New Studies Currently Taking Place Clinical Studies: What You Need to Know

1. New Studies Currently Taking PlaceClinical Studies: What You Need to Know Cindy Murray NP Princess Margaret Cancer Centre

2. Objectives • What are clinical trials/clinical studies? • What is involved with being in a clinical trial/clinical study? • How to find the most up-to-date information about clinical studies.

3. What is a Clinical Trial or Clinical Study? • Answers scientific questions • Specific interventions according to a research plan or ‘protocol’ • Follows strict, scientific standards • Protects participants & helps produce reliable results • Evaluates: • Safety & efficacy of new treatments • New treatements to standard treatments • Quality of life NIH Clinical Trials.gov

4. Purpose of Clinical Trials 1. Are an important part of developing new treatments for MDS and other diseases 2. Help to improve diagnostic techniques 3. Identify new targets for treatment 4. May offer treatment options which are not otherwise available 5. Help to refine treatment strategies (such as refinement of the IPSS tool for MDS which is now the IPSS-R) 6. Help to improve side effect management 7. Offer a way to research quality of life while studying a disease or the treatment of disease Kurtin, S., Building Blocks of Hope. MDS Foundation.

5. Phases of Drug Clinical Trials • Phase I: first time a drug is used in humans. • designed to determine dosage & route of administration (oral, intravenous, or by injection), and schedule of administration (how many times a day or week). • begin to determine the drug’s safety • enrolls only a small number of people (15-30). • Phase II: Patients with the disease receive the drug at dose levels determined in the earlier phase. • begins to determine effectiveness of the drug & more information about its safety • trials usually include less than 100 people. • Phase III: drug is tested alone or against an approved standard drug. • enrolls a large number of patients (100-thousands). • If a comparison trial, patients may be randomly assigned to receive either the new drug or the standard intervention. • Phase IV In Phase IV, the drug, already approved & available but undergoes continued evaluation • designation is rare.

6. Members of the Research Team 1. Lead physician, scientist, or nurse researcher–primary investigator (PI) 2. Other clinicians: physicians, nurse practitioners, or scientists (Sub-Investigators) 3. Statisticians 4. Research nurses 5. Data Managers

7. How are Clinical Trials Monitored? • Institutional Review Boards (IRB): A group of experts from the institution conducting the trial or representing a cooperative group of institutions who review each trial for patient safety and scientific merit. The IRB will continue to monitor the conduct of the trial until it is completed along with the Primary Investigator and the research team. • Data and Safety Monitoring Boards: An independent committee of physicians, researchers, statisticians, and other experts.

8. Key Elements of a Clinical Trial • Patient protection: • potential risks and benefits of each trial are reviewed carefully by a number of groups. • Patient privacy is also a key component (data collected for the clinical trial will list patients by a study number and will only be shared with members of the research team who have signed a confidentiality agreement) • Informed consent: • research team must provide detailed information about the trial including purpose of the trial, potential benefits and risks, the treatment plan (protocol and schedule), and your right to withdraw from the study at any time • You will sign a consent form which provides this information and documents your informed consent. Kurtin, S., Building Blocks of Hope. MDS Foundation.

9. Eligibility for Clinical Trial • Each clinical trial has specific criteria for participation. • ensures specific research goals and patient safety criteria are being met • ‘screening’ for the trial occurs before enrollment to be sure criteria are met • Additional testing is conducted after informed consent has been signed • In some cases, after these tests are obtained, a patient may not meet the criteria for the trial and cannot proceed to treatment Kurtin, S., Building Blocks of Hope. MDS Foundation.

10. Questions to Ask About Participating in a Clinical Trial • Why is the trial being done? 2. What are the potential benefits of the trial? 3. What is the potential risk of the trial? 4. What can I expect from day to day while I am on the trial? (Frequency of visits, types of testing, length of visits) Kurtin, S., Building Blocks of Hope. MDS Foundation.

11. Questions…… 5. If I experience side effects, whom should I contact? 6. What are the costs of participating in the trial? 7. What other treatment options do I have if I do not participate in the clinical trial? 8. How long will I be in the trial? 9. What happens if the treatment is not working? Kurtin, S., Building Blocks of Hope. MDS Foundation.

12. How do I find Information about Clinical Trials? • many web-based resources available for patients, caregivers, health care providers & researchers • Not all studies will be found because there is no requirement by law for registering a study • Information is provided by sponsor or principal investigator of study

13. Canadian Database Canadian Cancer Society Research -supported by Canadian Cancer Society

14. Canadian Partnership Against Cancer

15. Health Canada’s Clinical Trials Database

16. ClinicalTrials.gov -database of private & publicly funded clinical trials around the world -maintained by National Library of Medicine at National Institute of Health -most comprehensive database

17. Clinical Trials.gov

18. Patient Support Organizations MDS Foundation

19. AAMAC