afinitor® met primary endpoint of reducing SEGA tumor size in patients with tuberous sclerosis
Novartis announced today Phase III trial results that showed more than one-third of patients taking Afinitor® (everolimus) tablets* experienced a 50% or greater reduction in the size of their subependymal giant cell astrocytomas (SEGAs), non-cancerous brain tumors associated with tuberous sclerosis complex (TSC)[1],[2],[6].
- (1888PressRelease) July 08, 2011 - This study, the largest prospective clinical trial to date in this patient population, is being presented on Saturday, July 9 at the International TSC Research Conference in Washington, D.C.
* Largest Phase III study in tuberous sclerosis complex (TSC) showed 35% of patients treated with everolimus had a 50% or greater reduction in SEGA volume versus 0% on placebo[1]
* Subependymal giant cell astrocytomas (SEGAs) are non-cancerous brain tumors that can cause life-threatening brain swelling in children and adults with TSC[1],[2],[3],[4]
* Study showed clinically meaningful results in secondary endpoints including time to SEGA progression and improvement in skin lesions[1]
* This study supports the findings from a separate Phase II study of everolimus which now provides data on some patients up to three years[5]
Tuberous sclerosis complex affects approximately one to two million people worldwide and is associated with a variety of resulting disorders including seizures, swelling in the brain (hydrocephalus), developmental delays and skin lesions[2],[6]. Also known as tuberous sclerosis (TS), TSC is a genetic disorder that may cause non-cancerous tumors to form in vital organs and can affect many different parts of the body, most commonly the brain and kidney[6],[7]. Signs and symptoms of TSC vary depending on which system and which organs are involved[6]. SEGAs occur in up to 20% of patients with TSC. In countries where everolimus is not approved, brain surgery is the only treatment option for patients with growing SEGAs[2].
The 117-patient, randomized, placebo-controlled Phase III EXIST-1 (EXamining everolimus In a Study of TSC) trial met its primary endpoint of SEGA response rate, with 35% of patients (27 of 78) receiving everolimus experiencing a 50% or greater reduction in SEGA volume (sum of volumes of all target SEGAs) relative to baseline versus 0% of patients (0 of 39) on placebo (p
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