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Clinical Trials

Let’s Talk About Clinical Trials

Clinical trials provide early access to potentially beneficial treatments, advance science, and play an integral role in developing new and improved treatments for brain tumors. Often, the lengthiest aspect of the drug approval process is finding people to participate in trials, even though the National Comprehensive Cancer Network (NCCN) believes the best management of any patient with cancer is a clinical trial.

Explore key terms, important questions to ask, and other educational resources about clinical trials as you navigate your brain tumor experience.

Key Questions to Ask About Clinical Trials

Review the collection of questions below about clinical trials. We encourage you to bring these questions with you to your next appointment. This content is provided by the National Cancer Institute.

  • What is the purpose of the trial?
  • What phase is the clinical trial?
  • Why would the clinical trial treatment be better than the standard of care — or any other treatment option to treat my tumor?
  • How does the clinical trial treatment work to treat my brain or spinal tumor?
  • If the clinical trial is a drug therapy, how is the drug given (orally or intravenously)?
  • What procedures are required as part of the clinical trial?
  • What are the criteria to determine if I am eligible for the trial?
  • How long will I be in the trial?
  • How often do I have to be seen in the clinic or hospital as part of the trial?
  • Can I be part of the clinical trial at a location closer to my home?
  • How do you evaluate if the treatment is working?
  • How often would I need to have imaging, and would this be part of the trial evaluation?
  • How often do you evaluate if the treatment is working?
  • How will I be informed of the results?
  • How long do I have to make up my mind about joining this trial?
  • Who can I speak with about questions I have during and after the trial?
  • Who will be in charge of my care?
  • If I want a second opinion, whom would you recommend I see?

  • What are the possible side effects or risks of the new treatment?
  • How are side effects managed?
  • Are there other health risks associated with participating in the trial?
  • What are the possible benefits?
  • How do the possible risks and benefits of this trial compare to those of the standard treatment?

  • How will my health information be kept private?
  • If the treatment isn’t working, can I stop the trial?
  • Can I stop participating in the trial at any point I choose?
  • If I decide to leave the trial, what other options will I have?

  • Which costs do I have to pay if I take part in the trial?
  • What costs will my health insurance cover?
  • Who can help answer questions from my insurance company?
  • Who can I talk with about costs and payments?

  • How could the trial affect my daily life?
  • How often will I have to come to the hospital or clinic?
  • Will I have to stay in the hospital during the clinical trial? If so, how often and for how long?
  • How far will I need to travel to take part in the trial?
  • Will I have checkups after the trial?
  • Will the clinical trial require rehabilitation services, such as speech therapy or physical therapy?

  • What are my other treatment choices, including standard treatments?
  • How does the treatment I would receive in this trial compare with the other treatment choices?
  • What will happen to my cancer if I decide not to have any treatment?

Key Terms to Know About Clinical Trials

The research world has its own set of lingo for the important work they do. Read our list of important terms to know as you familiarize yourself with clinical trials.

Clinical Research
Clinical research is medical research that involves people to test new treatments and therapies.

Clinical Trial
A clinical trial is a research study in which one or more human subjects are prospectively assigned to one or more interventions, which may include a placebo or other control to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.

Exclusion Criteria
Exclusion criteria are factors that do not allow someone to participate in a clinical trial.

Healthy Volunteer
A healthy volunteer is a person with no known significant health problems who participates in clinical research to test a new drug, device, or intervention.

Inclusion Criteria
Inclusion criteria are factors that allow someone to participate in a clinical trial.

Informed Consent
Informed consent explains risks and potential benefits about a clinical trial before someone decides whether to participate.

Patient Volunteer
A patient volunteer has a known health problem and participates in research to better understand, diagnose, treat, or cure that disease or condition.

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help researchers answer different questions.

  • Phase I trials: An experimental drug or treatment is administered to a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects.
  • Phase II trials: The experimental drug or treatment is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.
  • Phase III trials: The experimental drug or treatment is administered to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, and compare it with standard or equivalent treatments.
  • Phase IV trials: After a drug is licensed and approved by the FDA, researchers track its safety, seeking more information about its risks, benefits, and optimal use.

A placebo is a pill or liquid that looks like the new treatment but does not have any treatment value from active ingredients.

A protocol is a carefully designed plan to safeguard the participants’ health and answer specific research questions.

Principal Investigator
A principal investigator is a doctor who leads the clinical research team and, along with the other members of the research team, regularly monitors study participants’ health to determine the study’s safety and effectiveness.

Randomization is the process by which two or more alternative treatments are assigned to volunteers by chance rather than by choice.

Single- or Double-blind Studies
Single- or double-blind studies, also called single- or double-masked studies, are studies in which the participants do not know which medicine is being used, so they can describe what happens without bias. In single-blind studies, you are not told what is being given, but the research team knows. In a double-blind study, neither you nor the research team are told what you are given; only the pharmacist knows. Members of the research team are not told which participants are receiving which treatment to reduce bias. If medically necessary, however, it is always possible to find out which treatment you are receiving.

Types of Clinical Trials

  • Diagnostic Trials: Diagnostic trials determine better tests or procedures for diagnosing a particular disease or condition.
  • Natural History Studies: Natural history studies provide valuable information about how disease and health progress.
  • Prevention Trials: Prevention trials look for better ways to prevent a disease in people who have never had the disease or to prevent the disease from returning.
  • Quality of Life Trials: Quality of life trials, or supportive care trials, explore and measure ways to improve the comfort and quality of life of people with a chronic illness.
  • Screening Trials: Screening trials test the best way to detect certain diseases or health conditions.
    Treatment Trials: Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.


Courtesy: National Institutes of Health

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Informed in 30:
Understanding Clinical Trials

Watch our informative 30-minute video that breaks down clinical trials, including key terms and common questions, with the help of a leading expert in the field.


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