The Most Commonly Asked Questions About Clinical Trials

1. What exactly is the goal of a clinical trial?

The usual goal of a clinical trial is to answer a specific question about a certain medical plan. Many women think of clinical trials as an option only after other treatments have failed. Clinical trials exist for women in this situation, but many equally important trials are available for women earlier in their fight against cancer. Researchers may call the medical plan to be tested an “intervention.” The intervention can be anything, such as the types of care in this list.

  • A new test
  • A new drug
  • A new kind of treatment
  • A new procedure or a new method, such as screening for a cancer

The researchers for each trial set eligibility, or enrollment, requirements. These are a list of guidelines that each patient must meet to be part of the trial. The goal of the guidelines is to find patients who will best help answer the questions researchers are asking. For instance, only patients in a certain age group or who have or haven’t had certain treatments before may join.

2. What does is mean if the clinical trial is phase 1, phase 2, or phase 3?

Each phase represents a progression that starts with testing a new intervention in humans and ends with proving that the intervention is the best. If it is the best, it becomes part of the new standard of care for that cancer.

The various phases of a clinical trial also affect these 2 issues.

  1. The scientific questions being asked
  2. The number and type of patients who can be part of the trial

Phase 1 Clinical Trials. A phase 1 clinical trial generally involves a new product or drug that has shown some promise in animal studies. Phase 1 trials test the new product in humans.

The goal of these trials is to answer the questions: “What is the right dose and what is the right way to use the drug in future trials?”

Sometimes, phase 1 cancer trials accept patients who don’t have the same type of cancer. That’s because the goal of these trials is to learn how to use the new drug in humans. Although some study participants may benefit from the new drug, the study’s goal is not to measure how well the drug treats a specific cancer.

The size of these trials is generally small, including 12 to 20 patients. Because of the small group, these trials are usually done in a single medical center or institution.

The process of phase 1 trials usually follows this pattern. Small groups of 3 to 6 patients are enrolled at a time. Each person gets a specified dose of the new drug. And, of course, they are closely checked for bad side effects.

The next small group of patients gets treated with a higher (or lower) dose of the medication based on what researchers learned from the first group.

Because of this type of enrollment, the trials may go through times of being open or closed for enrollment. That means even though your center may be doing the trial, there could be a period of time that the trial is temporarily closed for enrollment. This can be frustrating for those eager to join, but because doses are being checked, limited, timed enrollment is all about safety.

Once the proper dose of the new drug is found, researchers recommend that the drug move into the second phase of study, or a phase 2 clinical trial.

Phase 2 Clinical Trials. These trials are designed to learn how well an intervention (which can be a drug, more than one drug, a test, or a process) works when used for a specific disease.

The goal of these trials for cancer is to answer the question, “Is this intervention effective in treating the cancer and what are its side effects?”

The size of these trials is generally larger than phase 1 trials, including 20 to 100 patients. They also have more specific or tighter eligibility rules.

The process of phase 2 trials usually follows this pattern. Enough patients are enrolled to meet these 2 goals.

  1. To safely evaluate whether the drug or product can be tolerated at the dose and schedule recommended
  2. Whether the drug or product has enough benefits to compare it against a standard treatment, which are the treatments usually used for the same type of cases

Most patients who enroll in phase 2 trials have the same kind of cancer. And they’ve had roughly the same amount of treatment for the cancer before joining the trial. These trials may be available in more than one medical center across the country.

Phase 3 Clinical Trials. These trials are designed to test a new product or drug against a benchmark, or standard treatment.

The size of these is the largest of the 3 phases of trials, including more than 100 patients, and in some cases more than 1000.

The process of phase 3 trials usually follows this pattern. There are at least 2 types of treatments being tested. The type of treatment a patient gets is selected randomly. That means neither the patient nor his or her doctor can pick the treatment. This random selection allows for an honest comparison to be made between the tested treatments.

Since the differences between 2 or more drugs may be small, many patients may be needed to accurately tell if there is a difference between the tested treatments. The required numbers mean phase 3 trials are often available at many medical centers across the country. Sometimes they are even available in other countries. They often take many years to complete.

The “winners” from phase 3 trials sometimes go to the U.S. Food and Drug Administration to be evaluated for FDA approval for use in the disease studied.

3. What are the benefits of being in a clinical trial?

These are some of the many benefits of joining a clinical trial.

  • Patients have a chance to help themselves and help others who have the same type of cancer.
  • Many new treatments are from the National Cancer Institute, pharmaceutical companies, or other scientists. And they are only available in clinical trials.
  • In general, participants get the new drugs, tests, or processes at no cost to them, although they may still have to pay for some parts of their care.
  • Many trials give additional testing and use intense safety checks. Plus, patients are closely checked in follow-up visits during and after the trial.

4. What are the risks of being in a clinical trial?

The risks of being in a clinical trial depend on the phase of the trial and the drug or treatments being studied.

In phase 1 clinical trials, the proper dose has not yet been established. And there has been limited previous human exposure to the new treatment or test. To respond to these risks, those trials have many safety-nets built in. Their process may be changed more than once in response to the side effects researchers see.

For any trial you are in, you will be told about all the possible risks and side effects during the informed consent process. As you can imagine, this list can be long. Still, the researchers will make sure you understand how likely these effects are. Plus, if anyone in the trial has new or unexpected side effects, every participant—and future ones—are told about it. Finally, if unexpected bad side effects are seen, the trial will be stopped.

5. Why are the criteria to be part of a clinical trial so strict?

The criteria for joining a clinical trial are strict for 2 main reasons.

  1. To protect you
  2. To most effectively answer the scientific question, or goal, of the trial

People may have side effects from any treatment. It’s important for researchers to be sure that any side effects are from the treatment being tested, and not from something else. For them to know that, they may exclude these people.

  • Those with certain illness or co-existing medical conditions, since these may cause their own set of symptoms and side effects
  • Those who have had, or who are still getting, certain treatments, since they may cause side effects that could be confused with the treatment being tested

Certain situations may increase the risk for a complication to the new drug. When this is suspected, researchers make a list of specific people to exclude so that the drug doesn’t hurt someone unnecessarily.

Depending on the phase and goal of the trial, certain patients may not be able to join because the new test or treatment is not known to be of benefit to them. For instance, women with uterine cancer may not be able to join a drug study for women with ovarian cancer.

6. What is a randomized clinical trial?

A randomized clinical trial is a trial where the drug or treatment is assigned to the patient by a random, or chance, method (like flipping a coin).

Each choice a patient has for treatment is called an “arm.” Usually, there is a standard treatment that makes up the “control arm.” And, the new drug or treatment is the other arm. Occasionally, there is a “no-treatment arm.” In most situations, this is a standard arm. When a no-treatment arm is part of a clinical trial, it usually means that normally, no additional therapy is used for people with the condition.

Patients are randomly selected for each arm of the trial. If there are 2 arms in the study and each arm has the same number of patients, each participant has an equal chance of being treated with the new drug or the standard (control) drug.

Sometimes you and your doctor know what treatment you’re getting. Other times one of you may know. And sometimes neither of you knows. The last case is called a “blinded” trial. The goal of a blind trial is to keep factors outside the study’s goals from interfering with the interpretation of the results.

7. How do I find out about clinical trials that may be right for me?

Here are several ways you can find out which clinical trials are occurring and if you are a candidate.

8. Why doesn’t my doctor know about clinical trials?

There are many clinical trials in medical centers all over the world. It is almost impossible for any doctor to know all the trials that are recruiting patients with your type of cancer. If you are interested in joining a trial, follow one of these steps.

  • Ask your doctor to look for specific trials for you.
  • Ask your doctor to refer you to a medical center that often does clinical trials so you can get another opinion.

If you have a gynecologic cancer, call (800) 225-3053 to ask about specific, open clinical trials.

9. What is a cooperative group clinical trial?

Cooperative groups are large organizations of healthcare professionals. They share the goal of being interested in running clinical trials for patients with specific diseases. Their large structure means more than one trial can be run at the same time. These groups usually have several teams that design, develop, and run clinical trials. Their intent is to recruit patients over a large geographic area from diverse backgrounds. That means a trial that opens in a cooperative group may be opened at many medical centers or offices. The groups that run these trials are experts. They make sure the trials are safe. And they set up trials to efficiently answer the specific scientific questions posed at the start of a trial. Often times, the results of these trials change how healthcare experts take care of people with cancer.

10. Why are some trials done in only one or two medical centers?

Regulations are strict about where trials take place to keep patients safe. These are some of many reasons to limit the number of medical centers that offer a clinical trial.

  • Only a few centers may have the needed experts to run the trial
  • The trial may need only a small number of patients
  • Only a few centers may have the investigative new drug application (IND) from the government

If only a few centers offer a clinical trial, patients may have to travel to get the drug or treatment being tested.

11. Why can’t I get a new drug without being in a clinical trial?

The simple answer to this question is safety. The clinical trial process requires doctors and healthcare professionals to closely monitor patients and keep detailed records. These log certain effects people have from taking the treatment. These data are sent to the study’s sponsor. The FDA also gets the data to determine if the trial is safe to continue. Some trials require this information for each patient be sent with every treatment so that important results are found as quickly as possible.

12. Am I a “guinea pig” if I’m in a clinical trial?

This is an unfortunate concept in the clinical trial world because it no doubt negatively affects potential participants. While a study is, in a sense, an experiment, it is not a laboratory or animal experiment. The connotation of “guinea pig” makes one think that there is no benefit to the patient in the trial and no effect except bad ones.

Here are a few things for you to keep in mind. Clinical trials have specific goals, and they are under peer review. That means experts besides the researchers believe there is a potential benefit to patients who join. It also means there is close oversight to ensure safety. It is specifically from past clinical trials that today’s progress was made. Tomorrow’s therapy depends on today’s clinical trials. We can’t try to cure cancer without this process!

13. Can I be in more than one clinical trial?

Sure. Whether you are eligible depends on the criteria set for each trial. In some cases, the treatment you had in a prior trial may exclude you from being in a later one using the same or a related treatment. However, being in one clinical trial will not necessarily keep you from being in another one later.