Transcript Intro Director of IMD Nutrition Clinic at TCH

Understanding Clinical
Trials
Laurie Bernstein MS RD FADA
Associate Professor
Director of IMD Nutrition
University of Colorado Denver
NPKUA July 2012
Disclosure
• I am a member of the Nutricia Elements
advisory board.
• I am involved in Kuvan and PEG-PAL clinical
research funded by Biomarin.
• I have current educational grants from
Biomarin and Nutricia.
The Role of Clinical Trials
• Research study done to evaluate new potential
treatments in people
• Before a new treatment is available to the
general public:
 Carefully studied ( study drugs have been
studied in labs and in animal studies are now
deemed safe)
 Human studies are referred to as clinical
studies
Clinical Study – gain more info to see if the study
drug is safe and effective and to look at both
risks and benefits in people.
**Important**
• When we begin talking to you about a
study, a lot of work has already been
done to develop a PROTOCOL
PROTOCOL
• Study plan
 Specific details of what will happen
in the clinical study
Study:
precise
carefully designed
KEY PURPOSE: Safeguard the
health of the participants
Design of the protocol
• Specific research question that
the study is designed to answer
• We cannot answer this question
without YOUR participation
The protocol describes the following
information:
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Who may participate in the study?
Exact schedule of tests
What procedures are involved
What the study drug is
Dose
Nothing is random – No personal
preference
Another important point
• Significant guidelines:
 if not, the study is not a success
• You can discontinue:
 no consequences
I would say make sure that you have checked
off the boxes that address your concerns.
Then you can comfortably start this and finish
this.
Risk and benefits
• Side effects
• Requirement of study
 Strict adherence
 Extra clinic visits
 Extra blood draws etc.
• Medical care –more interaction
• You are helping with medical research
• Future generations
Important questions before agreeing to
participate
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Purpose
Who is doing the research
Why do researchers believe it will be effective
Length of the research
What kind of tests will be involved
Daily life
Birth control
Safety
Access to t he treatment after the trial
Costs
What happens to the
information that is collected
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Confidential
Some info: IRB/ FDA/ Health Canada
Name never
Initials/dob/ethnic background/study code
Know this info before consent
Placebo
• Looks like the study drug but it does
not have the medicine in it.
 Inactive liquid or powder
 No treatment value
• Double blind
• Eliminates power of suggestion
• Keeps the research honest
Staying organized
• Reminder system that works best for
you.
 Daily calendar
 Stickers
 Beep on the phone
 Apps
 Ask for encouragement
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Who is my contact person
Best number
First visit
How do I get there
Bring
Park
Reimbursed
Resources
• www.clinicaltrials.gov
• www.Medlineplus.gov
• www.patientpower.info
 What to consider in assessing a clinical study
 CD’s available at the Biomarin booth
Thank you
• To our patients whose commitment and bravery
make the future of PKU management a reality.
• Blake Shewey
• Partners for Medical Education