1. How to Find and Participate in a GIST clinical trial
(aka---how to know where to look and who to call)

2. GIST Rare disease, 5000-7000 new cases of GIST every year
Compare 230,000 new invasive breast CA cases/year
GIST studies tend to be opened at fewer sites
OHSU(Portland), Dana Farber (Boston), Fox Chase (Philadelphia), MD Anderson (Houston), Memorial Sloan Kettering (New York)

3. Where are you coming from?
Oregon - 16
California -15
Washington - 10
Colorado - 4
Nebraska - 2
Alaska
Arkansas
Hawaii -2
Idaho -5
Illinois
Indiana
Michigan
South Carolina
Tennessee
Utah
Louisiana
Montana
Texas
Nigeria 10 1 16 5 1 2 1 1 1 15 4 1 1 1 1 1 2 1
Most of our GIST studies include travel reimbursement

4. Phases of Clinical Trials
Phase I: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase II: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase III: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use
Source:

5. Phase I Definitions
Dose Escalation
Dose Expansion

6. Dose Escalation Dose Escalation
The guiding principle for dose escalation in phase I trials is to avoid unnecessary exposure of patients to subtherapeutic doses of an agent (ie, to treat as many patients as possible within the therapeutic dose range) while preserving safety and maintaining rapid accrual (J Natl Cancer Inst May 20; 101(10): 708–720)

7. Dose Escalation Example
3 + 3 Design
3 patients 100 mg, wait one month for toxicities-none
3 patients 150 mg, wait one month for toxicities-none
3 patients, 200 mg, wait one month for toxicities-2 patients with moderate toxicities
Need to add 3 more patients to continue to evaluate for safety and wait one month for toxicities
IF MORE MODERATE TOXICITIES AT 200 mg then dose would go down to 150 mg for further testing
150 mg would usually be called the Maximum Tolerated Dose (MTD)

8. Dose Expansion
Use the MTD (Maximum Tolerated Dose) that was found during dose escalation (ie. 150 mg)
Given to more patients (approximately 25-50) to continue evaluating for safety, toxicity and efficacy
All patients in this group receive the same dose

9. Don’t Be Shy

10. What to do BEFORE you need a clinical trial
Check with your insurance
Does your insurance allow you to participate?
Myth: Clinical trials do NOT “pay for everything”
As of January 1, 2014, the Affordable Care Act (ACA) includes the requirement that private insurers cover routine costs during participation in a clinical trial
At OHSU we will check this for you prior to study participation

11. “Do you know your mutation?”
KIT (exon 9/11/13/17)
PDGFRA D842V (exon 12/14/18)
Wild Type--- what does that mean?
Recommend getting tissue mutation testing ASAP (with first biopsy or surgery)
At a minimum, mutation testing after Imatinib (Gleevec) failure
Do not usually need another biopsy/surgery to get this done

12. GIST mutations
Source: Michael Heinrich, MD

13. Be Aware of your Mutation
Tracy and I have an Exon 9 mutation

14. Keep Records Date of diagnosis Keep a copy of your pathology report(s)
Date(s) of surgery and location where surgery was done
Previous treatments and dates (be specific)
Ex. Diagnosed 2008
Gleevec 2008-Dec 2012, stopped for progression
Sutent Dec 2012-Dec 2013, stopped for progression
Regorafenib Jan 2013-Feb 2013, stopped for hand/foot syndrome

15. How Do I Find a Clinical Trial?
BLU (open at OHSU)
CELLDEX (open at OHSU)
DCC (coming soon)
PLEXICON (coming soon)

16. Be Empowered!

17. OHSU Knight Cancer Institute
Contact Information
Tracy Walker, RN, BSN
OHSU Knight Cancer Institute