A Retrospective and Prospective Study of a New Immunotherapy Regimen (MyVaccx) for Patients With Adenocarcinoma.
Conditions: Adenocarcinoma
Sponsor: Gary Onik MD

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
1 
 
CONFIDENTIAL 
 
 
 
 
NIRPA 
 
An Investigator Sponsored Retrospective/Prospective Study 
of a New Immunotherapy Regimen (MyVaccxTM) for 
Patients  with Adenocarcinoma 
 
Protocol Number:  NIRPA #1 
March 20, 2018 
 
 
 
 
Study Sponsor: 
Gary Onik, MD 
Medical Director - The Center for Recurrent Prostate Cancer 
Adjunct Professor - Department of Mechanical Engineering  
Carnegie Mellon University 
12 Southeast 7th Street, 6th Floor 
Ft Lauderdale FL 33301 
 
Phone: 321-297-4489 
Fax: 407-289-4069 
 
 

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
2 
 
CONFIDENTIAL 
Through the practice of medicine, using FDA cleared or approved devices, methods and 
medication in a unique off-label manner, Dr. Gary Onik has treated twenty-four patients with the 
MyVaccxTM System for Adenocarcinoma in the following types of cancer: 
• 
Prostate cancer (19 patients) 
• 
Lung cancer (1 patient) 
• 
Pancreatic cancer (1 patient) 
• 
Colorectal cancer (1 patient) 
• 
Bladder cancer (1 patient) 
• 
Unknown primary cancer (1 patient) 
This is a retrospective/prospective study to document the pertinent patient medical history, 
treatment performed and outcome using standardized Case Report Forms (CRF’s) for those 
patients who meet the selection criteria. The information generated by the data included in the 
First Treatment and Second Treatment CRF’s will be summarized in a White Paper authored by 
Dr. Gary Onik.   
The patients who met the subject selection criteria will be prospectively monitored through their 
normal standard of care for their original cancer, which will be documented on the Follow-up 
CRF.  Individual Follow-up CRFs will be generated for each follow-up event.  The data 
collected will be used to update the White Paper on a periodic basis.   
The subjects will not incur any additional costs above their routine follow-up for their original 
cancer and will not receive any additional experimental procedures.  
BACKGROUND 
MyVaccx™ cancer therapy enables the patient’s immune system to fight off cancer by 
amplifying the abscopal effect.   The Abscopal Effect is a phenomenon in the treatment of 
metastatic cancer where localized treatment of a tumor causes not only a shrinking of the treated 
tumor, but also a shrinking of tumors outside the scope of the localized treatment.  R.H. Mole 
proposed the term “Abscopal” in 1953 to refer to effect of ionizing radiation “at a distance from 
the irradiated volume but within the same organism.”  
The MyVaccx Cancer Therapy is initiated by: Proprietary focal tumor “priming” with antigen 
presentation (medical device system) immediately followed by the intra-tumoral injection of a 
proprietary depot formulation containing multiple FDA approved checkpoint inhibitors and an 
FDA approved cytokine, fostering the creation of an autologous vaccine that impacts the cancer 
systemically.  The patient is then receives daily systemic injections for two months of the 
cytokine to boost the immune system’s ability to ramp up to address the large metastatic tumor 
burden.  My Vaccx consists of up to two applications of the therapy, the initial and a 

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
3 
 
CONFIDENTIAL 
second  “booster” that are 6-8 weeks apart (if required and the subject has agreed to a second 
“booster”).   
The rationale for treating patients with the MyVaccx system is because the patients have run through and 
out of alternative therapy options, so My Vaccx provides a new therapy option for very late stage disease 
where none currently exists.  
The active drugs used in the depot formulation are a PD-1 (either OpDivo, or Keytruda) and CTLA-4 
(Yervoy) checkpoint inhibitors, plus a sagramostim (Leukine), a GM-CSF cytokine.  The checkpoint 
inhibitors are used to mitigate the tumor’s ability to locally shut down the immune system, even after the 
tumor has been primed with antigens having been presented, and the GM-CSF cytokine is utilized to 
“ramp up” the local immune response to the tumor antigens once they have been recognized.  The depot 
formulation also includes iodinated omnipaque contrast media, and ultrasound contrast media (perflutren 
– Definity) to aid in insuring that the intra-tumoral injection of the depot is optimally positioned.  Post 
procedural medication includes Leukine daily for two months, which aids the immune system to handle 
the tumor burden on a systemic level.    
Animal Study 
There has been one (1) animal study conducted using the ablation modality which is part of the 
MyVaccx procedure.  This study was published in the Journal of Clinical & Experimental 
Pathology, the abstract follows.1 
Abstract:  Electrical membrane breakdown (EMB) is a novel form of non-thermal treatment that 
has not, to our knowledge, been previously evaluated for its potential utility as an ablation 
mechanism. The findings with EMB immediately after treatment were compared with other 
forms of ablation (cryoablation and IRE (irreversible electroporation)) in the porcine liver 
clinically, ultrasonographically, and by light microscopy and ultrastructural analysis. Clinically, 
EMB did not induce muscular contractions, in contrast with IRE. By ultrasound, all lesions were 
hypoechoic when compared to the untreated liver. EMB created a consistent pattern of 
immediate tissue destruction at the light microscopic and ultrastructural level, characterized by 
pulse-dose-related coagulative necrosis and nuclear pyknosis, preserved blood vessels and 
adjacent structures, and sharply demarcated margins. We conclude that the profile of EMB 
ablation is distinctive and unique, inducing necrosis by immediate electrical rupture of cell 
membranes non-thermally while preserving proteins and adjacent vessels with potentially 
superior stimulation of the immune system than other ablation modalities 
OBJECTIVES 
The purpose of the proposed research study is to (1) document the findings of the patients which 
were treated with the MyVaccxTM System who meet the subject selection criteria and (2) 
prospectively monitor the treatment and longer-term outcome for these patients.  The 

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
4 
 
CONFIDENTIAL 
information obtained from this research study will be used in the development process for a 
commercially viable MyVaccxTM System.   
SUBJECT SELECTION  
Subjects are included if they were treated with the MyVaccxTM System and are greater than 18 
years of age. Two subjects with known visceral disease are being excluded based on the standard 
of other immunologic prostate cancer studies.  Therefore, this study will include a maximum of 
twenty-two patients (total number to be defined based on subjects who sign the Informed 
Consent) out of the twenty-four treated with the MyVaccxTM System. 
 
STUDY PROCEDURES 
Informed consent from either the subject or their legal guardian will occur within approximately 
a one (1) month time-frame after receiving IRB approval to conduct the study.  The Clinical 
research staff who have been trained on Good Clinical Practices by a CITI certified Clinical 
Research Associate will obtain signature of the informed consent of the subject or the subject's 
legally authorized representative by facsimile or e-mail.  The consent interview will be 
conducted by telephone when the subject or subject's legally authorized representative has read 
the consent form during the discussion. After the consent discussion, the subject or the subject's 
legally authorized representative will sign and date the consent form and return the document by 
facsimile scanning and returning it through a secure e-mail account, or by posting it to a secure 
internet address. Alternatively, the subject may bring the signed and dated consent form to 
his/her next visit to the clinical site or mail it to the clinical investigator. The signed document 
will be filed with the subject's case history. In addition, the person signing the consent form will 
receive a copy of the consent form. The study staff will sign the consent after it has been 
returned to the site and a copy of the signed consent by subject or legal authorized representative 
(LAR) and staff will be sent back to the subject or LAR via email (pdf version). Data collection 
will not begin until the consent has been signed by both the subject or LAR and site staff.  
For the retrospective portion of the clinical study, the data will be obtained from the subject’s 
medical records and recorded on the appropriate CRF.  Data will be collected from the first 
patient treated (06/03/2015) to the last patient treated (09/20/2017) with the MyVaccxTM  therapy. 
For the prospective portion of the clinical study, information will be obtained from the subjects’ 
records as they receive standard of care according to the physician judgement for this patient 
population (Adenocarcinoma) and recorded on the Follow-up CRF.  For the retrospective portion 
of the clinical study, the data will be collected from the medical records and will continue to be 
reviewed for the life of the subject.  
 

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
5 
 
CONFIDENTIAL 
 
RISK/SAFETY INFORMATION  
There are non-physical risks associated with taking part in this study, such as the risk of 
accidental disclosure of your personally identifiable medical information. 
 
MONITORING/REPORTING OF AE/SAE 
The study will be monitored by Syntactx, LLC a Contract Research Organization, the following 
is their address and contact information. 
Address: 
Syntactx, LLC 
4 World Trade Center 
150 Greenwich Street, 44th Floor 
New York, New York 10007 
 
Contact Information: 
p 212-228-9000 
f 646-375-3183 
 
STUDY OVERSIGHT 
The study may be prematurely terminated by Dr. Gary Onik if the research regarding the 
MyVaccxTM is terminated.  The study will be made available for monitoring, auditing, IRB 
review and regulatory inspection by providing direct access to study related source data.  
 
DATA MANAGEMENT 
The data from the approved Case Report Forms will be entered into a database (Apples 
Numbers) by the Clinical Research Staff.  The database is password protected and has change 
traceability.  All subjects will be given a unique identifier and all information and data 
concerning subjects will be considered confidential. The study results will be interpreted and 
summarized in a white paper by Dr. Gary Onik.  All data used in the analysis and reporting of 
the study will be without identifiable reference to the subject. 
 

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
6 
 
CONFIDENTIAL 
 
 
IRB REVIEW/ETHICS/INFORMED CONSENT 
The protocol, informed consent document and relevant supporting information will be submitted to the 
Schulman IRB for review and must be approved before the study is initiated. In addition, any subject 
recruitment materials will be approved by the Schulman IRB prior to being used.  
 
This study will be conducted in accordance with the ethical principles that have their origin in 
the Declaration of Helsinki and that are consistent with Good Clinical Practice and applicable 
regulatory requirements.  
 
The Sponsor must submit any change to the protocol to the Schulman IRB for review and 
approval before implementation. A protocol change intended to eliminate an apparent immediate 
hazard to subjects may be implemented immediately provided the Schulman IRB are notified 
within 10 working days.  
 
It is the responsibility of the investigator (Dr. Gary Onik) to ensure each subject has full and 
adequate verbal and written information using the Schulman IRB approved informed consent 
document, including the objective and procedures of the study and the possible risks involved 
before inclusion in the study. Informed consent must be obtained prior to enrollment in the study. 
A copy of the signed informed consent must be given to the study subject. 
Inclusion of subjects with legally authorized represntatives (LAR) who will sign the informed 
consent are being included to maintain data integrity to fulfill study objectives.  The informed 
consent will not be conducted on subjects with cognitive impairment, only those subjects who 
are deceased will have the informed consent signed by the LAR.  
CONFIDENTIALITY 
Information about study subjects will be kept confidential and managed according to the 
requirements of the Health Insurance Portability and Accountability Act of 1996 (HIPPA).  The 
confidentiality of the subjects will be maintained by: 
1. Limiting access of study-related medical records to the monitor, auditor (if used), the 
Schulman IRB, and/or other regulatory authorities; 
2. Maintaining confidentiality of the study-related records identifying the subject by a 
subject ID # on the CRF’s and, to the extent permitted by applicable laws and/or 
regulations, will not be made publicly available;  

  
Center for High Risk and   
 
Retrospective Case Report Form 
Recurrent Prostate Cancer  
 
Version 1.0 
 
 
 
 
 
 
7 
 
CONFIDENTIAL 
3. Maintaining confidentiality of the subject’s identity if any results of the study are 
published.  
 
INTENDED USE OF THE DATA 
The use of the data collected from this study will be used to write a White Paper in a standard 
abstract format.  The White Paper will assist in writing a subsequent Prospective Study for the 
appropriate subjects with Adenocarcinoma. 
 
 
 
 
1 Onik GM, Bostwick DG, Miessau Esq JA, Webb Z, Friedman MB (2017) Electrical Membrane 
Breakdown (EMB): Preliminary Findings of a New Method of Non-thermal Tissue Ablation. J 
Clin Exp Pathol 7: 319. doi:10.4172/2161- 0681.1000319.