Safety Study of a Recombinant Protein Vaccine to Treat Esophageal Cancer

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to determine the biological recommended dose of IMF-001.

Detailed Description
-----------------
The prognosis of esophageal cancer is improved with the improvement of surgery, chemotherapy and radiation therapy. However, there are no standard therapies established for recurrent esophageal cancer. NY-ESO-1 antigen is expressed in 33% of patients. NY-ESO-1 protein is applicable without limitation by HLA types, and injected as a complex with cholesteryl pullulan (CHP), forming nano-particles (IMF-001), it can activate both CD4+ and CD8+ T cells. In this phase 1 study, the safety and the biological recommended dose will be determined.

Official Title
-----------------
IMF-001 Phase 1 Study With Refractory Esophageal Tumor

Conditions
-----------------
Esophageal Cancer

Intervention / Treatment
-----------------
* Biological: IMF-001


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Radically unresectable stage III/IV esophageal tumors that have failed the standard treatment (including chemotherapy and radiotherapy), recurrent metastasis after radical surgery and not responding to the standard treatments, or recurrent metastasis after radiotherapy before/after radical surgery. Primary esophageal tumors confirmed by pathological diagnosis Tumor cells expressing NY-ESO-1 antigen (by tissue-immunostaining method or quantitative RT-PCR method) Performance status (PS) of 0, 1 or 2 (ECOG Scale) Life expectancy >/= 4 months No serious disorders with major organs (bone marrow, heart, lung, liver and kidney) and meets the following criteria: WBC count >/= 2.0 x 10 9/L Hemoglobin >/=8.0g/dL Platelet count >/=75 x 10 9/L Serum total bilirubin: </=1.5 x ULN (3 x ULN if with liver mets) AST and ALT: </=2.5 x ULN (5x ULN if with liver mets) Serum creatinine: </=1.5x ULN Agree to use birth control including condoms from the time of obtaining the consent to 6 months after the final administration of the study drug [except females after menopause (1 year or more after the last menstruation and females/males after an operation for sterilization)] Given written informed consent Exclusion Criteria: HIV antibody positive Double cancer History of autoimmune disease History of severe anaphylaxis Active metastatic disease in the central nervous system (CNS) Within 4 weeks after treatment with an anti-tumor agent, systemically administered adrenocorticosteroids, immune suppressants or immune enhancers Pregnant or lactating Any other inadequacy for this study

Ages Eligible for Study
-----------------
Minimum Age: 20 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IMF-001<br>100 or 200 mcg, subcutaneously every 2 weeks. Number of Injections: 6 times. (The treatment may be continued if it is beneficial to the subject). | Biological: IMF-001<br>* 100 or 200 mcg, subcutaneously every 2 weeks. Number of Injections: 6 times. (The treatment may be continued if it is beneficial to the subject).<br>* Other names: CHP-NY-ESO-1;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the maximum tolerated dose, dose-limiting toxicities, type/frequency/degree of adverse events and NY-ESO-1 antigen-specific immune response of IMF-001 alone in patients with esophageal cancer. |  | First 12 weeks (during the first 6 injections) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate clinical activity (tumor response and time to progression). |  | Up to 2 years, or until progression of PS or no positive immune response from IMF-001. |