Stanford CF Research- Past, Present, and Future!

1. Stanford CF Research-Past, Present, and Future!

2. History of CF Clinical Researchat Stanford • Initially started in mid 1980’s • IV antibiotics • Long term use of prednisone • Keflex in CF • Lung transplant studies • No research team • Funding • Ross Mosier Classic Golf Tournament

3. In Memory Clyde Mosier December 19, 1941- August 12, 2012

4. History of CF Clinical Researchat Stanford • 1991 - Dr. Moss division chief • ALL CF patients seen at LPCH • Active in Pulmozyme trials • 1992 – hired first research coordinator (RC) • 1993 – hired second RC • Other studies: IV immunoglobulin, TOBI, Once daily IV Tobramycin

5. History of CF Clinical Researchat Stanford • Studies short, 1-2 at a time • Developed a good reputation • 1999 – hired Database Coordinator • CF Registry • Educational Materials • 1990’s - Number of studies steadily increasing • Need for more patients to participate • Need for increased resources at sites

6. Therapeutics Development Network (TDN) • Established in 1998 - 7 sites • 1999 - Stanford 8th site • Increased # of studies, increased staff • 2009 - 77 total sites • Translational Research Center • 12 sites – Stanford included

7. Growth • 40+ active studies • Approximately 10 studies actively recruiting • Newborns to adult • Adult CF center now separate from pediatric • Present staff: • Research manager, 2 fulltime RC’s, 2 part time RC’s, Regulatory Specialist, Database coordinator, Lab assistant

8. Stanford CF Research • Newborn screening program • Advanced Diagnostic Clinic • Doing our own research projects: • Hand held percussor vs. vest • Lung Clearance Index (LCI) • STARS 1 and 2 • CF Education Day • CF Newsletter

9. Stanford CF Research • Research focused educational material • Mentoring of new RC’s • NACFC planning committee • Moderating and speaking at the North American CF Conference • 2013 European CF conference

10. STARS 1 Survey To Assess Research Satisfaction at Stanford Davies, Zoe PNP, CCRC; Dunn, Colleen RRT, CCRC; Hernandez, Cathy; Zirbes, Jacquelyn RN, PhD, CCRC; Milla, Carlos MD; Moss, Richard, MD.

11. STARS 1 Developed a questionnaire • 28 questions (scale 1-5) • 5 categories: • Overall Research experience • How satisfied were you with experience? • Research Process • Were you adequately informed about the study? • Research procedures • Research environment • Was the research unit professional and comfortable? • RC professionalism

12. STARS 1 • Stanford Database Search: • 330 Stanford CF patients • 107 patients eligible to participate • 84 completed: 25 parent, 59 adolescent/adult • 23 unavailable

13. Overall Results: • Stanford Study Subjects • Satisfied • Safe • Well cared for • Experience was worthwhile

14. Stanford Research “Repeat Offenders”

15. How satisfied a subject was with research experience correlated with repeated research participation Level of Satisfaction with Research Experience Number of Research Studies

16. Factors that Correlated the Strongest with Level of Satisfaction with Research Experience • Time to consider participation • No pressure felt during enrollment • Adequately compensated • Center fosters research awareness and participation • Working with research coordinators as an incentive

17. Areas Identified for Improvement: • Discussion of compensation • Understand what they are being paid for • Education of clinic staff of present projects • Open studies for enrollment in a timely manner • Need to develop new recruitment strategies

18. STARS 2 Survey To Assess Research Satisfaction at Stanford - 2 Davies, Zoe PNP, CCRC; Dunn, Colleen RRT, CCRC; Everson, Cassie RRT; Zirbes, Jacquelyn RN, PhD, CCRC

19. Objectives • To determine the reasons for non-participation. • To determine if there was interest in participating in future studies. • To identify “who” patients would like to hear from. • To identify “how” patients want to hear about studies.

20. Methods • A brief survey was developed: • Participated in research at Stanford? • If not, what was the reason for non-participation? • A non-biased interviewer asked the questions. • In person or over the phone. • Answers entered verbatim onto paper form.

21. Total eligible CF patient population 2010 CF Registry=324 Patients 289 Eligible Patients

22. Results

23. Results

24. Results

25. Results

26. Conclusions • Of those patients surveyed who had NOT participated in research 55% said “they were not asked” • Despite increased educational and recruitment efforts • Similar to the CFF survey in 2006 – 54% • May be due to relocation and decreased research presence in the adult CF clinic • Continued need to educate, and face to face interaction believed to be crucial.

27. CYSTIC FIBROSIS RESEARCH STUDIES NEED PEOPLE THAT HAVE CYSTIC FIBROSIS!

28. Projected Numbers of Subjects Needed

29. CFF Drug Development Pipeline

30. How can you get involved? • Inquire about research in clinic • Call the research team • Talk to your doctor

31. What would you have to do? • First you must: • Qualify • Sign consent/assent • Attend all study visits • Protocol defined • Complete all procedures • Protocol specific • BE REALISTIC!!!

32. WHY ME?...Why Not! • Risks • Outlined in the consent • Updated • Benefits • There may be none • Close follow up • Altruistic • Because you can

33. CF center websitehttp://cfcenter.stanford.edu

34. Cystic Fibrosis FoundationCFF.ORG

35. www.clinicaltrials.gov

36. THANK YOU!!!! TO ALL CF PATIENTS PAST, PRESENT, AND FUTURE WHO HAVE PARTICIPATED IN A STUDY

37. Contact Information Colleen Dunn 650 736-0388 cedunn@stanford.edu or Zoe Davies 650 498-5315 zdavies@stanford.edu

38. Many thanks to CFRI