What Are Clinical Trials?

Clinical trials are experimental studies using groups of patient volunteers to test the efficacy of new medications, treatments, prevention methods, and more. A clinical trial can be held in a variety of settings, including hospitals, doctors’ offices, community clinics, and universities. Moreover, some trials may be conducted in multiple locations across the country or world.

The goal of clinical trials is to improve patients’ quality of life as well as search for cures. In the U.S., clinical trials are made up of four main phases. A team of doctors, nurses, and other health care professionals is led by a principal investigator (usually a medical doctor) through each phase of the trial.

For mesothelioma patients, clinical trials provide improvements in surgery and radiation procedures; new chemotherapy, targeted therapy, and immunotherapy medications; and other ways of extending the lives of patients.

Phases of Clinical Trials

For new treatments to be approved, a new drug or procedure goes through three of the four primary phases of clinical trials. The fourth phase often lasts years to monitor the treatment after approval by the Food and Drug Administration (FDA). Many studies include pre-clinical (or laboratory) studies to test drugs on cells or animals before moving on to human patients.

Also, Phase 0 may be included before the official clinical trial with fewer than a dozen patients and a few, small doses of the new medication. Phase 0 studies are not a required part of clinical trials.




I (1)

Confirm treatment is safe to treat the condition.

Phase I includes about 15 to 30 patients. It looks at safe dosage levels and how the body metabolizes the drug. Phase I usually lasts several months.

II (2)

Determine how well treatment works (or doesn’t work).

These trials are conducted with a larger group of people to determine how well certain dosages achieve the desired effect of treatment (such as pain relief, tumor shrinkage, etc.). Phase II also examines any side effects. Phase II can last several months up to a few years.

III (3)

Compare efficacy to currently available treatments.

Before FDA approval, the treatment is tested against the current standard treatments among several hundred patients, as doctors continue to watch for potential side effects. Phase III typically lasts longer than phase I and II.

IV (4)

Study long-term uses, side-effects, and reactions.

After FDA approval, Phase IV studies the treatment’s effect on thousands of people over a number of years. This phase uncovers any long-term side effects and proves the long-term quality of life and costs of treatment.

Are Clinical Trials Safe?

Because clinical trials are often the initial human tests for new treatments, there is a certain amount of risk patients must consider before entering. However, all clinical trials are reviewed, monitored, and approved by an institutional review board (IRB). An IRB ensures the trial is executed ethically and that patient rights remain protected. Consequently, the risk associated with participating in a clinical trial is often outweighed by the potential benefits (such as curing a patient’s cancer). 

Some clinical trials are also monitored by data monitoring committees or data safety boards. In addition, various government agencies – including the Office of Human Subjects Research Protections and the FDA – have the authority to oversee the fair and ethical treatment of clinical trial participants.

If you decide to participate in a clinical trial, the lead researcher will explain the details about the trial. Next, you will complete a form indicating your desire to join and awareness of your rights. This is called informed consent, and it’s intended to protect participants.

The researcher should provide enough information for you to understand the risks, potential benefits, and possible alternative treatments to the clinical trial. Regardless, signing an informed consent document is not a contract. Clinical trial participants may withdraw at any time, even if the trial is not yet over.

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Questions to Ask Before Consenting to a Clinical Trial

You should always feel free to ask questions before, during, and after the clinical trial, whether or not you have already signed a consent form. Below is a list of questions to ask to help you learn more about participating in a mesothelioma clinical trial:

  • What is the goal of the study?
  • Has this treatment been tested on humans before?
  • Why do researchers believe this treatment will work?
  • What are the possible procedures and medications that I could receive during the trial?
  • What determines which treatments I receive (for example, are they given to patients at random)?
  • Who will know which treatment I receive during the trial? Will I know? Will members of the research team know?
  • What are my responsibilities during treatment?
  • How long will the clinical trial last?
  • What tests and procedures are involved?
  • How do the risks, possible side effects, and benefits of this trial compare with those of my current treatment?
  • How often will I have to visit the hospital or clinic?
  • Will hospitalization be required at any time during treatment?
  • Who will pay for my medical care and participation in the trial?
  • Will I be reimbursed for other expenses, such as travel and accommodations?
  • If I benefit from the treatment, how do I get it after the trial ends?
  • Who is in charge of my medical care while I am participating in the trial?
  • What type of long-term follow-up care is available?
  • Will the results of the study be given to me?
  • What are my options if I am injured during the study?

Despite the potential risks, clinical trials can greatly improve a patient’s quality of life, especially for mesothelioma patients who haven’t responded favorably to standard first-line treatments and medications. Clinical trials can also lead to major breakthroughs in diagnostic procedures and emerging treatments for mesothelioma patients in the future (such as in the rise of the use of immunotherapy drugs).

What If I’m Not Eligible for a Clinical Trial?

Sometimes, a patient is not eligible to participate in a clinical trial but might still benefit from the treatment. Although drugs that are not yet approved by the FDA are usually only available to clinical trial participants, there are some exceptions.

Compassionate drug use is the use of new, unapproved medications to treat seriously ill patients when no other treatments are available. These patients may live too far from available clinical trial locations or they may not have been eligible to enter.

Talk with your mesothelioma specialist about whether compassionate use might be an option for you.

Current and Ongoing Mesothelioma Clinical Trials

Medical research is a vast field that is constantly changing. Oncologists (doctors who study cancer) are continuously looking for new ways to cure, treat, and prevent cancers. Recently, targeted therapy treatments have expanded the available methods in managing malignant mesotheliomas for patients with advanced forms of cancer.
Below is a list of ongoing mesothelioma clinical trials.

Clinical Trial Name


Cancer Type

Nivolumab and Ipilimumab in Treating Patients With Rare Tumors II Epithelial cell tumors, squamous cell carcinomas, and adenocarcinomas of the respiratory tract
Testing the Addition of Targeted Radiation Therapy to Surgery and the Usual Chemotherapy Treatment (Pemetrexed and Cisplatin [or Carboplatin]) III Stage I to IIIA malignant pleural mesotheliomas
Surgery, Chemotherapy, and Intensity Modulated Radiation Therapy II Stage I to III pleural mesotheliomas
Pevonedistat with or without Pemetrexed Disodium and Cisplatin I/II Epithelioid, sarcomatoid, or mixed-type malignant pleural or peritoneal mesothelioma
Nivolumab and Combination Chemotherapy before Surgery I Malignant pleural mesothelioma

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