1. An Educational Webcast
for Patients

2. Presenters
Catherine Gregor, MBA, CCRP, CCRC
Project Manager
ResearchMatch
Leslie R. Boone, MPH
Translational Research Coordinator
Vanderbilt University Medical Center

3. Today’s Objectives
• Discuss the role of clinical trials in advancing
research
• Review the methods and process of a trial,
including the phases of a study
• Review common features of trials for inflammatory
bowel diseases
• Discuss resources for more information

4. What is a clinical trial?
• Medical research studies that look for:
– New drugs or new combinations of drugs
– New procedures or devices
– New ways to use existing treatments
– Types of care, such as improving the quality of life for
people with chronic illnesses
• Also known as interventional study or clinical study
• Determine safety and effectiveness

5. The process from the start of basic research to
developing new drugs, treatments, or finding cures
takes an average of 13-20 years
2015… …2032
Lab
Clinical
Trials
Patient
Care

6. 10,000
compounds
250
Compounds
5 Compounds
1 drug approval
Drug Discovery Pre-Clinical Clinical Trials FDA Review Clinic
6.5 years 1.5 years6 years
What is the hold up?

7. What is the hold up?
• 50% of clinical research sites enroll one or no
patients in their studies.4
• Eighty percent of total trials are delayed at least
one month because of unfulfilled enrollment.2
• Each day a drug is delayed from market, sponsors
lose up to $8 million.5
All statistics from The Center for Information & Study on Clinical Research Participation: http://www.ciscrp.org
1.Beasely, "Recruiting". 2008
2.Lamberti, "State of Clinical Trials Industry", 292
3.Pierre, "Recruitment and Retention". 2006
4.Beasely, "Recruiting". 2008

8. Imagine if the whole process took closer to 5 years
2015… …2020?That’s much
better!

9. Insert Poll
• Have you participated (or currently participating) in
a clinical trial?
 Yes
 No

10. Insert Poll 2
• If you answered no, what would you say is the main
reason?
 No study in my area
 I do not meet criteria for a study
 I’m afraid of the risks involved
 I do not feel knowledgeable about clinical trials
 I am content with my treatments
 Other

11. Why Don’t People Participate?
• Lack of opportunity
• Lack of knowledge
• Lack of comfort

12.  Purpose  Compensation, treatment for injury
 Description  Whom to contact
 Procedures  Right to refuse or withdraw
 Risks  Confidentiality
 Benefits  Clinicaltrials.gov (if applicable)
 Alternatives
Deciding Whether to Participate:
Informed Consent
– Food and Drug Administration (FDA) requires that
volunteers are given information before they participate
– Informed Consent form includes:

13. Six Additional Elements
• Statement that there may be risks which are unforeseeable
• Under what circumstances investigator could terminate your
participation
• Additional costs to you
• Consequences of your withdrawal from the study
• When will research findings be disclosed to you
• Approximate number of people participating in the study
• You can still decide to withdraw from the study at any time

14. How do trials work? Study Methods
• 2 or more treatments
assigned by chanceRandomized
• Active drug or placebo?Double blind
• Inactive product
resembles study drug
• Continue course of
standard treatment
Placebo

15. Phases of Clinical Trial: Pre-Clinical
• In vitro (test tube or cell culture) and in
vivo (animal) experiments
• Wide-ranging doses to obtain
preliminary data on effectiveness,
toxicity and movement of drug within
body
• Determine if potential drug has
scientific merit for further development
as investigational new drug (IND)

16. Phase 1 Phase 2
Phase 4 Phase 3
Pre-Clinical

17. Phase 1
• Determine safe dose
• How does the body work? Phase 2
Phase 4 Phase 3
Pre-Clinical

18. Phase 1
• Determine safe dose
• How does the body work?
Phase 2
• Safety and Effectiveness
• Any short-term side effects?
• Narrow criteria selection
Phase 4 Phase 3
Pre-Clinical

19. Phase 1
• Determine safe dose
• How does the body work?
Phase 2
• Safety and Effectiveness
• Short-term side effects
• Narrow criteria selection
Phase 4
Phase 3
• Is it better than standard
treatment?
• Evaluate risks/benefits
• Most expensive and difficult
Pre-Clinical

20. Phase 1
• Determine safe dose
• How does the body work?
Phase 2
• Safety and Effectiveness
• Short-term side effects
• Narrow criteria selection
Phase 4
• Observation of serious
side effects
• Interactions with other
drugs
• Tests in groups or special
populations
Phase 3
• Is it better than standard
treatment?
• Evaluate risks/benefits
• Most expensive and difficult
Pre-Clinical

21. Common Types of Clinical Trials
• Treatment Trial
• Prevention Trial
• Diagnostic Trial
• Quality of Life Trial
• Compassionate Use

22. Clinical Research Team
• Principal investigator
– Plans and leads the trial
– Typically a physician
• Clinical Research Coordinator
– Coordinates daily trial activities
– Main contact for volunteers
and staff

23. Clinical Trials for Inflammatory Bowel
Diseases (IBD)
• Trials are appropriate for many different types
of people
• Specific requirements vary by trial
• The more people who take part in clinical trials,
the faster we will find better ways to treat and
potentially cure IBD

24. Common features of IBD studies
• Clinical Trials identify “Endpoints” or goals that the
medication is meant to achieve
• Common Endpoints for IBD related clinical trials:
– Symptom improvement
– Improve quality of life
– Small bowel or colon healing
– Reduction in need for other medication (ex.
Corticosteroid)
– Improvement in blood or stool markers of inflammation

25. Special considerations of IBD studies
• Pediatric clinical trials require parental consent
• Financial considerations:
– Clinical trials often cover medication under investigation
and any measures (i.e. lab work, clinic visits, etc.)
relevant to endpoint being investigated
– Other treatments may not be covered
• Some studies provide continued medication for
patients who respond after specified trial endpoint
– Specific to each trial

26. Where can I find more information?
• Clinical Trials on CCFA.org
– www.ccfa.org/research/participate-in-research/find-studies-and-clinical-trials
• ResearchMatch
– www.researchmatch.org/partner/ccfa
• ClinicalTrials.Gov
– www.clinicaltrials.gov
• CCFA Partners
– www.ccfapartners.org
• Your doctor

27. We all want the same thing
• Working together to find answers faster!
• Goes beyond the limits of financial
contributions and allows for a bigger
impact made by all
• www.researchmatch.org/partner/ccfa

28. Questions and Answers
IBD Help Center
9 AM- 5PM EST
(888) 694 8872, via email at info@ccfa.org
Or Answerchat on our website
www.ccfa.org

29. Irwin M. and Suzanne R. Rosenthal IBD Resource Center
(IBD Help Center) M-F, 9:00 AM-5:00 PM ET
– Phone: 1-888-694-8872
– Email: info@ccfa.org
Educational webcasts: www.ccfa.org/resources/webcasts.html
Connect with other patients
– CCFA Community website: www.ccfacommunity.org
– Support groups and Power of Two (peer mentors):
– www.ccfa.org/chapters
GI Buddy
Online tracking tool and mobile app www.ccfa.org/gibuddy
CCFA Partners: Patient Powered Research Network www.ccfapartners.org
Local educational events, visit: www.ccfa.org
Additional Resources for IBD Patients

30. THANK YOU
www.researchmatch.org/partner/ccfa