• Participation in a clinical trial can be the best first course of treatment for leukemia, even shortly after diagnosis.
• You can leave a clinical study at any time and for any reason.
• Many clinical studies for leukemia involve the standard treatment for that type of leukemia, while some may involve additional treatments.
• Your safety and right to privacy as a participant in a clinical trial are protected by many processes and layers of review.

Misconceptions about clinical trials — research studies testing the effectiveness of new treatments — are common. Many people with leukemia never consider participating in a clinical trial study and thus may miss out on opportunities that could present the most effective leukemia treatment option for their condition.

MyLeukemiaTeam spoke with Dr. Hetty Carraway to get answers to MyLeukemiaTeam members’ top questions about participation in clinical trials. Dr. Carraway is board-certified in medical oncology and internal medicine. She is a staff associate professor of medicine at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University and vice chair of strategy and enterprise development at the Taussig Cancer Institute in the Division of Hematologic Oncology and Blood Disorders at the Cleveland Clinic. Dr. Carraway specializes in the treatment of leukemias, including acute lymphoblastic leukemia; acute myeloid leukemia; chronic myeloid leukemia; and chronic myelomonocytic leukemia.

What would you say is the most common reason people don’t participate in cancer research?

I think people are reluctant because they’re not really sure what they’re signing up for. Whenever things are unclear or we’re unsure, we tend to hedge on being conservative and say, “I won’t do it because I’m not 100 percent sure.”

What are my rights as a participant in a clinical trial? How are my rights protected?

If at any point you are uncomfortable or not confident about the study, you have the right to leave it. A lot of people don’t realize that they can comfortably say no, even after signing up for a clinical trial. It’s important to know that you can say no at any point.

Patients in a clinical trial are cared for in a very deliberate manner. For clinical trials to open, they have to go through an intensive vetting process for patient safety. Clinical trials will not be open if they’re unsafe for patients. The entire program is evaluated for how patient safety is being monitored. There is a group of physicians and other caregivers that evaluate the clinical trial and make sure that it’s safe. There are patient advocates that read through the study and talk with a principal investigator before a trial opens at any institution. The process to get a clinical trial open is not an easy one.

When patients are enrolled in clinical trials, their identity is protected. All the information is deidentified. Patients’ names are not associated with their data or outcomes as they’re followed in the study. There’s a huge amount of work behind the scenes to ensure that information remains private and protected.

Would I be given a treatment that is totally untested, with no information about its potential risks or benefits?

That should never happen. For patients to enroll in clinical trials, they need to be informed about the risks and benefits involved. During a process called informed consent, it will be explained what the clinical trial is about, what drug or treatment is a part of that study, what’s going to be happening, what information or test results are going to be collected, and what the potential risks and benefits are to enrolling in this study.

All of that information should be carefully explained to every patient that is considering the trial. You should have a chance to ask any questions. Then typically the patient is given some time, whether it’s a week or a few days, to think about whether they want to sign up. You need time to process that information before making a decision.

Is there a chance I’ll be given a placebo (sugar pill or sham drug) instead of an active treatment for my leukemia?

I love this question. I think a lot of the assumptions about clinical trials are that patients are going to be getting a placebo. It’s rare that patients are getting a placebo only. It does happen, but you will be aware that it’s a possibility. It really depends on the type of clinical trial that a patient is enrolled in.

There are several phases of clinical trials. In phase 3 studies, we are trying to improve the standard of care because we think we have an agent that’s better than the current standard of care. We’re trying to compare one versus the other to get a drug to the FDA [U.S. Food and Drug Administration] for consideration for approval.

With any phase of a clinical trial, it’s possible that there’s a randomization where patients are given a placebo. It may be that some patients get the standard of care plus the placebo drug. But for any clinical trial, if there is a placebo arm, it should be very clear to you that there’s a possibility that you are going to be receiving a placebo. As you consider a clinical trial, you can ask about the possibility of receiving a placebo.

Are clinical trials only used as a last resort, when there are no other options for leukemia treatment?

That is just not true. Currently for people diagnosed with leukemia, we often lead with recommending clinical trials for an initial treatment, in addition to the standard of care, because of the exciting new options we have.

If leukemia should recur, or relapse, that is also a space where we have clinical trials. For instance, if patients are wanting to pursue a stem cell transplant. Clinical trials are not just for the last resort. Clinical trials are really part and parcel of much leukemia therapy.

Do I have to pay for treatment if I participate in a clinical trial for leukemia?

It’s pretty uncommon for patients to incur costs by going through a clinical trial. That is part of the vetting process — we work hard to make sure that patients aren’t incurring costs by doing clinical trials. We often need to get insurance approval before people can enroll in a clinical trial. Questions around cost are important for patients to ask during the informed consent process.

If I don’t live near a research institution, is it still possible I may be able to participate in a clinical trial?

Yes, that can be possible. It’s important to touch base with your physician and ask. At our institution we have clinical trials that are done at our main campus, but also those that are done in our regional satellite sites. Patients don’t have to live close by. Some of it depends on the kind of research that’s being done. Some of the clinical trials are done in regional areas because it’s easier to find participants — patients like to be treated closer to home.

If the treatment in a clinical trial is effective in treating my leukemia, will I lose access to it once the trial is over?

Most often, if patients are benefiting from the therapy, they’re able to stay on it, unless it’s specified in the consent form for the study. There are some clinical trials that say this duration of therapy is only for X number of months, or a year. And then after that period of time, it stops. For many patients, if that is the scenario, the physician often works with the company and tries to get compassionate use [expanded access] to the drug.

If the study leads to FDA approval of these agents, you may need to pursue getting the drug through the insurance company. Your care team on the study should help you navigate that.

If the treatment in a clinical trial is not effective and my condition worsens, will my treatment be switched?

It’s important for patients to know that if you feel like you’re not benefiting from a drug and you want to stop that therapy, whether it’s the standard of care or an experimental drug, you have the opportunity to speak up and say, “I think we need to change course and pivot to something else.”

It can lead to some reluctance to participate in clinical trials, if people feel like they need to stay on a study even if they’re not benefitting. That’s just not true. We don’t do that with our standard of care, and we don’t do that with our clinical trials. It’s really up to you as the patient and the physician that’s helping you.

How long do clinical studies last?

Every study is a little bit different. There’s not an average length because it will depend on the therapy that’s being given, how long the patient is being followed, and what the end points of the study are — are we looking at response to the drug, or are we looking at overall survival? So there’s a lot of variation.

Will I still be able to see my current doctors if I participate in a research study?

In most institutions, you won’t change doctors if you’re enrolling in a clinical trial. Each institution could be different, so that’s a very important question to ask during informed consent. At our institution, we have research teams that are different from our standard-of-care staff for leukemia. That’s not the case of every institution, so it’s important to ask, “Will my nurse be the same? Will my doctor be the same?”

How do I find out if I am eligible to participate in any clinical trials for leukemia?

Your physician usually works together with you to look at the eligibility criteria for studies. If there is more than one study that you’re eligible to participate in, they’ll help you with the challenge of deciding which study to consider. Your physician, the nursing staff, the entire care team can help make those decisions with you. It’s a shared decision as to whether or not a clinical trial is the right fit for you as your next therapy.

Learn More About Leukemia Treatment Options

Clinical trials lead to treatment advances that can benefit others living with leukemia. Read more about newer leukemia treatment options, including immunotherapy for acute lymphoblastic leukemia and treatment advances for myelodysplastic syndrome.

Talk With Others Who Understand

On MyLeukemiaTeam, the social network for people with leukemia and their loved ones, more than 11,000 members come together to ask questions, give advice, and share their stories with those who understand life with leukemia.

Have you considered participating in clinical trials? Share your thoughts in the comments below, or start a conversation with others on MyLeukemiaTeam.