A Phase I/II Study of Everolimus and Intravesical Gemcitabine in BCG-Refractory Primary or Secondary Non-Muscle-Invasive Bladder Cancer

Full Title
Phase I/II Study of RAD001 and Intravesical Gemcitabine in BCG-Refractory Primary or Secondary Carcinoma In Situ of the Bladder

Standard management for patients with high-risk bladder cancer that has not invaded the muscle and persists despite BCG therapy is removal of the bladder (cystectomy). There are a few drugs for patients who do not respond to intravesical BCG (giving the drug directly into the bladder through a small catheter), but they are associated with a low rate of cure.

The purpose of this study is to evaluate the safety and effectiveness of giving intravesical gemcitabine as well as oral everolimus to patients with high-risk non-muscle-invasive bladder cancer whose disease persists despite BCG therapy. Up to three doses of everolimus will be evaluated in the phase I portion of the study.

In a previous study, intravesical gemcitabine was shown to be effective in patients with high-risk non-muscle-invasive bladder cancer. However, the duration of response was short.

Everolimus is an oral drug that inhibits a molecule called mTOR, which helps cancer cells grow. Everolimus is approved for the treatment of renal cell carcinoma, but its use for treating bladder cancer is considered investigational.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a confirmed diagnosis of high-risk non-muscle-invasive bladder cancer that persists despite BCG therapy.
  • At least 4 weeks must have passed since completion of prior therapy and entry into the study, and patients must have recovered from any side effects.
  • Patients may not have received prior treatment with an mTOR inhibitor (everolimus, temsirolimus, or sirolimus).
  • Patients must be age 18 or older.

For more information about this study and to inquire about eligibility, please contact Dr. Guido Dalbagni at 646-422-4394.

Bladder Cancer
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