At the Emory site we have enrolled 52 subjects and have had no major adverse events (AEs). Please refer to the breakdown at the end of this document for details of this breakdown. After focusing on recruitment, our efforts have been on retention and compliance. For this we have integrated research visits and patient care visits to better monitor safety, make outcome measures and to enhance patient convenience and thus retention into a very long study. Most of the subjects are followed clinically at Emory which has helped retention. For that 20% that are followed elsewhere, or by physicians at Emory other than Dr. Juncos and Freeman, consistent communication with referring physicians has been key to promoting referrals and retention. Brief notes were forwarded to POPs or referring neurologist as laboratory abnormalities were detected, or there was a perceived need to make changes in PD medications or in other medications. This effort continues to work with excellent retention. Of the 52 subjects above, 1 failed screening, 1 died of unrelated and chronic cardiac causes, 2 were lost to follow-up, 2 withdrew from the study due to unrelated medical problems and failing health. These are the totals for the duration of the study with 46 remaining in the study at this site. All are SAEs have been unrelated to participation in the study. The two salient ones this year are: 1) A young onset PD subject had an unexplained syncopal episode followed by a negative work up. He continues to do well and is followed by an outside cardiologists;2) After stopping use of creatine, a second subject had a planned research hospitalization. Although no longer in the study, he is followed periodically by us as stipulated in the protocol. Other patient continue to participate without major complaints.
I have found the protocol to be safe and relevant for patients, but somewhat daunting because of the amount of experimental drug/placebo the subjects need to take. This makes me wonder whether, without measures to concentrate creatine dosing, can it be possible to convert this treatment into general use. Weight gain has been the most common non-serious AE. Non-specific elevations in serum renal function. Disclaimer: Please note that the following critiques were prepared by the reviewers prior to the Study Section meeting and are provided in an essentially unedited form. While there is opportunity for the reviewers to update or revise their written evaluation, based upon the group's discussion, there is no guarantee that individual critiques have been updated subsequent to the discussion at the meeting. Therefore, the critiques may not fully reflect the final opinions of th individual reviewers at the close of group discussion or the final majority opinion of the group. Thus the Resume and Summary of Discussion is the final word on what the reviewers actually considered critical at the meeting.
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