Clinical Trial: Everolimus for Cancer With TSC1 or TSC2 Mutation

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase II Trial of Everolimus for Cancer Patients With Inactivating Mutations in TSC1 or TSC2 or Activating MTOR Mutations

Brief Summary:

In this research study, the investigators are evaluating the clinical benefit of everolimus in cancer patients with inactivating TSC1 or TSC2 mutations or activating MTOR mutations.

This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called everolimus to learn whether the drug works in treating a specific cancer. "Investigational" means that the drug is being studied. It also means that the FDA (the U.S. Food and Drug Administration) has not yet approved everolimus for your type of cancer.

Everolimus is a drug that may stop cancer cells from growing by blocking an important factor (mTOR) involved in the growth of cells. This drug has been used in treatment for other cancers and is approved by the Food and Drug Administration for treatment of several types of cancer, including renal cell carcinoma. Treatment with this drug has been associated with responses in some patients whose cancers had mutations in TSC1 or TSC2. The investigators think that patients whose tumors have mutations in TSC1 or TSC2 may have a good chance of responding to treatment with drugs like everolimus.


Detailed Summary: Patients who fulfill eligibility criteria will be entered into the trial.The participant will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 28 days (4 weeks), during which time the participant will be taking the study drug orally (by mouth) once daily. The diary will also include special instructions for taking the study drug. In addition to the administration of the study drugs the participant will be asked to return to the clinic at various time points so that additional exams can be performed. These study visits may last as long as 2 hours.
Sponsor: Dana-Farber Cancer Institute

Current Primary Outcome: Objective Response Rate [ Time Frame: Baseline, Every 8 weeks, 2 Years ]

RECIST 1.1 criteria


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Duration of Response Rate [ Time Frame: Baseline, Every 8 weeks, 2 Years ]
  • Progression-free Survival Rate [ Time Frame: Baseline, Up to 2 Years ]
  • Overall Survival Rate [ Time Frame: 2 Years ]
  • Toxicity Rate [ Time Frame: 2 Years ]
    CTCAE v4.0


Original Secondary Outcome: Same as current

Information By: Dana-Farber Cancer Institute

Dates:
Date Received: July 23, 2014
Date Started: September 2014
Date Completion: January 2022
Last Updated: February 8, 2017
Last Verified: February 2017