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

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.

Study Overview

• Status: Completed
• Conditions: Refractory Solid Tumors
• Intervention / Treatment: Drug: sirolimus

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 4

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 99999
    • Samsung Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: sirolimus; sirolimus 1mg will be administered orally daily	Drug: sirolimus; Rapamune

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Progression Free survival	6 Weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Samsung Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): February 5, 2017
• Primary Completion (Actual): December 30, 2020
• Study Completion (Actual): February 24, 2021

Study Registration Dates

• First Submitted: February 18, 2016
• First Submitted That Met QC Criteria: February 22, 2016
• First Posted (Estimate): February 23, 2016

Study Record Updates

• Last Update Posted (Actual): June 15, 2022
• Last Update Submitted That Met QC Criteria: June 13, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: 2016-02-052

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes