An introduction for laypeople on the types of clinical trials and the safety aspect inherent in their operations

1. David L. Graham, M.D.
Levine Cancer Institute

5. Phase I
Phase II
Phase III
Phase IV

6. How much can we safely give
May be looking at a new drug
Can also new at a new combination of existing
drugs
Will often include studies of drug elimination
Small numbers with escalating doses
Main endpoint is dose with tolerable toxicity

7. We know how much to give, does it work
Will treat a specific disease at a specific point
Usually smaller numbers of patients
30-60
Look for disease response rates

8. So it works but is it better than what we have
now
Patients randomly assigned to a standard
treatment or the newer regimen
Assignment will take into account certain factors
about the patients and balance them
What if there is no proven therapy
Have to compare to no therapy – placebo
Gold standard endpoint is survival

9. Once a drug or regimen approved
Looking at other issues
Not a common trial anymore

10. Staff training
Vetting of trials for patient safety issues
Ensuring the patient has appropriate
information to choose to participate

11. All staff involved in research care of patient
Our standard is CITI
4-6 hour online education course
Includes
History of clinical trials (warts and all)
Special population concerns
Patient consent

13. All trials must be reviewed by Institutional
Review Board (IRB)
May look at the science of the study
Main concern is patient safety
Approves all documents of trial and any
advertising
Avoidance of coercion

14. Patients are given an Informed Consent
document with is explained thoroughly to them
Best practice is for patient to take it home prior
to agreement
Must be written at 7-8th grade level
Must be in patient’s native language
Must clearly state that patient can decline trial
and it will not impact their care
Must clearly state that patient can withdraw at
anytime

15. Will require visits, treatments, labs, and
radiology on a fairly rigid schedule
Will require clear pathology
May require samples or tissue blocks to be sent
to central labs

16. Give patients access to state-of-art therapies
Help to define standard therapies
Adds cache to the entire institution