Clinical Trials

The goal of clinical trials is to find out whether new medical approaches that are being developed are safe and effective and better than those currently being used. Most drugs or medical procedures that are available for patients today went through clinical trial testing.

Clinical Trials brochureTo help you understand and share this information, click here to download and print a booklet that summarizes the detailed information in the following sections.

This phase of research is possible only if patients who have the condition that is being studied participate, so a better name for clinical trials would be “patient-assisted research studies.” Clinical trials are an important option for people thinking about lung cancer treatments because the newest treatment approaches are being tested in them.

What is a clinical trial?

Clinical trials are my new hope, my new best friend. At first I was terrified of them, now I’m excited to have options. Hope is 'a chance'!

- Linnea Duff, stage IV adenocarcinoma

Clinical trials are research studies in people that take place in a medical (clinical) setting. These studies test new medical approaches for their safety and efficacy among a group of volunteer participants.

For people with lung cancer, clinical trials test new ways to

  • Find and diagnose lung cancer
  • Treat lung cancer
  • Manage symptoms of the lung cancer or side effects of treatment
  • Prevent lung cancer in the first place (through chemoprevention, for example)

New lung cancer treatments are always tested in volunteers with the type and stage of lung cancer for which the new treatment is intended. Clinical trials are vital for adding to medical knowledge that can improve the care given to patients. The results of these research studies determine whether new treatments are approved by the FDA for prescribing by doctors for patients outside of clinical trials.

Each clinical trial has its own protocol, or study plan. The protocol outlines every aspect of how the clinical trial is to be conducted, including:

  • The reason for doing the trial
  • Who can join the trial (called “eligibility requirements” or “inclusion criteria” and “exclusion criteria”)
  • How long the study will last
  • What treatment is given, how the treatment is given, and how often the treatment is given (for studies of new treatments)
  • What medical tests will be done to measure whether the treatment is working
  • What types of information will be collected about the people taking part

Note: Patients who participate in treatment-focused clinical trials receive either a new treatment/combination of treatments or the treatment that is currently considered the “gold standard”—the best currently available proven treatment. A patient participating in a clinical trial will never be given less than the standard of care.

In the video below from Tampa HOPE Summit in March 2015, experts and survivors talk about why is it so important to seek out clinical trials.

Learn more about different types of cancer clinical trials on the National Cancer Institute website.

Where do clinical trials take place?

There is no one standard for where clinical trials take place. Some are available in just a few places; others have sites at hundreds of locations—across the United States and in many other countries. They may take place in doctors’ offices, community hospitals and clinics, cancer centers, veterans’ and military hospitals, or at the National Institutes of Health Clinical Center.

When you talk with your doctor about treatment options, ask about clinical trials. There may be one that is right for you taking place at your doctor’s office or nearby. You can find out more about whether there is a clinical trial that is right for you further down on this page, in the “Finding a clinical research study that might be right for you” section.

What are the phases of a clinical trial?

Clinical trials to test new cancer treatments involve a series of steps, called phases. There are three main phases of clinical trials. If a new treatment is successful in one phase, it will move on to further testing in the next phase. A drug or device usually has to show success in the first three phases before it is approved by the FDA for broader use.

However, the FDA also has a number of programs to support faster development and review of new drugs for areas of unmet medical need, such as lung cancer. These programs include breakthrough therapy designation, fast track designation, accelerated approval, and priority review. When a treatment is developed through one of those programs, a drug may be approved after strong phase 2 success, for example, and then monitored closely afterward.

You may also hear the term “phase 4”—this is also called post-marketing surveillance. The goal is to monitor the long-term effectiveness and safety of an approved treatment after it is on the market with FDA approval.

Before a drug is studied in humans, it has been studied in the pre-clinical research phase. During that time, the treatment is generally studied in laboratory animals for safety and efficacy.

The following table shows the numbers of patients who take part and the purpose of the most common phases.

Phase

Goals

Number of Patients

Phase 1

  • To find a safe dose of a new treatment
  • To decide how the new treatment should be given (by mouth, in a vein, etc.)
  • To see how the new treatment affects the human body (whether it causes side effects)
  • 15-30 people
  • May be a mix of people with different cancers

Phase 2

  • To find out if the new treatment has an effect on a certain type of cancer, like lung cancer
  • To see whether the new treatment causes any side effects
  • Fewer than 100 people
  • All the same kind of cancer

Phase 3

  • To compare the efficacy of the new treatment (or new use of a treatment) with the current standard treatment
  • From 100 to several thousand
  • All the same kind of cancer

Some researchers design trials that combine two phases (phase 1/2 or phase 2/3 trials) in a single protocol. In this combined design, there is a seamless transition between trial phases, which may allow research questions to be answered more quickly or with fewer patients.

How are people assigned to different treatments in clinical trials?

In some phase 2 and all phase 3 clinical trials, patients are assigned to groups that receive different treatments.  

The assignments are made randomly, usually by a computer, so that neither the researchers nor the patient knows which group the person is in. In the most basic trial design, one group receives the new treatment. This group is called the investigational group. The other group receives the current standard therapy. This group is called the control group.

When are placebos used in clinical trials?

In lung cancer clinical trials, patients are never given a placebo instead of an effective standard treatment.

Placebos are rarely used in cancer treatment clinical trials. They are used when there is no standard treatment, or they may be used in a clinical trial that compares standard treatment plus a new treatment with standard treatment plus a placebo, as in the image below. The placebo is designed to look like the medicine being tested, but it is not an active drug. Using a placebo in this way can help prevent patients and their doctors from figuring out which treatment group the patients were assigned to.

Placebo use in clinical trials

You always will be told if the study uses a placebo.

What happens after the clinical trial is over?

The clinical trial’s research team will remain in contact with you and will let you know about the trial’s findings and conclusions. They may ask you to continue to provide information about your health either through surveys or actual health examinations. This is in addition to the regular care provided by your own doctor.

Who can participate in a clinical trial?

Each clinical trial defines who is eligible to take part. Each trial must include only people who fit the patient traits for that study (the eligibility criteria). Criteria for who can take part in a trial might include:

  • A specific age range
  • Gender
  • The type of lung cancer—could be by histology and/or molecular mutation
  • The stage of lung cancer
  • Site of metastases—for example, whether someone has brain (or central nervous system) metastases
  • Previous treatment history—type of treatment or how many different types of treatment
  • Medical history
  • Current health status

Criteria such as these help reduce the medical differences among people in the trial. When people taking part in a trial are alike in key ways, researchers can be more certain that the results are due to the treatment being tested and not to other factors.

Also, some people have health problems besides lung cancer that could be made worse by the treatments in a trial. If you are interested in joining a trial, you will receive medical tests to be sure that you are fit for the trial.

The factors that allow someone to participate in a clinical study are called inclusion criteria, and the factors that disqualify someone from participating are called exclusion criteria.

How are patients in clinical trials kept safe?

People who take part in clinical trials are protected in a number of ways. In fact, the federal government has rules in place to help ensure both the safety and the ethics of clinical trials.

Ways to keep people safe include:

Informed consent process: Researchers must provide patients who are thinking about joining a clinical research study with detailed information about the study. This includes enough information about the trial’s purpose and possible risks and benefits that a patient can decide whether to take part. This information must be given in writing and may be discussed with the doctors and nurses in the research team. If a person decides to take part in a study, he or she will be given an informed consent document to sign, confirming understanding of this information.

This informed consent document is not a contract. Participants always have the right to leave the trial at any time, even if it is not over yet.

Review of the clinical trial protocol (plan) by an institutional review board (IRB): There is an IRB at every health care facility that does clinical research. This board includes doctors, researchers, and community members. The IRB’s role is to ensure that the clinical trial protocol is ethical and that the rights and welfare of the people who take part are being protected.

Ongoing monitoring of the clinical trial: Once the clinical trial is under way, it continues to be monitored by the IRB as well as by the sponsor of the trial, the research team conducting the trial, and, for the phase 3 portion of a clinical trial, a Data and Safety Monitoring Board (DSMB), which decides whether a trial should be changed or closed. The DSMB is made up of doctors, statisticians, and others who are independent of the people, organizations, and institutions that are sponsoring, organizing, and conducting the clinical trial. DSMB members are experts in clinical research and clinical trials. The FDA also meets with researchers and inspects the clinical trial sites.

How does a clinical trial fit in with a person’s overall health care?

The research plan (protocol) for the clinical trial determines how often a participant receives a drug treatment and what exactly that treatment will be. Most often, a person taking part in a clinical trial will continue to receive the rest of his or her health care from their usual doctors. This is true whether the person is receiving the study treatment there or not. The researchers and usual doctors can work together to ensure that the clinical trial protocol will not conflict with any other drugs or treatments that the person is taking.

What are the benefits and risks associated with a clinical trial?

Like with all treatment options, there are both benefits and risks for people who take part in a clinical trial. Before deciding to take part, you should carefully consider both. If you are thinking about taking part in a clinical trial, ask your doctors and the research team as many questions as you need to until you are satisfied that you understand exactly what is involved. Here is more information about possible benefits and risks.

Possible benefits:

  • You will have access to a new treatment that is not otherwise available, reflecting the latest thinking in lung cancer research and treatment
  • The research team will monitor your health closely, providing excellent care
  • If the treatment being studied is more effective than the standard treatment, you may be among the first to benefit
  • Even if you do not directly benefit, the information gathered during the clinical trial will still add to knowledge about lung cancer and can help other people

Possible risks:

  • The new treatment may be no better than, or even as good as, the standard treatment
  • New treatments may have side effects that doctors do not expect or that are worse than those of the standard treatment
  • You may be required to make more visits to the doctor or have more hospitalizations than if you were receiving standard treatment
  • You may need extra tests, and some of these may be uncomfortable or time-consuming
  • Even if a new treatment has benefits in some patients, it may not benefit you
  • Health insurance may not cover all patient care costs in a trial. In addition, you may have extra expenses related to extra doctor visits, such as travel and child-care costs
  • You may have to travel to the place the research treatment is being given

Paying for clinical trials

As you think about taking part in a clinical trial, you will face the issue of how to cover the costs of care. There are two types of costs associated with a clinical trial: patient care costs and research costs.

Patient care costs are costs related to treating your cancer, whether you are in a clinical trial or receiving standard therapy. These costs must be covered by most health insurance, under the Patient Protection and Affordable Care Act.

They include:

  • Doctor visits
  • Hospital stays
  • Lab tests
  • X-rays and other imaging tests

Research costs are those related to taking part in the trial. Often these costs are not covered by health insurance, but they may be covered by the trial’s sponsor.* Examples include:

  • The study drug
  • Lab tests performed purely for research purposes
  • Additional X-rays and imaging tests performed solely for the trial

Also, when you take part in a trial, you may have extra doctor visits that you would not have with standard treatment. These extra visits can add costs for transportation and child care. Information about resources that can provide help with these and other costs can be found in the Support & Survivorship section.

*Clinical research trials may be sponsored—developed and supported by—a number of different organizations and entities, including:

  • Pharmaceutical or biotechnology companies
  • Academic medical centers
  • Community hospitals
  • Government agencies, such as the National Institutes of Health and the US Department of Defense
  • Physicians
  • Patient advocacy groups

Finding a clinical research study that might be right for you

If you are considering participating in a clinical trial, start by asking your doctor whether there is one that might be a good match for you in your geographic area.

In addition, here are several resources to help you find one that may be a good match:

LUNGevity partners with EmergingMed, a clinical trials matching service, to help you with the decision of whether to participate in a clinical trial. EmergingMed helps you identify which lung cancer clinical trials you may be eligible for. The clinical trial navigators can also guide you through the process of getting enrolled if you choose to take part in a clinical trial.

Clinical trial navigators are available Monday through Friday from 8:30 am to 6:30 pm ET at 800-698-0931. Learn more about this free service and even fill out an online profile at http://www.emergingmed.com/networks/LUNGevity/

Information about available clinical trials is also available through these websites. The first two include all trials for all cancers, not just lung cancer. The last three focus on people with mutations.

  • National Cancer Institute (NCI): http://www.cancer.gov/clinicaltrials/search. This site has all of the 12,000+ clinical trials in the United States in all cancer types.  
  • Coalition of Cancer Cooperative Groups: http://www.cancertrialshelp.org/cancer-trial-search/. This site gets its information from www.clinicaltrials.gov too, but organizes the search and results in a different way.
  • My Cancer Genome: http://www.mycancergenome.org/. This resource is managed by a team of doctors at Vanderbilt University. My Cancer Genome gives up-to-date information on what mutations make cancers grow and related treatment options, including available clinical trials.
  • Lung Cancer Mutation Consortium (LCMC): http://www.golcmc.com/. Composed of 16 leading cancer centers across the country, LCMC’s goal is to prospectively examine the tumors of patients with the type of advanced (stage IIIB or IV) non-small cell lung cancer called adenocarcinoma, and match patients to the best possible therapies, including clinical trials.
  • Lung Cancer Master Protocol (Lung-MAP): http://www.lung-map.org/. For patients with squamous cell lung cancer, Lung-MAP is a collaboration of many research sites across the country, using a unique approach to matching patients to one of several drugs being developed.

In addition, if you are interested in a specific drug or other treatment that is being developed, you can often find information about studies for that drug on the website of the company developing it.

Which mutations identified in lung cancer are currently being studied in clinical trials?

Currently clinical trials are open for many drugs that inhibit the effect of genetic mutations seen in NSCLC. The targeted treatments are being studied alone and in combination with other targeted agents, chemotherapy, and radiation therapy.

See "Which mutations identified in lung cancer are currently being studied in clinical trials?" in the Targeted Therapy section for information on specific mutations.

Questions to ask your health care team about clinical trials

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  1. How do I know if I am a possible candidate for a clinical trial?
  2. Are clinical trials only for people who have failed all other options?
  3. If I am a candidate to receive an approved standard therapy, why should I participate in a clinical trial?
  4. Are clinical trials safe?
  5. What are some of the risks and benefits of participating in a clinical trial?
  6. What is the goal of this trial? Who is sponsoring it?
  7. What is known about the investigational drug being studied? Has it worked in previous trials? Is it the same as chemotherapy?
  8. How will I be given the drug? How often and for how long?
  9. Are there tests to determine if I am eligible for this trial?
  10. What types of tests, scans, or other procedures are required during the trial, and how frequently will they need to be performed?
  11. What side effects might I experience if I’m given the investigational drug? Are the side effects reversible, and can they be managed?
  12. Are the side effects from the investigational drug worse than those I might experience with standard treatment? How severe could these side effects be?
  13. Will I lose my hair?
  14. Will I be able to continue working or go about my daily routine?

Updated March 7, 2016.


References

  1. Clinical Trials Information for Patients and Caregivers. National Cancer Institute website. http://www.cancer.gov/about-cancer/treatment/clinical-trials. Accessed March 7, 2016.
  2. Learn About Clinical Studies. ClinicalTrials.gov website. https://clinicaltrials.gov/ct2/about-studies/learn#WhatIs. Reviewed December 15, 2015. Accessed March 7, 2016.
  3. Frequently Asked Questions: Breakthrough Therapies. US Food and Drug Administration website. http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmendmentstotheFDCAct/FDASIA/ucm341027.htm. Updated May 6, 2015. Accessed March 7, 2016.

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