Clinical Trials

Listed below are common questions asked about clinical trials. If you have questions regarding a specific trial, use the contact information provided in the study listing.

What is a clinical trial?

Clinical trials follow laboratory research initiatives that focus on improving public health and decreasing the occurrence of illness in people. They depend heavily on the cooperation of participants and volunteers and can be as simple as allowing yourself to be observed, or as complex as receiving new drugs, therapies, treatments, devices or surgeries.

How do clinical trials work?

The way a clinical trial is run depends largely on the type of study being performed. Depending on the study design, participants may have to meet with the researcher for tests and office visits while others may be asked to fill out questionnaires. Before participating in a study, the research team will review the protocol for the trial which clearly outlines the requirements and qualifications for participating.

What are the phases of a clinical trial?

Clinical trials are conducted in phases to ensure that the data collected accurately reflects the findings of the trial.

Phase I trials are those that are conducted in a small group of about 20 — 80 people. In this phase, researchers are evaluating the safety and setting standards for how they will administer and apply their research findings to the participants.

Phase II trials involve a much larger group (100-300) and exist to test not only the safety, but also the efficacy.

Phase III trials expand to an even larger group (1,000 — 3,000) and are used as a measure of confirmation and as a way to compare the new treatment with the one currently accepted as the standard of care. If the proposed therapy or treatment continues to prove to be both safe and effective, it progresses to the final phase.

Phase IV of clinical trials aims to extract additional information from the previous phases and move the experimental treatment into standard practice. Here researchers further evaluate the risks and benefits as well as outline the manner in which the therapy should be marketed and used.

Who sponsors clinical trials?

Clinical trials can be sponsored by a multitude of organizations, individuals such as doctors or foundations, or they can be funded federally by a federal agency such as the National Institutes of Health (NIH).

Does anyone monitor the research while it is happening?

Clinical trials are monitored by Institutional Review Boards also known as IRBs. The IRB is put in place to review the initial study plan, protocol, and ensure that the trial is being conducted in a safe and effective manner. Once approved, the IRB continues to monitor the trial at least once a year. If a researcher needs to adapt the protocol in anyway, it must be approved by the IRB and then relayed to study participants. The IRB also makes sure that participants are fully informed, treated fairly and that their privacy and confidentiality is protected at all times.

What is informed consent?

The purpose of informed consent is to outline the details of the trial to participants and protect their rights. Informed consent is issued in a document that describes in detail the goals of the study, possible risks or benefits associated with participating, the purpose, the requirements for participating, key information including the names and contact information for the researchers in charge, and the projected duration. If a participant does not speak English, the informed consent document will be translated for them.

Who is a part of the clinical trial team?

Clinical trial teams can include doctors, researchers, nurses, social workers and other health care professionals. The role of the trial team is to monitor the status of the participants' health and record the results of the study. It is also their responsibility to ensure the comfort and safety of the participant.

What is a control group?

In clinical trials, the control group is a group of people who receive the current standard of care, rather than the experimental one being tested. For example: if the clinical trial is testing a drug to better control blood pressure, the control group would be given a drug that is the current medication prescribed by doctors.

Control groups are just as important as those receiving the experimental treatment or therapy because the results of the other group are measured against the control group. Having these two groups allows researchers to compare their results and determine how much more or less effective their research findings are.

What is a placebo?

A placebo is a pill, liquid or powder that is given to participants, but has an inactive formula. The purpose of the placebo is to form a control group that believes that they are receiving a treatment to measure the full effectiveness of the experimental drug. These studies are called single-blind trials since the participants are unaware that the medication they are receiving is not treating them. There are also double-blind trials in which neither the participant nor the researcher knows who is receiving the experimental treatment. This helps prevent researchers' bias or beliefs from skewing the results.

What happens when the study ends?

A clinical trial usually ends for a participant when they have completed everything outlined in the protocol or if they decide to leave the study. Researchers may require additional contact with participants after the study has ended, but this should be outlined in the protocol and explained before signing the informed consent document. After the clinical trials are complete, researchers will analyze the data they have collected and publish their findings. This process could take several years and not all researchers are able to reveal the results of the trial at its conclusion. Be sure to address this with the study investigator before enrolling.

Listed below are common questions asked about participating in clinical trials. If you have questions regarding a specific trial, use the contact information provided in the study listing.

Where do I find clinical trials that are recruiting?

Advertising for clinical trials can be found in several different places including on the radio, television, through advertising efforts in print publications, and community common areas such as waiting rooms. You can also ask your physician if s/he is conducting any studies or if they know of any colleagues who are. Generic search engines may yield some results or you can use those that are dedicated to clinical trials such as ClinicalTrials.gov.

What are the benefits of participating?

By participating in a clinical trial, you are taking an active role in your own health care and contributing to the betterment of health care for others. For some participants, clinical trials offer exposure to new treatment methods that are not otherwise available. Participating in a clinical trial can also be very beneficial for those who struggle to find ways to effectively control or manage their disease.

Can anyone participate?

The main goal of a clinical trial is to study a predetermined research question. In order to effectively pursue an answer to that question, researchers create a list of criteria that study participants must meet in order to be considered for the study. These criteria are put in place not only to ensure that the end results of the trial are being targeted toward a specific result, but also to keep participants safe.

What does being a participant involve?

The processes involved from one study to another will vary. Things you could be asked to do include: filling out questionnaires, taking an experimental drug, undergoing a new therapy, using a newly-designed device, regular meetings with doctors or researchers, or changing your lifestyle. Participants could be asked to do one or any combination of these things, but each study will have a predetermined set of responsibilities that must be agreed upon to participate in the study. All of these details will be outlined and explained in the study protocol which is given to participants as one of the first steps.

Is it safe to be a study participant?

Researchers and the IRB (Institutional Review Board) affiliated with the study work to ensure that the study is conducted in the safest way possible. However, depending on the study there may be some risks involved. These risks will be explained to you before the study begins and if any new risks are discovered, researchers will notify you and ask if you wish to continue to participate in the study. If at any time you feel that participating in the study is unsafe, you may address your concerns with the researcher and discontinue your participation.

Will I get paid?

Some studies offer payment for participation, others compensate participants for their time and expenses such as parking, while some may not have the funds to pay participants at all. Your motivations for participating in a clinical trial should not involve taking risks you wouldn't normally to receive payment. The terms of payment will be explained before beginning participation.

Does it cost me anything?

Just as not all trials offer payment for participation, not all trials come at no cost to the participant. It is important to direct all questions about cost to the researcher including (if applicable) any costs that may be billed to your insurance. In addition to verifying this with the research team, you should discuss concerns about cost with your insurance provider.

Will I still see my regular doctor?

Since most clinical trials are a short-term commitment you should continue to work with your primary care physician. Including your physician in your plans to participate in a trial is beneficial for you to prevent any possible side effects or adverse reactions occurring as a result of your current treatment plan being combined with the study protocol. Your doctor will also have a better idea of whether or not participating in the study could be helpful or harmful for you.

Am I allowed to leave the study?

Participants are allowed to leave a study at any time, no questions asked. If you have concerns prior to leaving a study, you should address them with the researchers before terminating your participation. Whether you choose to leave the study or not is your choice and you shouldn't be pressured into making your decision. However, if you do decide to leave the study, explaining your reasons for leaving to the researcher could further contribute to the research process.

What happens with my information?

Researchers collect participant's information to inform and enhance their research. Before agreeing to participate, you will have to sign a consent form that states you agree to share your information with the research team. Though you are releasing your information to the researchers, your personal health information will remain confidential and protected by HIPPA guidelines. HIPPA ensures that all of your identifying information such as name, date of birth, SSN, address, etc. are concealed and only referenced when absolutely necessary by a health care professional. This information will never be published with study results and no one will know you're participating in the study unless you tell them.

Participating in a study is one of the greatest and most fulfilling ways to give back to your community. In research, communities are classified in a variety of ways including people with the same disease, those who are healthy versus those who are not, people living in a specific geographical location, and even people of the same sex, age, or ethnic group. These characteristics are a strong component in the foundation of any clinical trial.

Treatments prescribed during a clinical trial are considered experimental as they have not been approved by the FDA. Though there are risks associated with every new treatment, doctors and researchers conducting clinical trials take every step necessary to ensure that study participants are safe.

For people who have been diagnosed with life-threatening or chronic illnesses, clinical trials offer the chance to try a new treatment. This is especially beneficial for those who have had little or no success with the standard FDA-approved treatments. Another common reason people choose to participate in a study is that is offers a way to champion a cause and the battle to prevent and defeat a specific illness or condition—a swaying factor for many who have loved ones who have struggled with chronic disease.

People who elect to participate in a clinical trial aren't just protected physically—they are protected by HIPPA (Health Insurance Portability and Accountability Act) which mandates that personal information related to the patient or participant be guarded and protected. This ensures that patients' information is never leaked or compromised, keeping the details of their medical history safe and confidential.

Choosing to participate in a trial is a personal decision that should be considered carefully. However, most study participants find it to be a fulfilling if not beneficial experience. If at any time you wish to withdraw from the trial, you have full control to do so. This ensures that you are always comfortable with your commitment. If you decide that being a study participant is right for you, the next step is finding a trial that is compatible with your needs or characteristics. Remember that researchers aren't just looking for people with illness or disease—they often need healthy participants too.

There are five main categories of clinical trials and each targets a different, yet specific, element of the health care process.

1. Treatment Trials aim to test the efficacy of a new or experimental treatment such as drugs, surgeries, devices, or radiation therapy. In treatment trials, participants with a specific condition or disease have the opportunity to try a treatment that would otherwise be unavailable to them.
2. Prevention Trials target ways to prevent a disease from presenting or recurring and can include drugs, lifestyle changes and even alternative therapies such as vitamins and nutritional supplements.
3. Diagnostic Trials test new and more effective ways to identify a disease or condition.
4. Screening Trails, similar to diagnostic trials, target methods of finding the best way to detect a disease or condition.
5. Quality of life Trials differ the most as these studies focus on individuals with chronic illness and analyze various ways to improve their quality of life.

Clinical trials are also sometimes classified as interventional or observational. Interventional trials expose new treatments and treatment methods to individuals of a chosen group in a controlled environment. Observational trials take a given health issue and assess that issue as it applies to a large group of people in a natural setting (e.g. students in a specific school district or residents of a geographical area).

As with any new process, it is extremely important to ask questions of the PI (Principal Investigator of the study) and your PCP (primary care physician). You should never feel embarrassed to ask a question as it will not only improve your understanding of what you're volunteering for, but it will also inform the PI of any concerns you have and uncover elements of the trial that need a better explanation.

In the days before meeting with the PI or your PCP to discuss your involvement in the clinical trial, write down any questions you have, so you don't forget them at your meeting. Listed below are a set of questions that can be used as a starting point:

• What is the purpose of the study?
• What will I have to do to participate in the study? If needed, will a schedule be provided for me?
• Can my condition be improved or worsened if I volunteer as a research participant?
• What are the potential side effects I could experience?
• How long will I be required to participate?
• Will my participation require assistance at home for the administration of drugs or therapies?
• Who should I contact if I have any concerns about my condition or my participation in the study?
• Is any additional follow-up required after my participation ends?
• If I experience negative side effects, what happens?
• What are the costs associated with participating? Are they covered by my insurance?
• Is there a specific location where I have to go to participate? Are travel expenses covered?
• How does my participation aid the purpose of the study?
• Am I allowed to discuss the nature of this study with loved ones or doctors?

Remember, it is better to ask a question and receive a firm answer than it is to assume you know the answer. The research team is there not only to conduct research, but to support the research participants through the process. Make sure you know who to contact if you have any additional questions or need extra support while participating in the trial. Clinical trials are an imperative step in the evolution of medical science — without dedicated research participants, the process of improving health care and medical science becomes infinitely more difficult.

It is encouraged that researchers register their trial with ClinicalTrials.gov's Protocol Registration System (PRS). It's not only a necessary step in the research process, but also the best way to attract study participants and publication opportunities. Below is an overview of the registration process.

How to Register a Trial:

In order to register a trial, you must have a Protocol Registration System (PRS) account. These can be obtained by either an organization or an individual investigator. Organizations are able to register several users allowing for multiple trials to be registered easily. To prevent duplicate registration, take care to have a registration system in place. In instances where there are multiple sites or several sponsors involved, the lead sponsor should be responsible for registering the trial.

Why Register a Trial?

As mandated by the Food and Drug Administration Amendments Act of 2007, Section 801, “applicable clinical trials” must be registered and publically report results. Applicable clinical trials are commonly defined as those that are interventional studies (studies consisting of one or more research arms) and those that are trialing devices, drugs or biological products that would require FDA approval.

Trials may be registered before obtaining IRB approval, but they cannot begin recruiting until they have received approval from their institution's IRB. At that time, principal investigators should update study status from $ldquo;not yet recruiting” to “recruiting” and make any necessary updates to the protocol.

How to Report Results:

Results must be reported for all registered trials. To do this, results can be submitted manually, or synched with ClinicalTrials.gov's XML structure through an organizational account. Results must be reported within one year of the final data collection.

For more information on reporting results, read “Reporting ‘Basic Results’ in ClinicalTrials.gov,” published by CHEST Journal.