Clinical Trials 101

What are clinical trials?

In the most basic sense, a clinical trial is a way for researchers to test and develop new treatments for patients. This could mean a treatment that is better at killing cancer cells or one that creates fewer side effects.

Clinical trials are an important step in developing new cancer treatments. Without clinical trials, all the new treatments for multiple myeloma patients that have been developed over the past 15 years wouldn't exist.

Why are clinical trials important?

Clinical trials help researchers, scientists and doctors develop new treatments that help patients with Multiple Myeloma live longer with fewer side effects. Each clinical trial attempts to answer a specific question about the new treatment being studied. Some examples of questions that a clinical trial may be used to answer are:

  • Does this new therapy work better than the current treatments that are available?
  • Does this new therapy create fewer side effects?
  • What is the best way to combine drugs so that we can maximize the impact while minimizing the side effects?
  • Can we stretch the dosing schedule or decrease the dosage of treatments?
Will I receive a placebo?

Placebos are very uncommon in cancer clinical trials. Even so, when they are used, you will never receive a placebo by itself. It's unethical. The only way that placebos are used in cancer clinical trials is in combination with known therapies that have been proven to be effective against your cancer.

An example of when a placebo would be used is to help researchers and doctors determine if a new drug is more effective or provides less side effects than a standard therapy.

I still have treatment options, are clinical trials a last resort?

No. Clinical trials are always an option for you. Whether you are newly diagnosed or on your fifth relapse, clinical trials should be considered as an option at every step of the way.

Am I just a 'guinea pig'?

Absolutely not. You are in no way, shape or form a 'guinea pig.' Every patient who participates in a clinical trial is first and foremost treated as a human being who is receiving treatment for their cancer.

What if I change my mind and no longer wish to participate in a clinical trial?

No problem. You can leave the study and stop participating at any point in time. You should never feel pressured or forced into continuing a clinical trial if you are uncomfortable or unable to continue for any reason. Once you decide to stop participating, speak with your doctors and they will help determine a more appropriate treatment for you.

What if the treatment in the clinical trial doesn't work?

Whether you are participating in a clinical trial or on standard therapy, the same thing will happen if you are not responding to treatment. Your doctor is continually tracking your myeloma's response to treatment. In the event that your myeloma is getting worse, your treatment will change.

Will my insurance cover my clinical trial?

There are as many answers to this question as there are insurance policies and providers. The best thing to do is to call your insurance company and the have the conversation about whether or not clinical trials are covered and to what extent. Regardless of your treatment, it is a good idea to stay in close contact with your insurance company to understand what is covered and what you are responsible for. You can also work with a trial nurse or a financial counselor at your hospital for help in determining costs associated with cancer treatment.

How are new drugs developed?

The U.S. Food and Drug Administration (FDA) is a government agency whose job it is to make sure medical treatments are safe and effective for people. Clinical trials are used by researchers to prove to the FDA that the new, experimental treatment being tested is safe and effective. If the results from the clinical trials are positive, the FDA will grant approval for the experimental treatment to become standard-of-care. The treatment will then be widely available to patients.

It is a long process for a new treatment to go from being discovered, through clinical trials, to finally being approved as a new treatment option. On average, researchers will begin with thousands of compounds. It will then take somewhere between 12 to 15 years for researchers to develop one new treatment that will receive FDA approval.

What are the phases of a clinical trial?

Clinical trials are set up to progress through three main phases. Each phase is designed to answer a specific question as researchers determine if the new drug is safe and effective for patients.

Phase 1

Is the treatment safe?

This is the foundation of every phase of a clinical trial. A patient's safety is the first thing that researchers and scientists consider.

Phase 1 clinical trials help researchers determine the safest dose for the treatment (this is also known as the maximum tolerated dose). This is the highest dose of the treatment that is safe for the patient to receive. These trials typically involve a small number of patients (between 10-20).

Phase 2

Does the treatment work?

Just like in a phase 1 trial, phase 2 clinical trials help researchers determine that the experimental treatment is safe but they are now also looking to determine if the treatment works against the specific type of cancer. This phase still involves a small number of patients (typically less than 100).

Phase 3

Does the treatment work better than the current standard of care?

As well as continuing to determine if the treatment is safe and if it works, researchers are now determining if the experimental treatment works better than the current standard of care. Hundreds or thousands of patients may be involved in this phase of a clinical trial. This is the last phase before the treatment goes to the FDA for approval.

FDA Approval

New Standard of Care

In order to receive approval from the Food and Drug Administration (FDA), all new treatments must go through the clinical trial phases to prove that:

  1. It is safe,
  2. It works against the specific type of cancer, and
  3. It works better than the current standard of care.

The FDA will then review the information from the prior phases of its clinical trials and approve or deny it as a new standard of care.

Am I eligible for a clinical trial?

All clinical trials have guidelines (or criteria) about who can participate. Any patient who wants to take part in a trial must meet the criteria. Common criteria may include:

  • A specific diagnosis,
  • The status of your Myeloma,
  • Previous treatments that a patient has had,
  • The period of time since a patient last received treatment,
  • Results from specific lab tests,
  • Current mediciations a patient is taking
  • A patient's age or sex
Are clinical trials safe?

Several levels of safeguards are in place to help protect the patients who participate in clinical trials. While there are risks with any study and any treatment, there are safeguards in place to reduce this risk as much as possible.

Informed Consent

In order to join a trial, each patient must first read and sign an informed consent document. This document explains:

  • Your rights (including the right to leave the trial at any time for any reason),
  • The purpose of the trial,
  • The procedures involved,
  • A guarantee that your information will remain confidential,
  • And, the risks and benefits of participating.

The Food and Drug Administration (FDA)

The FDA's job is to ensure that medical treatments are safe and effective for patients to use. The FDA monitors most clinical trials to protect the rights of patients and to ensure that the trial is being run according to plan.


Institutional Review Board (IRB)

Each hospital or site that participates in a trial has an IRB that reviews, monitors and approves the plan for the clinical trial. The IRB is typcially made up of healthcare professionals, clergy members and other individuals from within the community The role of the IRB is to:

  • Review the plan for the trial to ensure patient's safety,
  • Continues to review the trial on an ongoing basis,
  • Stop a trial if it appears to cause unexpected side effects for patients or if there is clear evidence that the new treatment is causing harm to patients.

Data Safety Monitoring Board (DSMB)

The DSMB is an independent committee made up of statisticians, physicians and other scientists. The role of the DSMB is to ensure the safety of patients and to review all trial results. If patients experience unexpected or severe side effects, the DSMB may stop the trial.

What questions should I ask my doctor about clinical trials?

When thinking about enrolling in a clinical trial, it is important to understand all of your treatment options so that you can have an informed discussion with your doctor. And, even more importantly, remember that it is your choice. The choice to be treated with standard therapy or on a clinical trial belongs to you. General knowledge of your treatment and trial options help you be your own best advocate as you consider your options and help you to feel comfortable when making your choice.

The following are some questions to consider when discussing clinical trials with your doctor.

  • What treatment options are available?
  • Within each of these treatment options, what is the risk/benefit of each (including side effects)?
  • Are there clinical trial options available?
    • What phase is the clinical trial?
    • What is the purpose of the trial?
    • How many people have taken this new drug or therapy?
  • What is expected of me on standard treatment or the clinical trial?
    • How many office visits will I have?
    • What tests are required of me?
    • How is the drug administered: in pill form on through an IV?
What is an NCT number?

An NCT Number is a unique code given to every clinical trial that is registered through the National Institutes of Health (and provided through the National Library of Medicine). It follows a format of "NCT" followed by an 8-digit number. This number may be used by hospitals to help identify a specific trial.

Find Eligible Clinical Trials for Your Myeloma