Phase 2
N=30
Everolimus for Cancer With TSC1 or TSC2 Mutation
TSC1 · TSC2 · Tuberous Sclerosis Complex · MTOR
Bottom Line
View on ClinicalTrials.gov: NCT02201212 ↗Enrolled (actual)
30
Serious AEs
10.0%
Results posted
Sep 2020
Primary outcome: Primary: Objective Response Rate — 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Everolimus (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Dana-Farber Cancer Institute
- Primary completion
- Jun 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Objective Response Rate |
2 | — |
| SECONDARY Duration of Response |
12.7 | — |
| SECONDARY Progression-free Survival |
2.0 | — |
| SECONDARY Overall Survival |
7.27 | — |
| SECONDARY Toxicity Rate |
3 | — |
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.
Eligibility Criteria
Inclusion Criteria: Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- Participants must have histologically confirmed advanced malignancy that is either metastatic and/or unresectable and/or recurrent, with confirmed inactivating mutations in TSC1 or TSC2, or activating mutations in MTOR, identified in any CLIA-certified laboratory. All genetic findings must be reviewed by the study PI, Dr. David Kwiatkowski, prior to study entry.
- Biopsy of a primary or metastatic lesion must have been performed within the past two years. Sufficient pathologic material must be available to enable whole exome sequencing at the time of study entry.
- Participants must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded) as ≥ 20 mm with conventional techniques or as ≥10 mm with spiral CT scan. See section 10 for the evaluation of measureable disease.
- Participants may have received any number of prior therapies, from 0 to > 10, but prior treatment with PI3-kinase or mTOR inhibitors is not permitted.
- Age ≥ 18 years.
- ECOG performance status 8% despite adequate therapy. Patients with a known history of impaired fasting glucose or diabetes mellitus (DM) may be included, however blood glucose and antidiabetic treatment must be monitored closely throughout the trial and adjusted as necessary.
Data sourced from ClinicalTrials.gov (NCT02201212). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.