Investor, how many seizures do you think the 15 Rett girls will have during the 7 week interval before A273 as a baseline?
Although some Rett patients have very poorly controlled seizures (especially the non-classic Rett cases that do not have MECP2 mutations), the majority have no seizures or rare seizures. The poorly controlled girls may have tried and failed multiple drugs for seizures while well controlled might be on well tolerated monotherapy.
I would not be surprised if they limit the study to "classic Rett" which will be girls with a MECP2 mutation (about 80% of Rett Syndrome is classic). In this very large (n > 1000) longitudinal study over many years, they found that only 32% of Rett patients had seizures over the preceding 6 months (some very poorly controlled with daily events). Of course most of 68% seizure free patients were on anti-epileptic drugs. I doubt they will take them off for the study of a non-tested agent with some girls expected to get low dose. Add-on is much more likely (and appropriate). So 5 or 6 of the 15 girls (6 or 7 if non classic Rett also included) may have experienced a seizure in the last 6 months but, per the same reference, only 17% classic (2 to 3 of 15 split among all doses) will have weekly or more seizures where a significant reduction might be noted over 7 weeks if the drug is an effective anti-convulsant at the given dose.
More likely, seizures in the girls who experience them will be one of the many exploratory measures and captured by a parent diary and/or the Caregiver Top 3 concerns VAS.
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