More about Clinical Trials
More about Clinical Trials

How do clinical trials come about?
A clinical trial is often the result of many years of preparation. The whole process starts out with a question a scientist or group of scientists want to answer. Let’s look at a fictional (made up) example:

Joy Abequa, MD
Northern University

Dr. Joy Abequa is a young Native American doctor who wants to answer the following question:

  • Does taking vitamin E daily reduce the chances of developing breast cancer?

Before this question is tested in a clinical trial with people, many small studies are done in the laboratory, and using animals.

Pre-Clinical Studies
Dr. Abequa will first try to find information on what other researchers have done to study vitamin E and cancer. Based on what she learns from other researchers’ results, she will develop a set of her own methods to test her question.

For example, Dr. Abequa may do tests on breast cells in a test tube to see if vitamin E helps protect them from cancer causing chemicals. If these results show promise, then she may apply for permission to test the question using animals.

Dr. Abequa then tests vitamin E on several mice that have been specially bred so that they get cancer after they are a few weeks old. She gives some of the mice viatmin E, but not all of them. After some weeks have passed, Dr. Abequa looks to see if there is any difference between the mice that got vitamin E and those that didn’t. She will also look to see if the vitamin E did any harm to the mice that got it.

If the results with the mice show promise, further testing with animals will be done to make sure it is safe to take larger quantities of vitamin E.

Finally, after a lot of testing and proving that the vitamin E is safe in animals and that the vitamin is working to protect against cancer…«FINISH< WILL RETURN»>doesn’t give via special type of mouse that has been bred so that it gets cancer gets cancer will then choose to test After getting permission from her The testing with animals will help show whether the vitamin E works in a living animal and if it is safe. in This type of study is often called basic research because it takes place First, scientists find that they need to answer a scientifiBy the time a clinical trial is ready to enroll particpantsClinical trials, also called clinical studies, start out as a scientific question. A scientist or group of scientists

Not everyone can take part in a clinical trial study

  • You have to meet the "eligibility" rules (such as the size of your tumor, other health conditions)
  • Not everyone should take part in a clinical trial study
  • Not every trial is the right choice for you personally may be due to cultural or other reasons
Insert interactive question here please:

Which of the following is a characteristic of most of the people who take part in clinical trials?
They are:

  1. Poor
  2. Minorities
  3. On Medicaid
  4. College educated
  5. Don't know / Not sure
Have another screen come up with "The correct answer is "4. college educated"
Most clinical trials participants are:
  • well educated (college degree)
  • middle class or wealthy
  • white
  • almost all have private insurance
Native patients, other minorities and poor people are less likely to receive information about clinic trials
Do providers know of a cure but don't share it with the public?
  • Most providers who work in cancer care have family members with cancer.
  • Providers are as anxious as anyone else for cures to all cancers to be found.
What are the Stages of the Clinical Trials Process?

Linda U. Krebs, RN, PhD, AOCN - Oncology (cancer) nurse
"Dr. Krebs" describes Stages of Clinical Trials;
to play Video Vignette - click

Clinical Trials studies are part of a thorough research process.
It starts with a scientist who gets an idea about:

  • A new treatment for cancer
  • A different way to give a treatment for cancer
  • Or, a way to reduce the side effects of a treatment
    • This process begins in the laboratory (pre-clinical trials studies) and once the drug or technique looks like it may be both safe and effective, it is tried out on people who volunteer to take part in the study
    • There are different risks and benefits related to each stage or phase of a clinical trial.
    • That is one reason why two people may be enrolled in a breast cancer clinical trial, but they may have totally different experiences.
This shows the complete process for creating a new, more effective drug for cancer treatment. It goes from laboratory science in a test tube to animals or computers and then to people (sometimes called, "from bench to bedside")
Stage #1 of the Clinical Trials Process for a new cancer treatment Add Linda Krebs 19:20 to 19:47 Basic Research
Basic Research
  • Done in a laboratory
  • Usually uses a microscope or other technical equipment
  • Looks at cells and smaller parts (genes)
The end result is a new drug that may be helpful in treating cancer.

"Dr. Krebs" describes Preclinical studies;
to play Linda's Video Vignette - click

Preclinical Studies

  • Experiments using the potential drug on mice, rats, pigs, dogs and other small animals
  • Newer methods use computers rather than animals
  • The end result is a new drug that may be helpful in treating cancer in humans

"Dr. Krebs" describes

"Cisplatin example"; to play Video Vignette - click

"Cisplatin used head, neck, lung, testicular"; to play - click

Preclinical studies and animals w/ Adrymycin example
"Adrymycin example"; to play Video Vignette - click

Basic Research Preclinical Studies

Clinical Trials in People

"Dr. Krebs" - Basic science to preclinical studies ;
to play Video Vignette - click

There are 4 phases involved in clinical trials process in people:

"Dr. Krebs" - Overview of the Phases;
to play Video Vignette - click

Clinical Trials
Phase I Safety

  • Phase II Efficacy
  • Phase III New vs. Standard
  • Phase IV Post-marketing
Characteristics of each Clinical Trials Phase

"Dr. Krebs" describes Phase I of Clinical Trials;
to play Video Vignette - click

Phase I. Safety

  • To learn the best and safest way to give a new treatment and begin to learn about possible side effects
    • The patients have advanced cancer and:
  • Have gone through standard treatments
  • But the treatments are not working
  • Or, their disease changed
    • Because there is no better treatment to offer the patient, these patients are willing to try something experimental
    • Done with small numbers of patients (usually less than a total of 30)
  • Groups of three people at a time try a new treatment
  • They try one dose of the new drug and see what happens
  • If the drug seems to be working, another group of three try a stronger dose of the drug
  • If that dose still seems to be working, another group of three try an even stronger dose of the drug
  • This continues until the dose has too many bad side effects
    • Patients are not expected to personally benefit, but the information learned is likely to help other cancer patients
  • The patient may or may not benefit, but "personal benefits" cannot be promised to the patient.
  • It is important that the patient realizes the benefits of taking part in the study will be passed on to other cancer patients
Phase II. Efficacy Add Linda Krebs 32:10 to 34:12 Phase II Efficacy and 33:29
  • To learn if the new drug works better for different types of cancer
  • To learn about possible side effects
  • Done with larger numbers of patients (usually less than a 100)
  • The patients have:
    • Never been treated
    • Shown little to no response to previous drug treatment
    • Or, relapsed after standard treatment

"Dr. Krebs" - Phase III ;
to play Video Vignette - click

Phase III. New versus Standard Treatment

  • To learn how a possible new treatment compares to the current standard treatment for a specific type and stage (early; advanced) of cancer
  • Done with large numbers of patients (100's to 1,000s)
  • The patients:
    • Have never been treated
    • Or, a previous treatment didn't work for them
  • In cancer clinical trials, no "sugar pill" (placebo) is used
    • If a treatment exists for your type of cancer and you are in a clinical trial:
      • You will always receive at least the standard treatment given to others with this type of cancer
      • You may also receive a treatment that may be even better than the standard treatment
  • These studies may see if a new drug or combination of drugs is better than what is currently given as standard treatment
    • Maybe drugs "A" "B" and "C" are compared to drugs "A" "B" "C" and "D" ("D" is the new drug)
  • Many clinics across the US and Canada will offer these types of trials
  • These are done to get Food and Drug Administration (FDA) approval of a new drug
Phase IV. Post Food and Drug Administration (FDA) Approval
  • To learn how a treatment may also work in different types of cancer
    • After TaxolŽ was found to work for breast cancer, then in a Phase IV study it was tried in males with prostate cancer
  • These are very rare studies
  • These studies are becoming more common
  • These are done to get FDA approval of a new use of an approved drug or a new method of giving a new drug
  • Done with large numbers of patients (100's to 1,000s)
  • Similar to a Phase III, but with patients who have a different type of cancer

"Dr. Krebs" - whole process reviewed ;
to play Video Vignette - click

So, Phase I trials are only available in a few locations and is for people who have not responded well to existing treatments.
Phase II trials are limited to a small number in only a few locations
Phase III trials are available in clinics throughout North America
Phase IV are rarely available, but are becoming more common
Do you want to hear a story about how a new treatment evolved through these phases?
Yes [If yes, add each segment of Linda Krebs' the cisplatin examples
20:38 to 22:06 Cisplatin example
22:15 to 23:56 Cisplatin used head, neck, lung, testicular; Lance Armstrong
32:22 to 33:01 Cisplatin Phase II example
34:58 to 35:25 Cisplatim for ovarian for Phase III
30:50 to 31:33 Cisplatin given as IV



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