1 / 21

Clinical Trials in PKU

Learn about the importance of clinical trials in PKU treatment, the role of blinding and randomization, understanding study arms and outcome measures, determining sample sizes, assessing results, and the key people involved in trials. Find answers to common questions and barriers to participation. For more information, visit ClinicalTrials.gov.

hardyc
Download Presentation

Clinical Trials in PKU

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Clinical Trials in PKU Georgianne Arnold, MD Professor of Pediatrics University of Pittsburgh Medical Center Pittsburgh Children’s Hospital

  2. Disclosure • I am a member of the PKUDOS advisory board • I am involved in Kuvan and PEG-PAL clinical research funded by Biomarin • I have been an advisor for Biomarin

  3. Why a Clinical Trial? • The effect of a new drug or treatment can be influenced by the power of suggestion, random chance, and other factors • A clinical trial attempts to minimize these influences and give us a scientific way to determine if the drug or treatment really works

  4. Why can’t I just have the treatment? • Blinding/randomization reduces bias • Investigator bias • Subject bias • Chance bias • The power of the human mind and expectation are not to be underestimated!

  5. What do they mean by…? • Blind – one party does not know what treatment the subject is getting • Double blind – neither the subject nor the researcher know what treatment the subject is getting • Randomized: the patient is assigned to a study arm…. randomly

  6. Why can’t I just have the treatment? • Blinding/randomization reduces bias • Investigator bias • Subject bias • Chance bias • The power of the human mind and expectation are not to be underestimated!

  7. What does “arm” mean? • Subjects are divided into various treatment groups. Measures of outcome are planned (in this case changes in brain structure on MRI and changes in neurocognitive performance). • Randomization Kuvan Diet Nonresponder Responder Outcome - MRI/Cognitive changes

  8. Sample study schemes Treatment A Outcome Treatment B Outcome

  9. How do you decide how many study subjects are needed? • Short answer – ask a statistician • Long answer – depends on what size effect you are looking for • The larger the change, the smaller the sample size needed to see it • When sample size is small, there may be some constraints to your conclusions

  10. How do you decide if it worked? • There is always the possibility that the results were obtained by a fluke • Researchers use statistics to determine the chance that the result is real • Generally accepted standard is the likelihood that the result is real is ≥ 95% • So, if the chance of error is ≤ 5% (0.05 or less), then we trust the result • Try to make the chance that you missed a real effect ≤ 80%

  11. Who are all these people? • Study overseen, and often developed, by the PI (Principal Investigator) • PI is assisted by study coordinators, nurses, technicians, and many others • Other people you may meet include psychologists, medical specialists, etc. • Statisticians, data entry assistants, student assistants and others may also see your data, but also undergo confidentiality training • Written document, must be provided and carefully reviewed prior to anyone agreeing to participate. • Review, ask questions, explain medical terms, understand all important info

  12. Can I Learn More? • More basic information on clinical trials is available in a later session • Check out www.ClinicalTrials.gov

  13. Barriers to Clinical Trial Participation

  14. Barriers to Clinical Trial Participation • Time! • Travel! • Executive Functions!

  15. What Makes Studies Tick • Study Design • Study Funding • Study Execution • Study Subjects

  16. Clinical Trials Central Additional information on clinical trials is available from the National Institutes of Health Clinicaltrials.gov Medlineplus.gov

More Related