Clinical trials 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 show to be safe and effective.

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 Ethics Committee representing the public interest. These agencies monitor the progress of these studies.

The protocol determines:

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

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

Phase 1 Studies

Phase 1 studies include a small number (20-80) of participants, usually healthy volunteers, in some cases participants with advanced disease (e.g., cancer, mental health disorders) and are 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 (pharmacokinetics/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 participants receives the investigational product, while another group (the “control group”) receives a standard treatment or placebo. In order to provide unbiased comparative information, these studies are often “blinded” which means neither the participants 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 studies conducted successfully move on to Phase 3.

Phase 3 Studies

In Phase 3 larger numbers (can be up to thousands) of participants for most diseases or between 200-300 for rare and orphan diseases are included to:

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

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

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.

By taking part in a clinical trial, you may have an opportunity to try an investigational 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 study 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 seek to ensure that people of different ages, races, ethnicities, and genders are included in clinical trials.

Some reasons to participate in a clinical trial:

• Potential access to investigational treatments not available on the market.
• Access to no-cost study medication.
• 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 investigational treatments, new combinations of study drugs, or new approaches for study 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 trials compare an investigational 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 compare the therapeutic effectiveness of investigational 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 of 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

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

Participant Information and Consent Form (PICF)

Screening Visit

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

Study Visits (Treatment Visits)

End of Study Visit

Follow-up Safety Visit

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 study treatment is stopped. However, some research subjects experience side effects that can be permanent or require medical attention. Some side effects appear during study treatment, while others may not show up until after the study 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 PICF 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, study participants are given complete information about the study before they agree to take part.

PICFs 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 PICF, 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 PICF, showing that you have been given this information and understand it. The PICF 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 trial:

• 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 investigational treatment being studied?
• What side effects can I expect from the investigational treatment being tested? How do they compare with side effects of standard treatment?
• How long will the study last?

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 the PICF 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 trial from recruitment, data collection, analysis and interpretation of results, but some tasks can be delegated to other research team members (Sub-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 or Study Coordinator: The Research or Study 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/Study 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 PICF, 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 confidential. Your information will not be shared, except as required by applicable law or as otherwise described in the consent form.

If you choose to submit your information 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 at no cost. If you qualify for one of our research studies, you may be reimbursed for travel associated expenses. The amount of compensation (as well as any expenses not covered by the study) will be reviewed during the PICF process.