Transcript Dustin-Clinical Trials

Patient Centered Drug
Development
Bench to Bedside
Families in Clinical Trials
Disclosures
• NINDS DIR
Objectives
1. Define patient centered research
2. Describe what is involved in a clinical trial
3. Discuss the SGS-742 Trial at NIH
Patient Centered Research
Patient-focused
Patient-centric
Patient-driven
Patient Engagement
Patient Engagement
& Clinical Trial Success
• 6200 industry-sponsored clinical trials
• 1.1 million participants
• 50 states
• 41 new drugs approved in 2014
• 80% of trials delayed
• 50% enroll one or no patients
Tockarshewsky, 2015
Patient-Centered Outcomes
Research (PCOR)
• Examples
– Genetic Test Results: putting parents and
providers on the same page
– Addressing children’s weight problems: learning
from success stories
National Center for Advancing
Translational Sciences (NCATS)
• Share information with patients
• Raise awareness among investigators
• Understand transparency issues
Maximizing Patient Participation
• 40% don’t understand clinical trials
• 32% say they would consider participating
after they understand what a clinical trial is
Tockarshewsky, 2015
What is involved in a clinical trial?
• Clinical trials look at new ways to prevent, detect, or treat disease
• Begins with an idea in the laboratory, in animal studies, and increasingly
from patients and patient advocacy groups
• Most promising experimental treatments are moved into clinical trials
• During a clinical trial, more information is gained about an experimental
treatment, its risks, and its effectiveness
www.Clinicaltrials.gov
The Protocol
• Plan for conducting clinical research
• Designed to safeguard the participants’ health and answer
specific research questions
• A protocol describes
– Who is eligible
– Details about tests, procedures, medications, dosages
– Length of the study
– What information will be gathered
The Protocol
• Led by a principal investigator (PI)
• Members of the research team are associate
investigators (AI)
• Monitor the participants’ health to determine
the study’s safety and effectiveness
• Table of Contents
• Précis:
3
• List of Abbreviations (if applicable)
5
• 1. Introduction and Background
5
• 2. Study Objectives
5
• 3. Subjects
5
• 4. Study Design and Methods
6
• 5. Management of Data and Samples 7
• 6. Additional Considerations
9
• 7. Risks and Discomforts
9
• 8. Subject Safety Monitoring
9
• 9. Outcome Measures
10
• 10. Statistical Analysis
10
•
11. Human Subjects Protection
10
•
12. Anticipated Benefit
11
•
13. Classification of Risk (for the study as a whole)
12
•
14. Consent Documents and Process
12
•
15. Data and Safety Monitoring
13
•
16. Quality Assurance
14
•
17. Reporting of Unanticipated Problems,
14
•
Adverse Events and Protocol Deviations
•
18. Alternatives to Participation
16
•
19. Privacy
16
•
20. Confidentiality
16
•
21. Conflict of Interest
17
•
22. Technology Transfer
17
•
23. Research and Travel Compensation
18
•
24. References
18
•
25. Attachments/ Appendices
18
•
26. Consent Forms
19
IRB Review
• Clinical Trials are approved and monitored by an
Institutional Review Board (IRB)
• Ensure the risks are minimal and worth any potential
benefits
• Independent committee of physicians, statisticians,
nurses, members of the community
• Ensure clinical trials are ethical and rights of participants
are protected
Sponsors
• Clinical trials are sponsored or funded by:
– Organizations
– Individuals
– Foundations
– Medical Institutions
– Voluntary Groups
– Pharmaceutical companies
– Federal agencies (NIH, VA)
Informed Consent
• Process and a document
• Provides potential participants with the key facts
about a clinical trial before they decide to participate
• Includes the purpose, duration, procedures,
risks/benefits, who to contact for more information
• Not a contract, volunteers are free to withdraw or
refuse particular treatments or tests at anytime
Types of Clinical Trials
• Natural History – how disease and health progress
• Prevention – better ways to prevent disease such as medicines, vaccines,
or lifestyle changes
• Screening – test the best way to detect certain diseases or health
conditions
• Diagnostic – determine better tests or procedures for diagnosing
• Quality of life – explore ways to improve quality of life for those with a
chronic illness
• Treatment – test new treatments, drug combinations, new surgery
approaches
Phases of Clinical Trials
•
Phase I – testing an experimental drug or treatment in a small group of people for
the first time to evaluate safety and identify side effects
•
Phase II – administration of experimental drug or treatment to a larger group of
people to determine its effectiveness and further evaluate safety
•
Phase III – administration of experimental drug or treatment to large groups of
people (1,000 – 3,000) to confirm its effectiveness, monitor side effects, compare
it with standard treatments, and collect information that will allow it to be used
safely.
•
Phase IV – following FDA approval and available to the public, tracking drug
safety, seeking more information about risks, benefits, and optimal use.
Clinical Trial Concepts
• Placebo – an inactive product that resembles the test product, but
without its treatment value
• Randomization – assigning two or more alternative treatments by
chance to demonstrate the new products therapeutic effectiveness
• Single or Double Blind – study participants do not know which
medicine is being used so they can describe what happens without
bias and preventing investigators from influencing results
• Cross-over design – study participants get both the placebo and
experimental treatment but do not know the order
SGS-742 Trial at NIH
• Phase II
• Double-blind cross-over
• Randomized
• Study Phases
– Screening and baseline
– Period I – 24 weeks
– 7-week washout
– Period II – 24 weeks
– Safety visit
Study Pills
•
Pink capsules
– Dose based on weight
– 3-month supply
– Pill counts/Return unused portion/empty bottles
•
Taken 3 times per day one hour before or two hours after meals because food reduces absorption of the drug
•
Use written calendar to record symptoms/side effects, missed doses
•
Missed doses
– Record on calendar
– take a missed dose if they remember within 4 hours; otherwise, to take the next scheduled dose and record the missed dose on
their seizure/medication calendar.
•
Side effects
–
the next scheduled dose increase will be delayed until side effects resolve
–
if side effects do not resolve, the dose will be reduced until side effects resolve
What to Expect
Side effects reported less frequently included headache, body aches,
swelling in hands and feet, infections and skin disorders. These did not
differ between subjects taking active drug and placebo.
• Regular contact to record any adverse events
– Phone
– CTSS
– Email
Clinical Trials Survey System
(CTSS)
http://ssadh.ninds.nih.gov
[email protected]
Planned Participation
• Capacity/Guardianship and consent/assent
• Travel plans (transportation, transfers,
overnight accommodations)
• Arrival times (security)
• Between testing (snacks, meals, downtime)
Following the Prot0col
(avoiding deviations)
• Study windows allow for flexibility of visits
• Visits outside the study window are
deviations
• Documentation - Importance of paperwork
– Inclusion/exclusion criteria
– Guardianship
– Pregnancy status
• Missing documentation is a deviation
Copyright 2015 National Center for Advancing Translational Science
Conclusions
- Patient centered research is essential to success of any
research program
- Goal is to engage patients and their families through all
stages of research
- The SGS trial is a product of collaboration between
scientists, clinicians, SSADH advocacy groups, patients
and families
- Be a part of the NIH SGS Study