Clinical Trials

Clinical trials. If you’re a frequent visitor to our website or social media sites, or if you receive our monthly e-newsletter, you hear about them all the time. But what exactly are they? What is their purpose? And why is the MMRF involved in so many?

Clinical trials are studies designed to evaluate new drug treatments. They identify which treatment choices are better for multiple myeloma patients and help the myeloma community understand multiple myeloma better. A great deal of progress in myeloma patient care has been made in the last 10 years. Survival rates have nearly doubled in that time, mainly because clinical trials have led to the development of treatments that are safe and effective, that produce fewer side effects, and that lengthen lives.

The results of clinical trials have enabled the Food and Drug Administration (FDA) to approve new treatments that are safe, effective, and shown to be better than older treatments. Remarkably, eleven new myeloma drugs have been approved by the FDA since 2003. All of these drugs, at one point or another, were studied on a clinical trial, and many had the support of the MMRF!

Patients who enroll in clinical trials have the opportunity to be among the first to receive the newest drugs and therapies in development. However, it is important to understand that new treatments may be equivalent to, more effective than, or not as effective as existing treatments. They may also have unexpected side effects. That’s why new treatments must be studied in clinical trials first.

Because clinical trial participation is a good option for many patients, it is important to understand what they are, how they work, and how to find a trial that is right for you.

Before a drug is considered for testing in people, it needs to show evidence of activity against myeloma in laboratory and animal studies—these are called preclinical studies.

When a drug is ready to be tested in patients, researchers carefully plan one or more clinical trials. Some of the considerations that go into designing a clinical trial are what dose of drug will be used, how many patients will be enrolled, and how and how often they will be monitored.

In a clinical trial, participants receive treatments according to a predefined research plan (called a protocol) created by investigators to determine the safety and effectiveness of a new treatment. Clinical trials take place at cancer centers, hospitals, clinics, or doctors’ offices. Before a patient enrolls, all details of the treatment are explained and the patient must consent to participate. Patients who agree to participate in a clinical trial are free to withdraw at any time.

Types of Clinical Trials

There are different types of clinical trials, and each type serves a distinct purpose. The different types of clinical trials are described below.

The main objective of phase 1 trials is to determine the best dose and schedule of a drug. Instead of focusing on how effective the treatment is against myeloma, phase 1 trials attempt to figure out a dose that is well tolerated. Patients in phase 1 trials can expect to give a lot of blood samples, as blood tests are needed to determine the levels of the drug in the bloodstream and to see how quickly the drug clears. These trials study only one drug and have only a single arm—that is, all the patients in the study are in the same group, receiving the same drug and undergoing the same tests. Phase 1 trials do not require very many patients to be enrolled to obtain useful results.

Phase 2 trials, which are conducted only after an optimal dose is identified in phase 1 trials, determine the effectiveness and safety of a new drug. Effectiveness is measured by how many patients respond, how long responses last, and whether survival is improved. These trials can look at a single drug in a single arm, or they can assign the participants into different groups and look at two different drugs. The number of patients enrolled in a phase 2 trial is around 100 to 200.

Phase 3 trials are the studies that definitively determine a drug’s efficacy and safety. These studies, when successful, lead to the approval of the drug being studied. Most drugs in phase 3 trials are compared to a standard of care—that is, the current recommended treatment—so patients enrolling in a phase 3 trial may receive either the existing standard of care or the new treatment. To obtain useful results, phase 3 trials require a lot of patients to participate, usually 200 or more.

Because clinical trials tend to be very expensive, new types of studies have been developed that allow researchers to study several treatments at once and to individualize treatments to patients based on their specific genetic blueprint. This approach allows for the careful selection of patients and lessens the need to enroll hundreds of patients. Two of these newer study designs are called umbrella (or platform) and basket (or bucket) trials. The MMRF’s MyDRUG trial, the first ever multiple myeloma platform trial, was described in a recent blog post.   

Other types of clinical trials include expanded access trials, in which access to a drug that has been found to be active but is not yet approved by the FDA is provided by its manufacturer. These trials get new drugs to people that need them as quickly as possible.

Participants in longitudinal and registry trials (also called observational trials) are monitored for periods of time to observe any changes in their health. One example of a longitudinal trial is the MMRF CoMMpass Study, in which myeloma patients were monitored for 8 years. In this trial, participants provided bone marrow samples at key points over the course of their disease, such as at diagnosis, at a response to initial treatment, and at relapse. In the Promise Study—an example of a registry trial—approximately 50,000 individuals at risk for myeloma precursors will be screened and then followed with the aim of identifying molecular markers for progression to active myeloma.


How to Enroll in a Clinical Trial

There are hundreds of ongoing clinical trials investigating new drugs, new modes of administration for existing drugs, and new drug combinations in patients with multiple myeloma. Many trials are being conducted by the Multiple Myeloma Research Consortium. There is really no reason not to consider enrolling in a clinical trial at any point in the course of your disease. Just by looking at clinical trials as a potential option in your care, you are one step closer to being treated with drugs with the potential to benefit you and to help other patients like you.

So how do you go about this? First and foremost, talk to your doctor. You need to find out if clinical trials are offered at your doctor’s practice. Talk to your doctor about your eligibility for a trial. Your doctor should explain a trial and how it works.

Patients eligible for participation should meet with a research nurse and other personnel to learn more about the trial. They can explain what to expect on the trial—the benefits and risks—and can answer questions about tests you would undergo (for example, blood draws, bone marrow biopsies, or imaging), what additional medications you may have to take, and what’s paid for (and what isn’t).  

It is extremely important for patients planning on participating in a clinical trial to carefully review the informed consent paperwork, which will describe potential side effects of the drug being studied, what you would need to do if you decided to withdraw from the study (which you are free to do at any time), and other important details.

Ultimately, clinical trials are essential to the development of new myeloma treatments, providing new therapeutic options for myeloma patients at all stages of the disease. The greater the number of people there are enrolling in clinical trials, the faster new treatments can be made available to patients. It is only through patient participation in clinical trials that we have achieved the high number and various types of myeloma treatments available today.

To search for a clinical trial near you, you can use our Clinical Trial Finder. Or, if you would like to learn more about clinical trials—or have questions about joining one—you can contact a Nurse Patient Navigator at the MMRF Patient  Navigation Center at 888-841-6673.


Additional Resources

MMRF. Clinical trial finder.  

MMRF. Clinical trials and experimental therapies.

MMRF. Participate in clinical trial.

MMRF. 2018 Patient Summits.

MMRF. 2019 Patient Webinars. Innovative clinical trials.