RAD001 in treatment of patients with SEGA associated with TSC
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC)
IRAS ID
17157
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2007-006997-27
Clinicaltrials.gov Identifier
Research summary
Tuberous Sclerosis Complex (TSC) is a rare genetic disorder that causes benign (non-cancerous) tumours. TSC is caused by genetic mutations within one of two genes which code for proteins that normally suppress tumour growth. People with TSC are therefore susceptible to tumour growths which typically occur in the brain, heart, liver, kidneys and lungs leading to severe complications in the effected organs. Brain tumours associated with TSC can result in seizures, autism, learning disability and epilepsy, and are the primary cause of death in childhood. Subependymal giant cell astrocytomas (SEGA) are tumours that form deep within the brain and are almost always associated with TSC. SEGA occurs in 5 to 15% of patients with TSC and by the time symptoms are noted they are often untreatable by surgical methods and standard chemotherapy or radiotherapies are ineffective. Current therapies for SEGA only manage symptoms (i.e. epilepsy) and do not address the underlying cause of the disease. RAD001 is drug that is currently used to prevent organ rejection in patients who have had kidney and heart transplantations. Recent clinical trials using RAD001 have shown it to be an effective anti-cancer treatment and potentially an appropriate treatment of TSC-associated SEGA. Novartis Pharmaceuticals is sponsoring this phase III study which aims to evaluate the anti-tumour activity of RAD001 versus a placebo (dummy drug) in patients with TSC-associated SEGA. This will be a randomised, double blinded study, meaning the participant will be randomly (like tossing a coin) allocated to receive either RAD001 or placebo. Furthermore neither the doctor nor the participant will know which medication is being given. However, should a patient progress whilst on study the investigator can proceed to find out which treatment they have been receiving, if the patient has been receiving placebo they can be offered further treatment with RAD001. It is anticipated that approximately 99 patients will be recruited.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
09/H0502/102
Date of REC Opinion
15 Sep 2009
REC opinion
Favourable Opinion