Clinical trials are essential for delivering new and better cancer treatments to patients. As a worldwide leader in cancer clinical trials, MD Anderson is driving the field of cancer medicine forward while also prioritizing the safety of our patients. Participating in a clinical trial can provide patients access to innovative therapies not otherwise available and provide physicians valuable information to help future patients. Funda Meric-Bernstam, M.D., chair of Investigational Cancer Therapeutics, describes the importance of clinical trials and what patients can expect. Request an appointment at MD Anderson by calling 1-877-632-6789 or online: https://my.mdanderson.org/requestappointment
As part of our mission to eliminate cancer, MD Anderson researchers conduct hundreds of clinical trials to test new treatments for both common and rare cancers. Look through our database to find studies for which you may be eligible.
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What are clinical trials?
Clinical trials are research studies in which patients may volunteer to take part. MD Anderson uses clinical trials to find better ways to prevent, diagnose and treat cancer. Doctors use treatment trials to learn more about how to fight cancer. This guide is for patients who may join a treatment trial.
Clinical trials are part of a long, careful process, which may take many years. First, doctors study a new treatment in the lab. Then they often study the treatment in animals. If a new treatment shows promise, doctors then test the treatment in people. Doctors do this in three to four steps, or phases. Your doctor may offer you a clinical trial as a treatment option.
Is a trial right for you? MD Anderson's clinical trials booklet contains a decision guide along with information on the clinical trials process.
How am I protected?
MD Anderson’s most important job is to protect patients. First, MD Anderson protects patients in clinical trials by following well-planned protocols.
A protocol:
- Explains the treatment plan
- Lists the medical tests patients will receive
- Gives the number of how many patients will take part in the clinical trial
- Lists eligibility criteria, which are guidelines to decide who may join the clinical trial
- Explains safety information
Second, MD Anderson protects patients by using a careful informed consent process.
Third, our Institutional Review Boards (IRBs) protect patients by reviewing protocols and monitoring trials. The IRBs are committees of doctors, nurses, chaplains, social workers, lawyers and patients. They make sure that trials follow federal laws and that patients are protected.
The U.S. Food and Drug Administration (FDA) audits the IRBs’ files. Also, FDA officials may visit MD Anderson at any time and review anything they choose related to clinical trials.
Did You Know?
In 2021, the FDA approved 48 new cancer drugs for public, clinical use. MD Anderson contributed to the development of 29 of those — more than 60 percent.
Clinical trials are research studies that evaluate a new drug or treatment to make sure that it is safe and effective for patients.
“Clinical trials are essential to be able to identify better treatments for patients with all tumor types,” says Funda Meric-Bernstam, M.D., chair of Investigational Cancer Therapeutics. “It’s also an opportunity for patients to access otherwise unavailable treatments and to contribute to better outcomes for future patients.”
So, how can you find out if a clinical trial is right for you? Use our answers to these common questions about clinical trials to help you have a more informed conversation with your care team.
What kind of clinical trials are there?
Many clinical trials are for treatments tailored specifically for a certain disease or condition to ensure the most efficient approach.
“We have over 1,000 active trials at MD Anderson at any given time, which means there is a very high likelihood of identifying a trial that would be a good fit for a patient,” says Meric-Bernstam.
How do I qualify for a clinical trial?
Clinical trials are available for patients at all stages of disease. You need to meet certain requirements, such as having the appropriate condition or biomarkers which are being examined. Additionally, some drugs have known side effects that could be harmful to certain people. This is why every clinical trial has a protocol with strict inclusion and exclusion criteria that are usually based on:
- specific age ranges
- type of disease (for instance, type of cancer and extent of cancer spread)
- prior treatment history
- chronic health conditions
- pregnancy or intent to become pregnant
- liver or kidney function
- medications you take that might interfere with the trial drug
- other considerations that vary depending on the treatment being tested
These inclusions and exclusions are one of many ways in which clinical trials prioritize your safety. It is important to talk openly and honestly with your care team about your medical history because there may be something about the treatment that might negatively affect your body. In turn, your care team will inform you of any potential risks or known side effects so that you can make a fully informed decision.
“It is very important for us to be open about what is known and what is not known about a treatment so that patients can be informed of their options,” Meric-Bernstam says.
What are the different phases of clinical trials?
For a drug or treatment to be approved for use as standard treatment, it must pass through several clinical trial phases. Each phase has a different purpose.
Phase I clinical trials are usually the first time an experimental drug or a new drug combination is given to a human. During this phase, researchers are trying to figure out the correct dose, or how much of the drug can be given safely. Phase I clinical trials usually only have between 15 to 50 patients, and doctors and nurses are monitoring very closely for any signs that a drug or drug combination might have unexpected side effects.
Phase II clinical trials usually enroll fewer than 100 patients to test if the established dose of the treatment shows signs that it works.
Phase III clinical trials enroll hundreds to thousands of patients to compare the standard treatment to the new experimental treatment to see if it works better than what is currently available. In some Phase III trials, neither the patients nor the doctors know if a participant is receiving the experimental treatment or the standard of care. This is to avoid any potential bias or expectations.
After Phase III clinical trials, the Food and Drug Administration (FDA) reviews the results and decides whether to approve the new treatment. If it gets approved, the drug becomes available for people who weren’t on the trial. The drug can even be tested to see if it’s effective in treating other diseases.
Related: 5 things to know about the FDA approval process
Are clinical trials safe?
While you’re on a clinical trial, a team of doctors and clinical staff will closely monitor you to make sure you are safe at all times. Your care staff will check your lab results constantly to see if the treatment is working as expected and make sure you don’t experience unintended side effects. They also will provide contact information to reach someone who can address your concerns at any time.
Remember that participation in a clinical trial is completely voluntary. You can withdraw your consent at any point and for any reason.
Are clinical trials right for me?
Talk to your care team to find out if you are a good candidate for a clinical trial.
It is important to consider your standard treatment options and the expected results based on your circumstances. Your doctor or specialist is your best resource for understanding all the potential risks and outcomes that may come with a given trial. They can help guide you to make the decision that’s right for you.
Your eligibility for certain phases of trials may vary based on your condition. You should weigh potential risks and side effects as well as additional tests, visits and travel requirements.
Clinical trials are not for everyone, but they can expand your treatment options if you meet the criteria and want to participate. When it comes down to it, your doctor or specialist is the best resource for determining whether or not you would be a good candidate. Their main priority, after all, is making sure you have access to the best possible treatment options that can be safely tailored to your needs.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
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From our Faculty Experts
Phase I clinical trials are the foundation for how we develop new cancer drugs. Typically, they involve only several dozen patients and study a new medicine’s effect on a variety of cancer types.
David S. Hong, M.D., sat down with us to explain more about Phase I clinical trials and answer some of patients’ most frequently asked questions.
What is a Phase I clinical trial?
Phase I clinical trials are the first time human beings are being treated with an experimental drug. It starts with researchers figuring out a pathway to kill cancer. A drug company then develops a medication that mimics that process. The primary purpose of a Phase I is to figure out how much of the drug we can safely give patients and see a benefit.
But that’s not the only purpose. Phase I trials also help us figure out which cancers benefit from these drugs.
Who should consider participating in a Phase I clinical trial?
Phase I clinical trials aren’t for everyone. Most patients who choose Phase I clinical trials are running out of options. Typically, they have metastatic disease, haven’t had success with standard chemotherapy, have mild or no symptoms and don’t want to go to hospice. They’re willing to see if they can help others, and possibly themselves, by participating in a Phase I clinical trial.
What questions do you hear most from patients considering Phase I clinical trials?
“Is there a placebo?” Unlike Phase III clinical trials, there’s usually not a placebo in a Phase I trial. Everyone gets the drug, just at different dose levels.
There are also a lot of questions about schedules. Phase I clinical trials are very intensive and require multiple visits from the patient. Since it’s the first time this drug has been used in human beings, we’re very vigilant.
We also get a lot of questions about side effects. Typical side effects include nausea, vomiting and diarrhea. But side effects are different for each patient in each trial. We try to give patients an overview of the most common side effects, those that are most concerning and then all possible side effects. Most side effects are reversible and treatable. We either stop the drug or we give another medication to address the side effects.
How long will a patient be enrolled in a Phase I clinical trial?
Patients can usually stay on a Phase I clinical trial as long as they benefit. A traditional Phase I clinical trial lasts about two years (from the time we start enrolling to the time that we actually close out a study).
What happens if a patient’s condition doesn’t improve?
If a patient’s clinical condition declines, it may just be because of side effects and we can address those issues. Or, it’s because the tumors are growing. We re-image patients on a regular basis, and we do measurements on the patient following a preset criteria. Depending on the drug and the set criteria, we’ll take the patient off the study if a tumor grows a certain percentage. What happens next depends on how the patient is doing overall. Many patients go on to another Phase I study or back to their medical oncologist because he or she has one more therapy to try. Or, if they are out of options, they may decide to go to hospice.
What are some of the most promising Phase I clinical trials we have at MD Anderson right now?
There’s lots of exciting research happening. In a Phase I trial of the drug LOXO-101, we’ve seen tumors shrink in patients with cancers that are linked to the gene fusion of NTRK. We’ve also seen promising results in a Phase I trial of a combination of inhibitor drugs that target a cancer cell’s signaling. Lastly, we have many immunotherapy Phase I clinical trials, but one that’s exciting is the upcoming MAGE-A10 study on the use of engineered cells of the immune system (called CAR-T cell and T-cell receptor therapies).
What advice do you have for caregivers of patients enrolling in Phase I trials?
My number one piece of advice is don’t push your loved one to do a Phase I. It’s not always the best choice for the patient.
Two, be prepared to support this patient timewise. It’s an intensive and time-consuming process.
Three, ask questions. You need to be informed.
Anything else you want patients to know about Phase I clinical trials?
What we’re seeing in the Phase I environment is a lot of hope. Within the past decade, there’s been an explosion of new drugs. There are over a thousand new cancer drugs in the pipeline. We’re seeing a lot more activity than before; drugs that are actually working in lots of different tumors types than before. We’re also getting much more sophisticated in how we identify patients. I’m an optimist, and I wouldn’t be in this field if I wasn’t.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
Clinical trials are how progress is made in medicine. “They’re really essential to drive our ability to deliver better cancer care,” says Funda Meric-Bernstam, M.D.
Enrollment in a clinical trial is entirely voluntary, but cancer patients can benefit from joining one while also helping future patients. The insights we gain from clinical trials today helps us improve treatment options that will benefit other patients.
Clinical trials are complex, though, and there are a lot of misconceptions surrounding them. Here's what you should know before you join a clinical trial – or decide it’s not for you.
Clinical trials are safe
“Safety is the most important thing,” says Ecaterina Ileana Dumbrava, M.D. Because patients on clinical trials are the first ones to receive experimental medications, there are many safeguards in place to ensure they’re not harmed.
In addition to the routine assessments cancer patients receive for standard treatment, clinical trial participants typically need additional clinical visits, lab work, imaging scans and biopsies.
Also, you’re not just communicating with your oncologist and your cancer care team. Patients on clinical trials also have continuous contact with a research team to ensure they’re doing well on the new drug.
“We’re talking to patients, often weekly, to see if they’re experiencing any side effects and just checking in to see if they have any questions,” Meric-Bernstam says. Your clinical trial team also provides guidance on next steps if the patient does experience any side effects. “There's a lot of help built in with clinical trials to make sure that patients are getting optimal care,” Meric-Bernstam adds.
But the focus on safety doesn’t start there – it happens well before patients are enrolled. “The FDA doesn’t come in just at the end of the trial to approve a drug; they approve the drug before it can be even be administered to patients in the trial,” Dumbrava says.
At MD Anderson, clinical trials are designed by experts and go through several rounds of approval to ensure they’re safe for patients. A proposed clinical trial receives its final approval from the Institutional Review Board (IRB), a committee made up of physicians, nurses, researchers, patients and lawyers.
Clinical trials occur in phases
There are four phases of clinical trials, and each has its own goal. When a new drug or a new drug combination is developed, it's tested in Phase I clinical trials. These usually have a small number of patients, about 15 to 50. “These trials are designed to determine the safety of the drug or drug combination, and what dose to use in following trials,” Meric-Bernstam says. “We’re also looking efficacy, and identifying which patients are the most likely to benefit from the drug, and, therefore, be enrolled in future studies."
Phase II clinical trials may focus on a specific cancer type and examine how it responds to the experimental drug or procedure. These trials may enroll patients with specific diseases or based on certain test results called biomarkers.
Phase III clinical trials test whether a new treatment is better than what’s being used as the standard treatment. At this point, the drug or procedure may go to the FDA for approval, but studies of the drug aren’t done. A fourth phase (Phase IV) reviews the new treatment’s long-term benefits and side effects.
Clinical trials don’t just examine new drugs
Although many clinical trials study new medicines, they help improve all aspects of cancer care. They can study new ways of dispensing treatment, explore a new dosage, test a new drug combination or examine a drug’s success in treating different cancer types.
They also focus on ways to prevent cancer or a recurrence as well as ways to reduce treatment side effects. “We want to save more lives, but we also want to maintain patients’ quality of life,” Dumbrava says.
Clinical trials don’t limit your access to other care
“No matter what the scenario, often the best treatment choice is a clinical trial,” Meric-Bernstam says. She notes that if there’s already an effective treatment approach, clinical trials offer the opportunity to build on that to make it more effective. They usually require patients to receive known effective treatment options before starting something that’s more investigational. If there aren’t clear, effective treatments, clinical trials offer access to something new that may work.
“Unfortunately, we don’t know if an experimental treatment will be effective, but that’s true for some standard treatments, too,” Meric-Bernstam says.
If your disease isn’t controlled on a clinical trial, your doctor will stop your participation and may offer another treatment option. In some cases, you may enroll in another clinical trial.
“By joining a trial, you’ll usually be getting a treatment that we think may be better than our current options,” Meric-Bernstam says.
Dumbrava agrees, adding, “At minimum, you’ll be receiving the best treatment available.”
And even if you personally don’t see clinical benefit from the experimental treatment, your participation still helps advance cancer research.
Clinical trials are for patients at all stages of cancer
Although all clinical trials have criteria for the participants, clinical trials are available to patients at all stages of cancer. The eligibility criteria, which is meant to ensure patients’ safety, may include the patient’s age, gender, cancer type and stage, previous treatments and overall health.
Your doctor is the best resource for knowing your treatment options. But don’t hesitate to ask if there are clinical trials that are available to you.
“It's also an opportunity to pay it forward and truly contribute to better outcomes for future patients,” Meric-Bernstam says.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.