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Clinical Trials in PKU

Clinical Trials in PKU. Georgianne Arnold, MD Professor of Pediatrics University of Pittsburgh Medical Center Pittsburgh Children’s Hospital. Disclosure. I am a member of the PKUDOS advisory board I am involved in Kuvan and PEG-PAL clinical research funded by Biomarin

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Clinical Trials in PKU

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  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

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