HER3 DNA Vaccine Phase I Trial FAQ

What is the purpose of this study?

The purpose of this study is to study the effects that an investigational cancer vaccine has on you, your immune system, and your cancer. The cancer vaccine is called pING-hHER3FL. Once it is taken up by your cells, it will make extra copies of the HER3 protein. The immune cells in your body will then attack cancer cells that express HER3.

How does this study work? What do patients receive?

You will receive the study vaccine every 4 weeks for a total of 3 vaccinations and will return 4 weeks after the third vaccination and then every 3 months for up to one year for follow-up. At each vaccination visit, you will receive 4 injections of the HHER3 Plasmid Vaccine into the skin of your thigh. You will also be followed for up to 5 years after the immunizations for evidence of recurrence of disease. If you have recurrence of your cancer, you will have a tumor tissue biopsy as part of your clinical care, and we will take a sample for research.

Who will be my doctor on this study?

If you decide to participate, Dr. Michael Morse, Dr. H. Kim Lyerly, or one of the other Duke Oncologists will be your doctor for the study and will be in contact with your regular health care provider throughout the time that you are in the study and afterwards, if needed.

Why is this study being done?

The purpose of this study is to study the effects that a cancer vaccine has on you, your immune system, and your cancer. The cancer vaccine is called pING-hHER3FL. HER3 is a protein. Proteins, which are made from a gene or gene product, are the building blocks of your body, cells, and organs. A gene contains information that determines in part the traits, such as eye color, height, or disease risk, that may be passed on from parent to child. The vaccine in this study is made from a circular strand of DNA called a plasmid that contains the HER3 gene. The HER3 protein is found on some cancer cells. When this plasmid is given to you as a vaccine, it can enter some cells in your body. Once it is taken up by your cells, the plasmid will cause cells to make extra copies of the HER3 protein. The cells will then chop up the HER3 protein into small pieces that can be displayed on the cell surface. By doing this, the cell is showing pieces of HER3 to the immune cells in your body to tell them to attack cancer cells that express HER3.
pING-hHER3FL will be administered using a PharmaJet Tropis Needle-Free Injection system. This system will deliver pING-hHER3FL by means of a high velocity fluid jet into your skin and may help your immune response to pING-hHER3FL compared to a needle injection.

pING-hHER3FL is an investigational drug. The word “investigational” means pING-hHER3 is still being tested in research studies and has not been approved by the U.S. Food and Drug Administration (FDA). The PharmaJet Tropis Needle-Free Injection system has previously been used in multiple human clinical trials but is not yet approved by the FDA.

This study will be the first time that the pING-hHER3FL vaccine is used in humans. The goal of the study is to determine whether pING-hHER3FL triggers your immune system to fight the cancer, to see if it is safe, and to see what side effects it may cause.

How many people will take part in this study?

Up to 18 people will take part in this study all at Duke.

What is involved in this study?

Screening
The screening tests and procedures will be performed prior to the first drug regimen visit and may take place over multiple visits from 1-28 days prior to study regimen administration. Some of these tests and procedures are part of routine cancer care, and may be done even if you do not join the study. After your screening tests are completed, your study doctor will check the results of your screening tests to see if you can be enrolled in the study. If you are qualified and choose to participate, you will need to return to Duke within 28 days to receive the study vaccine.

 You will be asked about your medical history
 You will have a blood pregnancy test (only for women who could possibly become pregnant). The test must be done within 7 days of the first vaccine administration, and must be negative in order for you to participate in the study.
 You will have a physical exam
 Your vital signs will be measured
 You will be asked questions about how you are feeling and how well you are doing
 You will have a blood sample collected for routine health monitoring
 You will have a blood sample collected for immunological research blood tests
 Tumor Tissue will be obtained from your clinical biopsy

Week 0, 4 and 8 Visit

 You will have a physical exam
 You will have the HER3 Plasmid Vaccine administered
 Your vital signs will be measured every 15 minutes for 1 hour after injection
 You will be asked questions about how you are feeling and how well you are doing
 You will be asked about what medications you are taking
 You will be asked if you are having any side effects
 You will have a blood sample collected for routine health monitoring

12 Week Follow-up Visit

 You will have a physical exam
 Your vital signs will be measured
 You will be asked questions about how you are feeling and how well you are doing
 You will be asked about what medications you are taking
 You will be asked if you are having any side effects
 You will have a blood sample collected for routine health monitoring

 You will have a blood sample collected for immunological research blood tests
 You will have a blood sample collected for immunological research blood tests

6, 9 and 12 month Follow-up Visit

 You will have a physical exam
 You will be asked questions about how you are feeling and how well you are doing
 You will have a blood sample collected for immunological research blood tests

Yearly Follow-up Visit

If you have a Recurrence

End of Study Visit

How long will I be in this study?

Your direct participation in this study will last about one and a half years. We may contact you by phone, email or in person (if you come for a clinic visit) up to five years after your last dose of the study vaccine, in order to obtain information about the long-term safety.
You can choose to stop participating at any time without penalty or loss of any benefits to which you are entitled. However, if you decide to stop participating in the study, we encourage you to talk to your doctor first.
Clinically relevant results of this research will be communicated with you when the results are published.

Can I continue to take my medications?

To participate in this study you may need to stop or change some of your current medications because they may affect how well the study drugs work. However, this is unlikely. You will be notified if any of your current medications need to be stopped or changed to allow you to participate in this study. If there are any changes, your doctor will let you know of any risks that may be associated with changes in your current medications. If you participate in this study, there may also be limitations to the other medications and supplements or foods that you can take while you are taking the study drug. Changing or limiting the medications you take for other conditions may be associated with additional inconvenience, costs, and/or side effects.

What are the risks of the study?

his is the first time pING-hHER3 will be given to humans. While participating in this study, you are at risk for side effects from the investigational plasmid vaccine. You should discuss these with your doctor. In addition to the side effects listed below, there may be other side effects that the researchers cannot predict. Other drugs may be given to make side effects less serious and uncomfortable. Many side effects go away shortly after the medications are discontinued but, in some cases, it is possible that side effects may be serious, long-lasting and permanent or may result in death.
The plasmid vaccine is designed so that it does not make any long-lasting changes to your cells or your DNA, and should only be in your cells for a short time. Although a plasmid containing a gene may in rare cases cause a disease or a new cancer, this possibility is very small.
The known side effects and possible risks of this research study are discussed below:
Study Vaccine Injections
Minor side effects of pING-hHER could include:

 Fever
 Chills
 Headache
 Injection site reactions

Major side effects of pING-hHER could include:

 Allergic reactions with swelling in such places as your throat or lungs which could lead to shortness of breath, breathing failure or death.

 Your immune system may be stimulated to attack your own body (called autoimmunity) leading to a low white blood cell count that result in an infection, skin rash, joint swelling, intestinal inflammation (chronic colitis), or fluid around the heart and lungs.
 Liver toxicity

What benefits are there to being in this study?

If you agree to take part in this study, there may be direct medical benefit to you. If the study drug works for you, you may live longer or have better control of your cancer, but this cannot be guaranteed. We hope that in the future the information learned from this study will benefit other people with your condition.

Will my information be kept confidential?

Participation in research involves some loss of privacy. We will do our best to make sure that information about you is kept confidential, but we cannot guarantee total confidentiality. Your personal information may be viewed by individuals involved in this research and may be seen by people including those collaborating, funding, and regulating the study. We will share only the minimum necessary information in order to conduct the research. Your personal information may also be given out if required by law.
In addition, your records may be reviewed in order to meet federal or state regulations. Reviewers may include representatives from the US Department of Defense (DOD), Food and Drug Administration (FDA), representatives of the National Cancer Institute, Duke Cancer Institute, Duke Office of Audit, Risk and Compliance, and the Duke University Health System Institutional Review Board. If any of these groups review your research record, they may also need to review your entire medical record.

Because this study involves a DNA plasmid containing a gene fragment, safety information must be reported to the Recombinant DNA Advisory Committee (RAC) of the National Institutes of Health. This information is available to the public. However, no information by which you can be identified will be reported with the safety information.
As part of this study, you will be asked to have certain tests and/or procedures. Some of tests and/or procedures would have been done as part of your regular care. The study doctor will use these test results both to treat you and to complete this research. These test results will be recorded in your medical record and will be reported to the research data office at Duke. Results of tests and studies done solely for this research study and not as part of your regular care will not be included in your medical record. The results of the immune monitoring tests will be performed only for research and the results of the immune monitoring will not be placed in your medical chart, only that blood was drawn.
The study results will be retained in your research record for 15 years after the study is completed. At that time either the research information not already in your medical record may be destroyed or information identifying you will be removed from such study results at DUHS. Any research information in your medical record will be kept indefinitely.

Will participating in this study cost me anything?

No. Duke will provide the study vaccine free of charge to you. You or your insurance provider will be responsible and billed for all costs related to your routine medical care, including copayments and deductibles. Routine medical care services are those that you would have received for your condition if you were not participating in this research study. Not all services are covered by insurance. Some procedures or scans may require pre-authorization by your insurance plan. We will notify you if we learn that a service is not covered by your insurance plan as part of the pre-authorization process. If it is not covered, you will be responsible for paying for it. The amount of your out-of-pocket expense will depend on your insurance plan. For beneficiaries with Medicare Advantage Plans, traditional Medicare is billed for the routine cost of a research study. You may have more or higher co-pays than with a Medicare Advantage plan. Please discuss the costs of the study with Dr. Morse. At your request, a Financial Counselor in the clinic may provide you with an estimate of costs for routine services.

The study sponsor, Duke, has agreed to pay for services and procedures that are done solely for research purposes. Please talk with the PI/study team about the specific services and procedures that the sponsor will pay for, and the ones for which you or your insurance will be responsible.

We will monitor your DUHS patient care charges to make sure that costs are directed appropriately. If you have any questions or concerns about appropriate billing, contact your study team coordinator so that he/she can help find a resolution.

Your study doctor may request that you return for a checkup before you stop the vaccine if he/she thinks that stopping it suddenly may harm you and may ask you to complete the tests that would ordinarily occur when a person completes the study.