Clinical trial NCT02688881: Study to Evaluate the Safety and Efficacy of Sirolimus, in Subject With Refractory Solid Tumors
Official title: Study to Evaluate the Safety and Efficacy of Sirolimus, in Subject With Refractory Solid Tumors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Refractory Solid Tumors
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
First posted: 2016-02-23
Enrollment: 6
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Progression Free survival
timeFrame: 6 Weeks
Secondary outcomes:
measure: Overall Response Rate
timeFrame: 24 months
measure: Time to progression
timeFrame: 24 months
measure: Overall survival
timeFrame: 24 months
measure: Number of subjects with Adverse Events as a measure of safety
timeFrame: 24 months
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
References: