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What You Need to Know About Clinical Trials Before You Sign the Dotted Line

Is a clinical trial right for you?

When you are dealing with a serious illness, there may come a time when you consider a clinical trial. Perhaps there are no known successful treatments, or perhaps you have tried all of the existing therapies and none of them have worked as you and your doctor hoped. The logical next step is often a clinical trial, but since all trials carry some degree of risks, it’s important for you to understand what you are getting yourself into before you move forward.

What exactly is a clinical trial?

Clinical trialsfemale lab technician examines specimens under a microscope are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. These studies might show which medical approaches work best for certain illnesses or groups of people. Most trials study one treatment, but there are studies that compare multiple treatment options.

The purpose of clinical trials is research, so the studies follow strict scientific standards which help produce reliable study results and protect patients. The federal government enforces rules to keep trial participants safe.

Clinical trials are one of the final stages of a long and careful research process, which usually begins in a lab, moves to animal testing (if it’s deemed promising), and then to humans (if it’s deemed promising and safe to try in humans).

For safety purposes, clinical trials start with small groups of patients to find out whether a new approach causes any harm. In later phases of clinical trials, researchers learn more about the new approach’s risks and benefits.

Results of a clinical trial typically fall into one of 3 categories:

  • Improves patient outcomes
  • Offers no benefit
  • Causes unexpected harm

The results of clinical trials provide the best data available to help patients and doctors make decisions for treatments and are an important part of advancing medical knowledge and patient care.

What are the different phases?

There are 5 phases (0-4) for clinical trials, although many trials are designed for 1 or 2 phases. Additionally, a trial can be stopped at any point if patients are being harmed or the treatment is not working.

The methodology and goals differ for each phase:

Phase 0

Usually patients are not involved until Phase 1. However, some studies enroll a small group for Phase 0 to learn if a treatment behaves the way they expect based on their lab studies. In Phase 0, patients receive a very small dose of the medication, a dosage too small to provide any curative effect (and also less likely to cause side effects).

In Phase 0 researchers are generally looking for the following:

  • Whether the drug reaches the target
  • How the drug behaves in the body
  • How the targeted cells in the body respond to the drug

The main aim of Phase 0 studies is to speed up the development of promising new drugs. Testing them in very small doses in humans, instead of animals, can be more reliable and therefore provides useful information more quickly.

Phase 1

The goals of phase 1 trials are to learn:

  • How much of the drug is safe to give
  • What the side effects are
  • How the body copes with the drug
  • If the treatment works (shrinks the cancer, etc)

Phase 1 trials are “escalation studies” and only involved a small number of patients. If the first few patients do well on a very small dose, the next group gets a slightly higher dose. The dose is gradually increased with each new group of patients. The researchers continue until they find the best dose to give.

Phase 2

If a treatment “passes” phase 1, researchers move to Phase 2 to learn:

  • If the new treatment works well enough to test in a larger phase 3 trial
  • Which types of illness the treatment helps
  • More about side effects and how to manage them
  • More about the best dose to use

There may be up to 100 or so people taking part in this phase.

Sometimes in a phase 2 trial, the new treatment being studied is compared with another treatment already in use, or with a placebo (a fake drug). In these situations, the studies are randomized – patients are randomly assigned to receive either the drug being tested, a standard medication, or a placebo.

Phase 3

If the results of phase 2 trials show that a new treatment may be as good as existing treatment, or better, it then moves into phase 3.

In phase 3, researchers try to discover if a new treatment will work and under what conditions. These trials may compare:

  • A completely new treatment with a standard, existing treatment
  • Different doses or ways of giving a standard, existing treatment

Phase 3 trials usually involve thousands of patients in many different locations. Most phase 3 trials are randomized.

Phase 4

Phase 4 trials occur after a drug has been shown to work and has been granted a license.

The main reasons for running phase 4 trials are to learn:

  • More about the side effects and safety of the drug
  • What the long term risks and benefits are
  • How well the drug works with widespread use

What are the possible benefits of enrolling in a clinical trial?

thumbs up drawing to indicate the positive aspects of participating in a clinical trialThere are many potential benefits from participating in a clinical trial, including:

  • Access to new treatments before they’re widely available.
  • If a new treatment works and you’re in the group getting it (as opposed to a placebo group), you might be among the first to benefit.
  • Close monitoring of your health:
    • Having more blood tests, CT scans or other cancer tests
    • Having check ups more often and for longer than usual with your doctor or research nurse

Even if you don’t directly benefit from the results of the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people enroll because they want to help others.

What are the possible downsides of enrolling in a clinical trial?

thumbs down image to indicate the potential negative aspects of participating in a clinical trialAll clinical trials carry a degree of risk and potential downsides, so be sure to consider the pros and cons of a trial, along with the outlook for your health if you decide not to enroll in a trial.

Every trial has potential downsides, including:

  • The new strategies and treatments being studied aren’t always better than available care.
  • The new approach being studied may not work for you, even if others are benefiting (which is true of all treatments).
  • A new treatment may have serious side effects or risks that doctors don’t know about or expect.
    • This is especially true during phase I and phase II clinical trials.
    • The risk of side effects might be even greater for trials with cutting-edge approaches, such as gene therapy or new biological treatments.
  • You may not be part of the group receiving the new treatment – you may be part of a control group which receives the standard treatment or a no-treatment placebo.**
  • Being part of a clinical trial can be inconvenient as it may require more office visits and/or hospitalizations.
  • Health insurance and health care providers don’t always cover all patient care costs for clinical trials. Find out ahead of time about your costs and coverage.

** It is important to note that in studies for medical conditions for which effective and safe treatments already exist, no one receives a placebo; patients get either the new drug being tested, or the accepted, approved treatment. Before you decide to participate, ask if the alternative treatment is a placebo or a known existing treatment.

What kinds of questions should you ask before enrolling in a trial?

When you are beginning any new type of treatment, you want to make sure you have a clear understanding of the proposed treatment, including how it could help your condition, how likely it is to help, and what the common side effects are. When considering a clinical trial, there are even more questions to ask because you are considering a treatment that is still in the testing phase.

I strongly urge you to visit Clara Health’s website for an extensive list of questions you should ask when considering clinical trials.

How can you find a clinical trial that might work for you?

Since there are so many trials underway, it’s almost impossible for doctors and patients to be aware of all potential trials. Don’t assume your doctor knows about all of your potential options.

Fortunately, there are many sites the provide helpful information. However, it’s important to realize that the information available on many websites contains a lot of medical jargon. As a result, patients may struggle to understand the scope of the trial and whether they would be a good candidate.

For a list of websites where you can research clinical trials, visit Zaggo’s Resource Center’s page on Clinical Trials.

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