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Sirolimus in Treating Patients With Solid Tumors That Are Metastatic or Cannot Be Removed By Surgery

Phase 1
Completed
Conditions
Unspecified Adult Solid Tumor, Protocol Specific
Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
Other: pharmacological study
Other: laboratory biomarker analysis
Registration Number
NCT01791088
Lead Sponsor
University of Chicago
Brief Summary

This clinical trial studies sirolimus in treating patients with solid tumors that are metastatic or cannot be removed by surgery. Sirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth

Detailed Description

PRIMARY OBJECTIVES:

I. To describe the magnitude, inter-individual variability and time course of sirolimus-induced changes in fasting serum glucose and triglycerides.

SECONDARY OBJECTIVES:

I. To assess candidate genetic variants for their correlation with changes in fasting glucose and/or triglycerides.

II. To assess tumor response by the Response Evaluation Criteria In Solid Tumors (RECIST, version 1.1) and explore whether there is any correlation between response and changes in fasting glucose and/or triglycerides.

III. To assess toxicity by the Common Terminology Criteria for Adverse Events (CTCAE, version 4.0) and explore whether there is any correlation between toxicities and changes in fasting glucose and/or triglycerides.

IV. To quantify and determine the functional status of circulating regulatory T cells (Tregs) before and during treatment.

OUTLINE:

Patients receive sirolimus orally (PO) on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
56
Inclusion Criteria
  • Histologically or cytologically confirmed solid tumor that is metastatic or unresectable and for which standard curative or palliative measures do not exist or are no longer effective
  • Weight >= 40 kg
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 1
  • Life expectancy > 3 months
  • Absolute neutrophil count (ANC) >= l500/ul
  • Hemoglobin >= 9g/dL
  • Platelets >= 100,000/ ul
  • Total bilirubin < 1.5 x upper limit of normal
  • Serum glutamic oxaloacetic transaminase (SGOT) and serum glutamic pyruvate transaminase (SGPT) < 2.5 x upper limit of normal for patients without liver metastases OR SGOT and SGPT < 5 x upper limit of normal for patients with liver metastases
  • Measurable or non-measurable disease will be allowed
  • Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation, up until 30 days after final study treatment; should a woman become pregnant or suspect that she is pregnant while participating in this study, she should inform her treating physician immediately
  • Patients taking substrates, inhibitors, or inducers of cytochrome P450 (CYP)3A4 should be encouraged to switch to alternative drugs whenever possible, given the potential for drug-drug interactions with sirolimus
  • Signed informed consent
Exclusion Criteria
  • Prior treatment with a mammalian target of rapamycin (mTOR) inhibitor (including sirolimus) is allowed; however, patients with >= grade 3 toxicities with an mTOR inhibitor are excluded
  • Fasting glucose > 126 mg/dL or fasting triglycerides > 150 mg/dL; patients are allowed to be on oral anti-hyperglycemic and anti-lipid therapies, but cannot be on insulin
  • Patients who have had chemotherapy or immunotherapy within 3 weeks of starting study drug, or radiotherapy within 14 days of starting study drug, or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
  • Patients may not be receiving any other investigational agents or any concomitant antineoplastic therapy, with the exception of androgen ablating agents (for patients with prior prostate cancer)
  • Serious underlying medical or psychiatric illnesses that would, in the opinion of the treating physician, substantially increase the risk for complications related to treatment; similarly, any unstable medical condition that in the opinion of the treating physician or study investigators, would interfere with determination of the study objectives
  • Pregnancy or breastfeeding
  • Major surgery within 4 weeks

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (sirolimus)pharmacological studyPatients receive sirolimus PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment (sirolimus)laboratory biomarker analysisPatients receive sirolimus PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment (sirolimus)sirolimusPatients receive sirolimus PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Primary Outcome Measures
NameTimeMethod
Change in fasting glucose and fasting triglyceridesBaseline to 29 days

These data will be analyzed by fitting mixed effects models for longitudinal data. A model linear in time with random patient intercept and slope effects will be fit initially and residuals examined. If a linear model does not fit the data adequately a quadratic term will be added. Simple paired t-tests of the glucose and triglyceride levels on day 8, 15, and 29 relative to baseline will also be performed.

Secondary Outcome Measures
NameTimeMethod
Change in tumor size assessed using RECISTUp to 2 years

The change in tumor size will be plotted against the change in glucose/triglyceride and Pearson correlation coefficients will be calculated.

Correlation of toxicities graded using CTCAE version 4.0 with glucose/triglyceride changesUp to 2 years

Proportional odds models will be fit to assess whether there is a correlation between toxicities (graded from 0 to 5) and glucose/triglyceride changes.

Regulatory T cells (Tregs)Baseline to 28 days

Descriptive statistics will be used to report the quantity and phenotype of Tregs on days 1 and 29 of therapy.

Association between genetic variants and changes in fasting glucose and triglyceridesUp to 12 months

Two-sample t tests will be performed. A Bonferroni adjustment will be applied to control for the number of candidate markers evaluated. Logistic regression models will be fit to determine whether changes in glucose and/or triglycerides are associated with tumor response (complete or partial).

Trial Locations

Locations (1)

University of Chicago Comprehensive Cancer Center

🇺🇸

Chicago, Illinois, United States

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