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Please Confirm

This website is an information resource intended only for Healthcare Professionals from U.S. who are interested in information on Clinical Research and EMD Serono clinical trials. 

By clicking “I am a healthcare professional” you are declaring and confirming that you are a Healthcare Professional inside the U.S. and have read and understood this disclaimer.

I AM NOT A HEALTHCARE PROFESSIONAL

ABOUT CLINICAL STUDIES

What Is a Clinical Trial?

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.

Clinical Trial Phases

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.

 

PRE-
CLINICAL
RESEARCH

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

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:

  • What the experimental drug's serious adverse effects are
  • The dose that can be given
  • How the experimental drug is absorbed and processed in the body

PHASE 2

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:

  • Learn if the experimental drug may work in people who have a certain disease or condition
  • Continue to learn about which dose of the experimental drug works

PHASE 3

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:

  • Confirm the medical adverse effects and the results seen in earlier phases
  • Learn how well the experimental drug works compared with another drug or compared with a placebo

PHASE 4

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:

  • Learn how a new drug works when people are taking it as part of their everyday lives
  • Identify and evaluate the long-term effects of a new drug over a lengthy period for a greater number of patients

Glossary of Additional Terms

 

Advanced disease

Advanced disease means that the cancer has spread to a nearby tissue or lymph node or spread to another part of the body.¹


ALK gene

A gene that makes a protein called anaplastic lymphoma kinase (ALK), which is involved in cell growth.²


Amplification

An increase in the number of copies of a gene.³


Antibody-targeting treatment

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


Combination therapy

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


EGFR protein

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

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

MET amplification occurs when several copies of the MET gene are created, resulting in increased levels of MET receptor on the cell surface.¹⁰


MET gene

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


Metastatic

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


Monotherapy

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


Osimertinib

(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


Performance status

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


Resistance

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

  1. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/advanced-cancer.
  2. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/alk-gene.
  3. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed June 11, 2021. https://www.cancer.gov/search/results?swKeyword=gene+amplification
  4. Cancer.Net. Accessed June 11, 2021. https://www.cancer.net/navigating-cancer-care/how-cancer-treated/personalized-and-targeted-therapies/understanding-targeted-therapy
  5. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/about-cancer/treatment/types/targeted-therapies/targeted-therapies-fact-sheet.
  6. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/combination-therapy.
  7. Merck Manual Consumer Version. Prevention and Treatment of Cancer. Accessed September 10, 2021. https://www.merckmanuals.com/home/cancer/prevention-and-treatment-of-cancer/combination-cancer-therapy.
  8. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/egfr.
  9. National Cancer Institute. SEER Training Modules. Accessed June 11, 2021. https://training.seer.cancer.gov/colorectal/intro/types.html#:~:text=Left%2Dsided%20colon%20cancer%20typically,is%20low%20in%20the%20rectum
  10. Drilon A et al. J Thorac Oncol. 2017;12:15-26.
  11. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/794425.
  12. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed June 11, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/metastatic
  13. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/monotherapy
  14. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/non-small-cell-lung-cancer.
  15. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/tyrosine-kinase-inhibitor.
  16. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed September 9, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/performance-status.
  17. National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed June 11, 2021. https://www.cancer.gov/research/annual-plan/scientific-topics/drug-resistance

 

Frequently asked questions
 

  • All patients should discuss their plans to participate in a clinical trial with their personal doctors. The doctor will assess if a clinical trial is the right option
  • All clinical trial patients should discuss their questions and concerns with members of the research team conducting the trial. See below for potential questions patients might ask before joining a clinical trial
  • Participating in a clinical trial takes time, commitment, and careful consideration
  • The clinical trial could end at any time. Even if the patient wants to continue to participate, the company making the experimental drug could stop the clinical trial
  • Clinical trial patients' conditions may not improve
  • Clinical trial patients may not have the opportunity to access the experimental drug after the clinical trial has ended
  • Clinical trial patients may experience unwanted adverse effects from the experimental drug
  • Clinical trial patients may be assigned to receive the experimental drug, placebo, or a comparator

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

  • What is the purpose of the clinical trial?
  • Why do the researchers think the experimental drug being tested may work? Has it been tested before?
  • Does the clinical trial involve a treatment that is already on the market or a placebo? What are the chances of receiving the experimental drug, treatment that is already on the market, or placebo?
  • Will I know which treatment I am receiving in the clinical trial?
  • Who is sponsoring the clinical trial?
  • Who has reviewed and approved the clinical trial?
  • What are the medical background and experience of the researchers and other staff?
  • How will the results of the clinical trial and safety of the patients be monitored?
  • How long will the clinical trial last?
  • When and how will the results of the clinical trial be shared?

Benefits and risks

  • What are the benefits that the patients may have right away?
  • What are the benefits that the patients may have over a long time?
  • What are the risks, such as adverse effects, that the patients may have right away?
  • What are the risks that the patients may have over a long time?
  • How do the risks and possible benefits of the experimental drug compare with the risks and benefits of other treatments?
  • What are the options if a patient is injured during the trial?

Participation and care

  • What kinds of treatment, medical tests, or procedures will the patients have during the clinical trial?
  • What other treatments are available?
  • How often will patients get the experimental drug, or have tests or procedures done?
  • Who will know what experimental drug the patients get during the clinical trial? Will patients know? Will the research team know?
  • Will the experimental drug, tests, or procedures hurt? If so, how can the pain be controlled?
  • How do the tests in the clinical trial compare with what patients not in the trial get?
  • Can patients take their regular medications while in the clinical trial?
  • If a patient benefits from the experimental drug, can the patient keep getting it after the clinical trial ends?
  • Where will the patients get their medical care? Will they be in a hospital? If so, for how long?
  • Who will be in charge of the patients’ care? Will patients be able to see their own doctors?
  • How long will patients need to stay in the clinical trial? Will there be follow-up visits after the clinical trial?

Personal issues

  • How could being in the clinical trial affect the patients’ daily lives?
  • What support is available for the trial patients and their families?
  • Can a potential patient talk with patients already in the clinical trial?
  • Can anyone find out if patients are in the clinical trial?

Cost issues

Costs can depend on a country’s laws and a patient’s health insurance.

  • Will patients have to pay for any treatment, tests, or other charges? If so, how much will it be?
  • Will patients’ health insurance have to cover some of the cost?
  • Will patients be reimbursed for other expenses, such as travel, parking, and childcare?
  • Where can patients find more information about cost issues?

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:

  • The US National Institutes of Health (NIH) requires that many clinical trials be posted on the website www.clinicaltrials.gov
  • CenterWatch keeps the New Medical Therapies (NMT) database, offering a concise overview of investigational therapies for a specific disease or condition. To search the NMT results, select a therapeutic area
  • For trials involving a an experimental cancer drug, visit the National Cancer Institute (NCI)