It could be that no trial is needed again with the effect size improvement being so much stronger than the approved Trofinetide. The smaller placebo group in Excellence trial was more effective than Trofinetide. Also obviously the adult trial was successful.
Kaufmann will make his points in regards to the trial and I think he will have weight with the FDA due to his expertise with RETT even if officially speaking the FDA shouldn't be influenced by this.
Kaufmann bio in regards to RETT:
"Dr. Kaufmann’s major focus of has been Rett syndrome, a field where he has published extensively on neurobiology and clinical aspects. He served as founder and leader of RettSearch, the international consortium of Rett syndrome clinical researchers. As such, he co-authored the current diagnostic guidelines for the disorder. He also edited the first clinical textbook on Rett syndrome, published in late 2017. Dr. Kaufmann has also served as Co-Principal Investigator of the NIH-funded Natural History Study of Rett syndrome (RDCRN program). Currently, he leads the Rett Syndrome Molecular Biomarkers Working Group for the Rettsyndrome.org foundation and the Rett Syndrome Behaviour Questionnaire Working Group. Dr. Kaufmann has played different roles, including site investigator, Principal Investigator, and DSMB member/chair, in almost 20 drug trials for neurodevelopmental disorders. In this context, he has been involved in virtually all neurobiologically-based drug trials for Rett syndrome.
Dr. Kaufmann has published more than 220 journal articles, most of them original publications. He has also served on several editorial boards and as reviewer for over 100 scientific journals."
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