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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 • I have been an advisor for Biomarin 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 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! 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 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! 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 Diet Kuvan Responder Nonresponder Outcome - MRI/Cognitive changes Sample study schemes Treatment A Outcome Treatment B Outcome 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 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% 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 Can I Learn More? • More basic information on clinical trials is available in a later session • Check out www.ClinicalTrials.gov Barriers to Clinical Trial Participation Barriers to Clinical Trial Participation • Time! • Travel! • Executive Functions! What Makes Studies Tick • Study Design • Study Funding • Study Execution • Study Subjects Clinical Trials Central Additional information on clinical trials is available from the National Institutes of Health Clinicaltrials.gov Medlineplus.gov