Everolimus for Cancer With TSC1 or TSC2 Mutation
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|ClinicalTrials.gov Identifier: NCT02201212|
Recruitment Status : Active, not recruiting
First Posted : July 28, 2014
Last Update Posted : November 5, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|TSC1 TSC2 Tuberous Sclerosis Complex MTOR||Drug: Everolimus||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of Everolimus for Cancer Patients With Inactivating Mutations in TSC1 or TSC2 or Activating MTOR Mutations|
|Study Start Date :||September 2014|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||January 2022|
- Objective Response Rate [ Time Frame: Baseline, Every 8 weeks, 2 Years ]RECIST 1.1 criteria
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02201212
|United States, Massachusetts|
|Dana Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|United States, New York|
|Memorial Sloan Kettering Cancer Center|
|New York, New York, United States, 10065|
|Principal Investigator:||David Kwiatkowski, MD, PhD||Dana-Farber Cancer Institute|