This pilot trial studies how well nanoparticle albumin-bound rapamycin works in treating patients with cancer that as has spread to other places in the body and usually cannot be cured or controlled with treatment (advanced cancer) and that has an abnormality in a protein called mechanistic target of rapamycin (mTOR). Patients with this mutation are identified by genetic testing. Patients then receive nanoparticle albumin-bound rapamycin, which may stop the growth of cancer cells by blocking the mTOR enzyme, which is needed for cell growth and multiplication. Using treatments that target a patient's specific mutation may be a more effective treatment than the standard of care treatment.
PRIMARY OBJECTIVES: I. To investigate efficacy of groups of patients defined by disease type, genomic aberration and treatment regimen. II. To assess the confirmed response rate of nanoparticle albumin-bound rapamycin (nab-rapamycin) in mTOR aberrant advanced cancers. (Sub-protocol Arm A) SECONDARY OBJECTIVES: I. To estimate other clinical outcomes (e.g., progression-free and overall survival) of groups of patients defined by disease type, genomic aberration and treatment regimen. II. To describe the adverse event profile of each regimen. III. To assess the clinical benefit rate of nab-rapamycin in mTOR aberrant advanced cancers. (Sub-protocol Arm A). IV. To estimate progression-free survival (specifically at 6 months) and overall survival of these patients. (Sub-protocol Arm A) V. To estimate the adverse event profile of nab-rapamycin. (Sub-protocol Arm A) TERTIARY OBJECTIVES: I. To describe patient health-related quality of life (HRQOL) and symptoms using the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core (QLQ-C)30 in groups of patients defined by disease type and/or treatment regimen and to correlative HRQOL/symptoms with genomic markers. II. To assess the rate of individual mTOR pathway aberrations and assess the association between individual mTOR pathway aberrations and clinical outcome both across disease indications and within disease indications. (Sub-protocol Arm A) OUTLINE: Patients receive nanoparticle albumin-bound rapamycin intravenously (IV) over 30 minutes on days 1 and 8. Treatment repeats every 21 days for 24 weeks in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up periodically.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
2
Correlative studies
Given IV
Ancillary studies
Mayo Clinic in Arizona
Scottsdale, Arizona, United States
Mayo Clinic
Rochester, Minnesota, United States
Proportion of confirmed responses, evaluated using the RECIST v1.1
The proportion of confirmed responses will be estimated by the number of confirmed responses divided by the total number of evaluable patients. An exact binomial confidence interval for the true confirmed response proportion will be calculated. Analysis will be carried out overall and within disease groups where warranted by sample size.
Time frame: Up to 21 days
Clinical benefit rate defined as the proportion of patients with a confirmed response or stable disease (complete response+partial response+stable disease) divided by the total number of evaluable patients
An exact binomial confidence interval for the true confirmed clinical benefit rate will be calculated. Analysis will be carried out overall and within disease groups where warranted by sample size.
Time frame: Up to 5 years
Incidence of adverse events, using the National Cancer Institute Common Terminology Criteria for Adverse Events v4.0
The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration. Analysis will be carried out overall and within disease groups where warranted by sample size.
Time frame: Up to 30 days after last dose of study treatment
Survival time
The distribution of survival time will be estimated using the method of Kaplan-Meier. Analysis will be carried out overall and within disease groups where warranted by sample size.
Time frame: Time from registration to death due to any cause, assessed up to 5 years
Time to disease progression
The distribution of time to progression will be estimated using the method of Kaplan-Meier. The 6-month progression-free rate will be provided. Analysis will be carried out overall and within disease groups where warranted by sample size.
Time frame: Time from registration to the earliest date of documentation of disease progression, assessed up to 5 years
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