Clinical trial NCT02079324: Genetically Modified Mesenchymal Stem Cell Theraopeutic Against Head and Neck Cancer
Official title: A Single Center, Open-Label, Accelerated Titration, Dose-Escalating, Phase Ι Study to Evaluate the Safety and Tolerability of IT Injection GX-051, Stem Cell Based Gene Therapeutics in Patients With Very Advanced Head and Neck Cancer
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Head and Neck Cancer
Keywords: head and neck cancer
gene therapy
stem cell therapy
immunotherappy
Lead sponsor: Genexine, Inc.
Sponsor class: INDUSTRY
First posted: 2014-03-05
Enrollment: 12
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: The purpose of this research is to evaluate MTD, Safety and efficacy of GX-051 after intratumoral injection in head and neck cancer patience.

Detailed description: The primary purpose of the study is to determine MTD(Maximum Tolerable Dose) of GX-051 administered in tumor. The second purpose is to evaluate safety and efficacy of GX-051.

Eligibility criteria: Inclusion Criteria:

* Are capable of understanding and complying with the requirements of the study and have signed the informed consent from (ICF).
* Very advanced head and neck cancer aged more than 19.
* Longest distance of the tumor is bigger than 1 cm and capable of Intratumoral injection.
* Baseline ECOG Performance Status 0, 1 or 2.
* Have a life expectancy more than 6 months.

Exclusion Criteria:

* Have no history of prior anticancer treatment.
* Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods.Subject who have participated in a clinical trial of an treatment vaccine or immunotherapy in an year.
* Have a tumor which is near a main vessel that can be occured embolism after injection or which has hypervascularity.
* Patients currently receiving anticancer immuno-therapies Patients who have received prior treatment with an stem cell therapy.
* Have autoimmune disease (multiple sclerosis, systemic lupus erythromatosis, thyroiditis, psoriasis, inflammatory bowel diseases etc.).
* Have Graft rejection reaction such as GVHD.
* Have immunodeficiency disease.
* Leukocytes\< 3.0 x109/L.
* Absolute neutrophil count \< 1.5x109/L.
* Platelet count \< 100 x 109/L.
* Have known positive test for hepatitis B surface antigen, hepatitis C antibody, and human immunodeficiency virus (HIV).
* Alanine Aminotransferase (ALT) \> 2.5xUNL.
* Aspartate Aminotransferase (AST)\> 2.5xUNL.
* Total Bilirubin\> UNL.
* Have blood Creatinine\> UNL.
* Known allergy to IL-12 or the excipient(s) of the study medication including fetal bovine serum.
* Second primary cancer Metastatic brain tumor or meningioma.
* Have a tumor near a main artery.
* Uncontrolled hypertension.
* Uncontrolled diabetes uncontrolled (arrhythmia).
* Heart failure (more than NYHA Functional Class II); unstable coronary artery disease; myocardial infarction within 6 months.
* Child-Pugh Class C hepatic impairment.
* Severe renal impairment (creatinine clearance \< 30 ml/min) or on dialysis.
* Have active infection or history of recurrent infection.
* Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study.
* Are considered ineligible by the investigator due to a mental disease or CNS disease.
* Administration of any other tumor therapy, including chemotherapy and radiotherapy within 4 weeks(6 weeks in case of nitrosoureas and mytomycin C) before the beginning of study treatment.
* Patients receiving chronic, systemic treatment with immunosuppressive agent(steroid) or immuno-modulator within 2 weeks prior to screening.
* Have participated in another clinical trial within 30 days prior to dosing.

Primary outcomes:
measure: MTD after GX-051 intratumoral injection
timeFrame: 2 months
Secondary outcomes:
measure: Adverse events after GX-051 intratumoral injection
timeFrame: 2 months
measure: Anti-tumor response by RECIST 1.1 on computed tomography
timeFrame: 2 months
measure: Changes of INF-γ and IL-12 levels in blood comparing to the baseline after GX-051 intratumoral injection
timeFrame: 2 months
measure: Changes of immune cell distribution in tumor tissue after GX-051 intratumoral injection
timeFrame: 2 months
measure: Evaluation of antibody generation against IL-12 which is active ingredient of GX-051
timeFrame: 2 months
measure: Evaluation of long term safety examined by vital sign, physical examination, clinical laboratory tests, CT etc
timeFrame: 17 monthes
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Seoul St.Mary's Hospital of the Catholic University of Korea
Study officials:
affiliation: Seoul St.Mary's Hospital of the Catholic University of Korea
name: Minsik Kim, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Genexine, Inc.
name: Hyun-Tak Jin, ph.D.
role: STUDY_DIRECTOR
References: