Clinical Trials: A primer

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

What many people think of clinical trials

Why do we do this?

Types of trials

Phase IPhase IIPhase IIIPhase IV

Phase I trials

How much can we safely giveMay be looking at a new drugCan also new at a new combination of existing drugsWill often include studies of drug eliminationSmall numbers with escalating dosesMain endpoint is dose with tolerable toxicity

Phase II trials

We know how much to give, does it workWill treat a specific disease at a specific pointUsually smaller numbers of patients

30-60Look for disease response rates

Phase III trials

So it works but is it better than what we have nowPatients 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 therapyHave to compare to no therapy – placebo

Gold standard endpoint is survival

Phase IV trial

Once a drug or regimen approvedLooking at other issuesNot a common trial anymore

#1 Concern is patient safety

Staff trainingVetting of trials for patient safety issuesEnsuring the patient has appropriate information to choose to participate

Staff Training

All staff involved in research care of patientOur standard is CITI4-6 hour online education courseIncludes

History of clinical trials (warts and all)Special population concernsPatient consent

Trial Vetting

All trials must be reviewed by Institutional Review Board (IRB)May look at the science of the studyMain concern is patient safetyApproves all documents of trial and any advertising

Avoidance of coercion

Patient Consent

Patients are given an Informed Consent document with is explained thoroughly to themBest practice is for patient to take it home prior to agreementMust be written at 7-8th grade levelMust be in patient’s native languageMust clearly state that patient can decline trial and it will not impact their careMust clearly state that patient can withdraw at anytime

Requirements of Trials

Will require visits, treatments, labs, and radiology on a fairly rigid scheduleWill require clear pathologyMay require samples or tissue blocks to be sent to central labs

Why do trials here?

Give patients access to state-of-art therapiesHelp to define standard therapiesAdds cache to the entire institution

Any questions?