Metastatic Breast Cancer Alliance and UM's Dr. Erin Cobain review trial phases, clinical trial structure and common patient questions

1. 11:00 AM EDT

2. An Introduction to
Clinical Trials
Erin F. Cobain, MD
Clinical Lecturer
University of Michigan
Comprehensive Cancer Center

3. What is a Clinical Trial?
• Definition: a research study that tests how
well a new medical intervention works in
people
• Types of new medical interventions include:
– A new test to screen for a disease
– Medication to prevent the development of a
disease
– Medication to treat an active disease
• Metastatic breast cancer

4. What are researchers hoping to
learn from a clinical trial involving a
new drug?
• Common objectives:
– Establish safety profile of a new drug
– Determine if new drug is more effective than
current standard therapies
– Determine if a new drug is less toxic than
current standard therapies

5. Cancer Clinical Trials: The Basics
• Most clinical trials of new drugs begin with
research in a lab, where the drug is first
tested on cancer cells and animals
• Drugs that inhibit growth of cancer cells or
shrink tumors in animal models often
move forward for testing in humans

6. Cancer Clinical Trials: The Basics
• Phases of Clinical Trials:
– Phase I
• First in human studies
• Primary objective is often to determine if drug is safe, not if
drug is effective treatment
• Establish standard drug dose
– Phase II
• If new drug is demonstrated to be safe in phase I, phase II
trials aim to determine if drug may be effective
– Phase III
• If new drug appears to be effective at treating disease in
phase II, phase III trial is typically a large randomized study
where new drug is compared to current standard of care
therapy

7. Cancer Clinical Trials: The Basics
• Phases of Clinical Trials:
– Phase I
• First in human studies
• Primary objective is often to determine if drug is safe, not if
drug is effective treatment
• Establish standard drug dose
– Phase II
• If new drug is demonstrated to be safe in phase I, phase II
trials aim to determine if drug may be effective
– Phase III
• If new drug appears to be effective at treating disease in
phase II, phase III trial is typically a large randomized study
where new drug is compared to current standard of care
therapy

8. Cancer Clinical Trials: The Basics
• Personnel:
– Principal Investigator: usually a physician that has developed to
concept for the clinical trial
– Co-Investigators: physicians who can enroll patients onto a
clinical trial but are not leading the study
– Research Coordinator: often will communicate extensively with
patients regarding testing required on the clinical trial, informed
consent processes and clinical follow up schedule
• Oversight:
– All clinical trials must receive approval by a protocol review
committee (PRC) and institutional review board (IRB)
– Any adverse events that occur to patients while on a clinical trial
are reported to and evaluated by the data safety monitoring
board (DSMB)

9. Cancer Clinical Trials: The Basics
• The Protocol: roadmap of the clinical trial
• Protocol contains the following information:
– The reason for doing the trial
– Who can join the trial (called “eligibility criteria”)
– How many people are needed for the trial
– Any drugs or other treatments that will be given,
how they will be given, the dose, and how often
– What medical tests will be done and how often
– What types of information will be collected about
the people taking part

10. Cancer Clinical Trials: The Basics
• Informed Consent: document that explains a
clinical trial to patients
– Outlines risk and potential benefits of participation
– Describes which patients are eligible to
participate
– Provides contact information for research
coordinator and principal investigator
– Requires patient signature prior to formally
enrolling in the clinical trial
– Patient participation is voluntary and can be
withdrawn at any time

11. Who should consider clinical trials?
• Myth: clinical trials are only for patients
with advanced cancer who are not
responding to standard treatments
• Clinical trials may be considered by
patients with all stages of cancer, at any
phase of treatment

12. Where are clinical trails conducted
and by whom?
• Most oncology clinics participate in clinical
trials, although some centers may have more
trial options than others (academic versus
community practice)
• Types of clinical trial sponsors:
– Cooperative groups, national cancer institute
• Often large studies, open at multiple centers
– Pharmaceutical industry
– Investigator initiated trial

13. National Clinical Trials Network
(NCTN)

14. National Clinical Trials Network
(NCTN) Lead Academic
Participating Sites (LAPS)
• Academic medical centers with
hematology/oncology fellowship training
programs
• NCI-designated comprehensive cancer
center
• Site must demonstrate ability to enroll
large numbers of patients into NCI-
sponsored clinical trials
• Currently there are 30 LAPS centers

15. Is participation in a clinical trial
safe? Could I receive placebo?
• Clinical trials have a great degree of
oversight to ensure that they are conducted
as safely as possible (IRB, PRC, DSMB)
– If a safety issue is recurrent within the context of
a clinical trial, the protocol is often altered via an
“amendment”
• Placebo: often incorporated into a study
when the standard of care involves no
additional recommended treatment
– Rarely incorporated into a clinical trial of patients
with metastatic (stage IV) cancer, where
treatment is almost always indicated

16. Things to consider before agreeing
to participate in a clinical trial:
• Risks/potential benefits of standard treatment
options
• Risks/potential benefits of the experimental
therapy being studied
• Physician input
• How is the clinical trial recruiting patients?
– Biomarker (defining feature of tumor that may predict
increased likelihood of benefit from certain therapies)
– Unselected patients with a particular cancer type
• Logistics:
– Convenience of treatment schedule, clinic location

17. If my oncologist has not mentioned
clinical trials, what should I do?
• ASK!
Questions:
• Does your oncologist’s clinic have open
clinical trials currently?
• Are there clinical trials that you may qualify
for currently?
• Are there other centers that may have
clinical trials available?

18. If my oncologist has not mentioned
clinical trials, what should I do?
• Search online for clinical trial options:
– NCI-sponsored clinical trials:
https://www.cancer.gov/about-cancer/treatment/clinical-trials/search

19. If my oncologist has not mentioned
clinical trials, what should I do?
• Search online for clinical trial options:
– https://clinicaltrials.gov/

20. Will being on a clinical trial cost me
money?
• Generally participation in a clinical trial will
not cost you any additional money
– Investigational drug and study related
testing/procedures are often paid for by the study
sponsor
– Standard of care procedures are billed to
insurance company
• Exception: decision to travel to participate in
a clinical trial
– Transportation costs are often not covered,
however, some studies do provide some
reimbursement for travel costs

21. QUESTIONS / DISCUSSION