Clinical studies are medical research studies that involve the participation of volunteers to help scientists understand and develop new ways to prevent, detect, or treat diseases and their symptoms. These investigational treatments may be new drugs, a combination of drugs, new ways to deliver an already marketed drug, or a medical device.

Before a new medication is approved, it must undergo a series of lengthy and rigorous tests, first in the laboratory, then in animals, and ultimately in humans. A new medication or medical device must not only be safe and effective, but in many cases it must also demonstrate a superior benefit when compared to similarly approved products.

A clinical study is conducted according to a research plan known as a protocol that has been developed by the pharmaceutical company and reviewed by the FDA along with an Institutional Review Board (IRB) representing the public interest. These agencies monitor the progress of these studies.

The protocol determines:

• The reason for the study
• The number of participants
• The criteria used to determine who is eligible for the study
• A schedule for procedures, tests, drugs and dosages
• The length of the study
• What information will be gathered about the participants
• And much, much more

Clinical studies are led by a principal investigator, who is often a medical doctor, along with a research team made up of doctors, nurses and other health care professionals. All of the participants are volunteers. It is important to conduct research in a variety of people to detect different responses to a single investigational product.

Investigational products such as medications, devices or procedures are conducted in various clinical trial phases to find different kinds of information.

Phase 1 Studies

Phase 1 studies include a small number (20-80) participants, usually healthy volunteers, in some cases patients with advanced disease (e.g. cancer, mental health disorders) and is often the first time tested in humans. The purpose of Phase 1 studies is to:

• evaluate safety
• identify side effects
• determine a safe dose range
• learn how the investigational product is absorbed and handled by the body (pharmacokentics/dynamics)
• Phase 1 studies generally take several months to complete. Approximately 70% of Phase 1 studies continue to Phase 2.

Phase 2 Studies

Phase 2 studies enroll more people than Phase 1 studies (100-300 participants) with the condition under study. In Phase 2 studies researchers further evaluate safety and determine if the investigational product has the intended effect in humans. These studies are sometimes randomized controlled trials where one group of patients receives the investigational product, while another group (the “control group”) receives a standard treatment or placebo. In order to provide unbiased comparative information, these study are often “blinded” which means neither the patients nor the researchers know who is receiving the investigational product.

Phase 2 studies usually take up to two (2) years to complete. About 33% of Phase 2 conducted successfully move on to Phase 3.

Phase 3 Studies

In Phase 3 larger numbers (thousands) of patients with the disease are included to:

• confirm or further evaluate an agent’s effectiveness
• monitor side effects
• compare it to commonly used treatments
• collect other information that will be used to determine whether the agent should be approved and marketed

These studies are usually randomized and blinded trials that generally takes up to several years (1-4 years) to complete. 25 to 30% of Phase 3 studies are successfully completed and are submitted to the FDA by the pharmaceutical company for approval to market the investigational product in the United States.

Phase 4 Studies

Phase 4 studies often called Post-Marketing Surveillance Trials are conducted after the investigational product has been granted approval from the FDA for consumer sale. These studies involve people in various populations and are generally intended to collect additional information after the investigational product is approved and marketed regarding its risks, benefits and use in various populations over a longer period of time. Results from Phase 4 studies can influence whether an investigational product should be taken off the market or if restrictions should be applied to the use of the investigational product.

By taking part in a clinical trial, you have an opportunity to try a new treatment that may or may not be better than those that already exist.

As a study participant you can also help others better understand how the treatment works in people of different races and genders because different people may respond differently to the investigational product. Regulatory agencies such as the FDA seeks to ensure that people of different ages, races, ethnicities, and genders are included in clinical trials.

Some reasons to participate in a clinical trial:

• Access to new treatments not available on the market.
• Access to free medication.
• Compensation for participating (in some trials).
• Satisfaction in helping advance medical research.
• Your participation is voluntary which means you can leave the study at any time.

Each clinical research study has specific eligibility criteria which a participant must meet in order to be enrolled in the study. Therefore, not everyone who applies for a clinical research study will be accepted.

There are many different types of clinical research studies, including:

• Prevention studies that look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
• Studies that test new treatments, new combinations of drugs, or new approaches for therapy.
• Diagnostic studies are conducted to find better tests or procedures for diagnosing a particular disease or condition. Screening studies test the best way to detect certain diseases or health conditions.
• Quality of Life studies (or Supportive Care studies) explore and measure ways to improve comfort and the quality of life for individuals with a chronic illness.

Usually, clinical studies compare a new product or therapy to something else to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product, or an existing, approved therapy.

In some studies, participants may be assigned to receive a placebo (a product with no therapeutic action that looks or acts like the test product). Comparison with a placebo can be the fastest and surest way to demonstrate therapeutic effectiveness of new products.

Potential participants are told before they enter a trial whether placebos are going to be used in the study and the risks and benefits of the study medication(s).

A clinical research study is conducted according to a research plan known as a study protocol which is designed to answer specific research questions and to assure the safety of the participants. The protocol includes the following information:

• The reason for conducting the study
• Who may participate in the study (inclusion/exclusion criteria)
• The number participants needed
• The schedule of tests, procedures, investigational product and their dosages
• The length of the study
• What information will be collected about the participants.

The following process generally occurs during the conduct of a clinical research study:

Pre-Screening

The clinical research group will review their existing patient databases or medical charts to identify potential patients who may be eligible to participate in the clinical research study. They may also place advertisements on the internet, newspaper, radio, or television to recruit interested participants. They may also conduct seminars, free health screenings, or forums.

Potential participants from these pre-screening efforts are contacted and briefly interviewed to confirm if they are potentially eligible to participate in the clinical research study.

Informed Consent

If you are potentially eligible to participate in a clinical research study, an appointment will be scheduled for you to come to the clinical research site and meet with one of the research staff members. Before any study related procedures are performed you will be required to sign an informed consent form which details the study design, risks and benefits, your rights as a study participant and who to contact in case of an emergency. During the informed consent process, you will have an opportunity to ask questions you may have regarding the study and your participation. You should receive a copy of the signed informed consent form for your records and future reference.

Screening Visit

Once you agree to participate in the clinical research study and have signed the informed consent form, the study screening procedures will be administered. The purpose of the screening visit is to determine if you meet the specific inclusion and exclusion criteria for the specific clinical research study you agreed to participate in. Generally, during the screening visit you may be asked to answer questions regarding your medical history, medications and other treatments you are taking, and complete questionnaires. A member of the research team may also assess your general health by performing a physical examination, EKG and collect blood/urine samples.

Generally, the investigational product is not dispensed during the screening visit.

Study Visits (Treatment Visits)

If you meet the specific inclusion and exclusion criteria (which includes a study acceptable laboratory and medical results) for the study you will be asked to return to the clinic for a series of study visits sometimes referred to as Treatment Visits. It is generally during these study visits that a study participant will be receive the investigational product or the comparator product (which sometimes can be a placebo). The number of study visits and the interval in which the visits occur varies from study to study.

End of Study Visit

Once you have reached the end of the treatment visit, an End of Study Visit is conducted. Generally, the same or similar assessments conducted prior to you receiving the investigational product is repeated. The research team will also discuss you follow-up treatment options (which may include receive standard of care treatments for your illness) and if required by the study you may be asked to return to the research site for a follow-up safety visit.

Follow-up Safety Visit

The number of follow-up safety visits and the interval in which the visits occur varies from study to study. The purpose of this visit is to assure you are not experiencing any lingering side-effects from the investigational product or from the overall participation in the study.

Risks are involved in clinical research, as in routine medical care and activities of daily living. Most clinical research studies pose side effects that are temporary and go away when the treatment is stopped. However, some research subjects experience side effects that can be permanent or require medical attention. Some side effects appear during treatment, while others may not show up until after the treatment is over.

Some investigational products that are being tested have side effects that can be unpleasant, serious or even life-threatening. Because the investigational products being studied are new, researchers don’t always know what the side effects will be in humans.

The specific risks associated with any research protocol are described in detail in the informed consent document, which you are asked to sign before taking part in research. In addition, the major risks of participating in a study will be explained to you by a member of the research team, who will answer your questions about the study. Before deciding to participate, you should carefully weigh these risks. Although you may not receive any direct benefit as a result of participating in a clinical research study, the information collected by your participation may help others.

To help you decide if you want to be in a study, the FDA requires that study participants are given complete information about the study before they agree to take part.

Informed consent forms should be written so the participant can easily understand it and should include:

• purpose of the research
• how long the study will take
• what will happen in the study and which parts of the study are experimental
• possible risks or discomforts
• possible benefits
• other procedures or treatments that the participant might want to consider instead of the treatment being studied
• that FDA may look at study records, but the records will be kept secret
• whether any medical treatments are available if the participant is hurt, what those treatments are, where they can be found, and who will pay for the treatment
• the person to contact with questions about the study, participant rights, or if the participant can get hurt
• the participant can quit at any time

If you don’t understand the information included in the informed consent form, be sure to ask the doctor or other research staff member to explain it. This discussion should take place in private, and you must be given enough time to make an informed decision. In most studies, you should be allowed to take the consent form home for further discussion with others, such as family, caregivers and primary care physicians. Make sure you understand all of it before you agree to be in the study.

Before you can be in the study, you must sign the informed consent form, showing that you have been given this information and understand it. The informed consent form is NOT a contract and you can leave the study at any time, for any reason. You may ask questions at any time throughout the study.

In addition, you must also be informed of any new information learned during the study that may affect your willingness to continue to participate in the study.

Here are some questions to ask your doctor to help you decide if you want to take part in a clinical study:

• What is the study trying to find out?
• What kinds of test and exams will I have to take while I’m in the study? How much time do these take? What is involved in each test?
• How often does the study require me to go to the doctor or clinic?
• Will I be hospitalized? If so, how often and for how long?
• What are the costs to me? Will my health insurance pay for it?
• What follow-up will there be?
• What will happen at the end of the study?
• What are my other treatment choices? How do they compare with the treatment being studied?
• What side effects can I expect from the treatment being tested? How do they compare with side effects of standard treatment?
• How long will the study last?

The study protocol outlines who can participate in a clinical research study referred to as the Inclusion/Exclusion Criteria. The criteria that allows a person to participate in a clinical research study are called inclusion criteria; and the criteria that disqualifies a potential research subject are called exclusion criteria. These criteria are based on factors such as age, gender, the type and stage of a disease, previous/current treatment history, and other medical conditions. A research subject is a person who meets the inclusion/exclusion criteria and agrees to take part in a clinical research study. A research subject may be either a healthy individual or an individual with a specific disease or condition.

Every clinical research study is led by a Principal Investigator who is often a licensed medical doctor, and a clinical research team that may include doctors, nurses, social workers, and other health care professionals. In general, the clinical research team roles and responsibilities will include:

• Principal Investigator (PI): A person responsible for the ethical conduct of the research study. This includes protecting human subjects’ rights, safety and welfare, protocol (study) compliance, and adherence to institutional, state and federal regulations and guidance. The PI is responsible for ensuring informed consent is appropriately obtained from each participant and for appropriately maintaining study records. The PI is also responsible for complying with the financial and administrative policies and regulations associated with the award, overall fiscal management of the project, and conflict of interest disclosure.The PI oversees all aspects of a clinical study from protocol design, recruitment, data collection, analysis and interpretation of results, but some tasks can be delegated to other research team members (Co-Investigators and Key Personnel). The PI is responsible for ensuring that all research team members have appropriate education, training and qualifications to assume delegated study tests. All study team members are responsible for ensuring that the conduct of the study is compliant with institutional, state, federal and industry guidance and regulations.
• Sub-Investigator: The Sub-Investigator may perform all or some of the PI functions, but they do not accept primary responsibility for the research study. The sub-investigator is under the supervision of the PI and is responsible for performing study–related procedures and /or to make important study-related decisions in compliance with the ethical conduct of the study.
• Research Coordinator: The Research Coordinator oversees and coordinates the daily activities of clinical research studies. They work closely with the clinical teams and investigators to ensure that all protocol required procedures and visits occur according to protocol specified guidelines. Research Coordinators generally manage participant enrollment and ensure compliance with the protocol and other applicable regulations. This includes but is not limited to: participant recruitment, obtaining informed consent, educating participants on the details of the research study, assessing participant eligibility, facilitating participant care and follow-up per protocol, creating source documentation, assisting in the assessment of toxicities/adverse events and reporting serious adverse events per IRB and sponsor requirements. They are generally the primary contact person for the research subject.

The clinical research team will make every effort to assure that your Personal Health Information (PHI) is kept extremely confidential. Your information will not be shared without your permission or except required by law.

If you choose to submit your information to use through this website, with your permission, this information will be entered into our clinical research database for both current and future study opportunities. You can ask to have your information removed at any time. If you choose to call in and speak to one of our research specialists over the phone, your information will be captured in the same electronic database. Again, this is voluntary and can be removed at any time.

In most clinical research studies all visits, tests, and procedures related to the study are free of charge. If you qualify for one of our research studies, you may be compensated for your time and travel. The amount of compensation (as well as any expenses not covered by the study) will be reviewed during the informed consent process.

The number of study visits, duration and frequency will vary depending on the study design. Typically, the study visits are at least 30 minutes, however some may be a few hours. The initial visit (screening visit) will generally be longer as it will include the informed consent process, an assessment of your medical history, collection of laboratory samples, and various study related questionnaires. During the informed consent process, participants will be provided detailed information regarding the study visits and overall duration of the study.