Researchers conduct clinical trials to try to learn if an experimental new drug works and is safe in humans so it can be approved. Clinical trials may also study a medical device or a medical procedure.
It takes a lot of research to develop a new drug treatment. Clinical trials are done in several steps, also called “phases.” Each phase has a specific purpose. The phases are designed to answer certain questions about the experimental drug being tested while aiming to keep the patients in each trial as safe as possible.
Researchers study experimental drugs in a laboratory before studying them in humans. This type of research study is called a "pre-clinical trial".
The goal is to collect information about the experimental drugs. Then researchers ask the government and other independent groups for permission to begin a clinical trial in humans. Pre-clinical trials are required before the experimental drug can be studied in humans.
Phase 1 clinical trials are the first step after the pre-clinical trials. Phase 1 clinical trials are usually the first time a new experimental drug is studied in humans.
A Phase 1 clinical trial tests the drug in a small group of people (usually 15 to 100 patients). Researchers design phase 1 trials to learn:
Phase 2 clinical trials assess how well the experimental drug is working in people who are diagnosed with a disease or condition.
There are usually 100 to 500 patients.
Researchers design phase 2 clinical trials to:
If the results of phase 1 and phase 2 clinical trials are positive, the experimental drug is studied in a larger number of patients with the disease or condition.
There are usually 1000 to 5000 patients.
Researchers design phase 3 clinical trials to:
Phase 4 clinical trials are conducted after the experimental drug is approved by regulatory authorities. These clinical trials may take place over many years and usually involve a large number of patients.
Researchers design phase 4 clinical trials to:
Advanced disease means that the cancer has spread to a nearby tissue or lymph node or spread to another part of the body.¹
A gene that makes a protein called anaplastic lymphoma kinase (ALK), which is involved in cell growth.²
An increase in the number of copies of a gene.³
There are different targeted therapies, one of them is antibody targeted treatment. Antibodies can target receptors on the outside of cancer cells, and can block or turn off signals that tell cancer cells to grow and divide.4,5
A therapy where a patient is treated with more than one type of cancer treatment. The different cancer treatments usually work in different ways to try and improve the effect of the treatment.6,7
A protein found on certain types of cells that binds to a substance called epidermal growth factor. The EGFR protein is involved in cell signaling pathways that control cell division and survival.⁸
Left-sided colon cancer typically constricts the bowel channel, causing partial blockage. Typical symptoms include constipation, a change in bowel habits, and narrow, ribbon-shaped stool when a cancer is low in the rectum.⁹
MET amplification occurs when several copies of the MET gene are created, resulting in increased levels of MET receptor on the cell surface.¹⁰
A gene that makes a protein called MET, which is involved in sending signals within cells and in cell growth and survival. Mutated (changed) forms of the MET gene may cause abnormal cells to grow and spread in the body.¹¹
Having to do with metastasis, which is the spread of cancer from the primary site (the place where it started) to other places in the body.¹²
The use of a single drug to treat a disease or condition.¹³
Non-small cell lung cancer
Non-small cell lung cancer is the most common kind of lung cancer. The three main types of non-small cell lung cancer are squamous cell carcinoma, large cell carcinoma, and adenocarcinoma.¹⁴
(a tyrosine kinase inhibitor)
A substance that blocks the action of enzymes called tyrosine kinases. Tyrosine kinases are a part of many cell functions, including cell signaling, growth, and division. These enzymes may be too active or found at high levels in some types of cancer cells, and blocking them may help keep cancer cells from growing.¹⁵
A measure of how well a patient is able to perform ordinary tasks and carry out daily activities.A measure of how well a patient is able to perform ordinary tasks and carry out daily activities.¹⁶
When cancer cells don’t respond to a drug that is usually able to kill or weaken them. Drug resistance may be present before treatment is given or may occur during or after treatment with the drug. Drug resistance can lead to a cancer treatment's not working or to the cancer's coming back.¹⁷
Requirements, also called “eligibility criteria,” can be related to age, sex, the type and stage of a medical condition or disease, previous treatment, and other medical conditions.
“Inclusion criteria” are the requirements a person must meet to be considered for the clinical trial. Allowing only certain patients to join a clinical trial helps researchers determine if the results of the clinical trial are caused by the experimental drug and not by other factors.
“Exclusion criteria” are a list of conditions and reasons that might prevent a person from being in a clinical trial. These criteria may help protect people whose health may become worse if they join the clinical trial.
Yes. A patient can leave a clinical trial at any time and for any reason. Patients may choose to tell the research team when they are leaving and why, but they are not obligated to do so.
It is important for people to ask questions before deciding to join a clinical trial. Some questions people might want to ask their doctor or nurse are below.
The clinical trial
Benefits and risks
Participation and care
Costs can depend on a country’s laws and a patient’s health insurance.
Clinical research done in people must follow strict rules. Every clinical trial has a plan called a “protocol.” The protocol is a set of instructions that everyone in the clinical trial must follow. It is the plan for how the clinical trial will be carried out.
Before a clinical trial begins, it must be approved by a government department (such as the FDA in the United States or the European Medicines Agency in Europe) and other organizations (such as ethics committees). Different countries require different approvals. Approvals seek to ensure that trial patients' rights and welfare are protected from unreasonable risks. Nonetheless, while efforts are made to control the risks to patients, some risks may be unavoidable since clinical trials are designed to learn more about an experimental drug.
A “placebo” looks like the experimental drug but does not have any medicine in it. In clinical trials, new drugs are sometimes compared with placebos. Some patients are given a placebo instead of the experimental drug. This helps to see if any effects seen are actually caused by the experimental drug being studied.
“Informed consent” is a process to make sure a patient understands important information about a clinical trial before agreeing to be in the clinical trial. Information includes the purpose of the clinical trial, one's rights as a patient, the experimental drugs used, and the risks and benefits.
If you are considering participating in a clinical trial, you can ask the doctors and nurses questions about the clinical trial to decide if you feel comfortable joining. You can also talk with family, friends, or your personal doctor before you decide.
You decide if you want to participate in the clinical trial and sign the consent form. The informed consent can be translated into other languages for the clinical trial patient. No one should pressure or influence your decision in any way. Parents or guardians decide whether children can participate in a clinical trial and whether to sign the consent form on their behalf.
If a patient decides to join a clinical trial, the patient must sign an informed consent form. Signing the form indicates that the patient understands the trial is research and that he or she can leave a clinical trial at any time. The research staff will help the patient do it safely.
If new benefits, risks, or medical problems are discovered during the clinical trial, the researchers must tell the patients. Patients may need to sign a new informed consent form to stay in the clinical trial.
If a patient does not meet the eligibility criteria to join a clinical trial, they should speak with their doctor or other healthcare provider to learn about what other options there are.
Below is a list of sources where more information about clinical trials may be found: