Understanding Clinical Trials

Laurie Bernstein MS RD FADAAssociate Professor

Director of IMD NutritionUniversity 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 studyStudy:

precisecarefully 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:

• 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

• 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

• 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

• 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