Study to Evaluate the Safety and Efficacy of Sirolimus, in Subject With Refractory Solid Tumors

Study Overview
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Brief Summary
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This study is a single arm, pilot study of sirolimus in patient with Phosphatidylinositide-3-kinase (PIK3CA) mutation, PIK3CA amplification , PIK3CA-AKT pathway aberration Refractory solid tumor and/or specific sensitivity to mTOR inhibitors by Avatar scan that has progressed following standard therapy or that has not responded to standard therapy or for which there is no standard therapy. sirolimus 1mg will be administered orally daily. To investigate the efficacy and safety of sirolimus in patient with Refractory solid tumor.

Official Title
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Study to Evaluate the Safety and Efficacy of Sirolimus, in Subject With Refractory Solid Tumors

Conditions
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Refractory Solid Tumors

Intervention / Treatment
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* Drug: sirolimus


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Provision of fully informed consent prior to study specific procedures. Patients must be >= 19 years of age Phosphatidylinositide-3-kinase (PIK3CA) mutation, PIK3CA amplification , PIK3CA-AKT pathway aberration( H1047R, E542K, E545K, PTEN LOSS) Refractory solid tumor and/or specific sensitivity to mTOR inhibitors by Avatar scan that has progressed following standard therapy or that has not responded to standard therapy or for which there is no standard therapy. ECOG Performance status0-2 Have measurable or evaluated disease based on RECIST 1.1 as determined by investigator. Adequate Organ Function Laboratory values Absolute neutrophil count >= 1.5 x 109/L, Hemoglobin >= 9g/dL, Platelets>=75 x 109/L Bilirubin <= 1.5 x upper limit of normal AST/ALT <= 2.5 X upper limit of normal(5.0 x upper limit of normal, for subject with liver metastases) Creatinine<= 1.5 X UNL Patients of child-bearing potential should be using adequate contraceptive measures should not be breast feeding and must have a negative pregnancy test prior to start of dosing Adequate heart function Exclusion Criteria: Patients with second primary cancer, except:adequately treated non-melanoma skin cancer, curatively treated in-situ cancer of the cervix, or other solid tumor curatively treated with no evidence of disease for <= 5 years. Has known active central nervous system(CNS) metastases Has an active infection requiring systemic therapy Pregnancy or breast feeding Patients with cardiac problem Any previous treatment with sirolimus

Ages Eligible for Study
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Minimum Age: 19 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: sirolimus<br>sirolimus 1mg will be administered orally daily | Drug: sirolimus<br>* Rapamune<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression Free survival |  | 6 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Response Rate |  | 24 months | 
| Time to progression |  | 24 months | 
| Overall survival |  | 24 months | 
| Number of subjects with Adverse Events as a measure of safety |  | 24 months |