Thanks, Gingerclam.
I refer to the following from
http://www.rettstudy.com/study-details/:
"Have study visits every 1-2 weeks for a total of 8 study visits."
This implies a 42 to 98 day on study meds, compared to 26 days during Phase IIa (assuming the same 2 week follow-up post treatment). Perhaps two groups, a 6 and 12 week group with a 2-week follow up for each, like last time?
These are exciting times for both Retts suffers and holders because researchers noted:
"(Retts Phase II) Results showed trends of increasing effect with the duration of treatment" in both dosages.
They also noted:
"The older age of the cohort and the short duration of the trial made it less likely to show a positive effect."
Based on the above consideration and the number and extent of improvements in both results and anecdotal (plenty covering improvements immune to placebo too for all the "healthy" skeptics) I am agreeable that a. this will enroll super fast, b. it has a very high pos% of success and c. will likely lead to a quiet BTD application and that gets granted given the trial design was co-created with the FDA.
As for the comments regarding publishing the results in a peer-reviewed journal, I think they're reasonable from an "academics" perspective, but that ignores
all sorts of IP and commercial considerations. I would much prefer that info be shared with Big Pharma behind confidentiality agreements during their D/D, rather than be released and potentially give "bread crumbs" for competitor pharma also chasing the austism "holy grail" only NEU is currently sitting on i..e both extremes of autism spectrum=1% of the population and potentially a lot more neuro related applications.
Anyway, here is a sample of some Retts anecdotal "newsflow" from the LOW dose trial as a refresher as to why this is such an exciting time for both Rett Syndrome sufferers and share holders. I will post more when I get back on my desktop comp which I used to track anecdotal "newsflow" on both Rett and FXS (open and closed groups):
Patient I
My daughter, Patient I age 27, has Rett Syndrome. She is a participant in the safety double blind study of NNZ-2566. This study is med vs placebo. She took a low dose of 35.5mg/kg twice a day. After her starting dose of 10mg/kg she went over 2 minutes at a time for over an hour without wringing her hands (which is a trait of Rett Syndrome). She became more attentive, used her right hand purposefully
(which she has never done), started making babbling vocalizations, and her body became less rigid. She has always been severely constipated and that ended also with us being able to get a bowel regime going. She was on her medication for 14 days. We saw no negative side effects from her medication. 36 hours after going off her medication, her hand wringing resumed with great intensity. She became rigid again. Her babbling decreased. Her eye contact went back to the way it was before. We do not know, nor do the doctors or any members of the research team know if Patient I had the med, NNZ or a placebo. I feel like she had the medication and not a placebo I would like for her to have the opportunity to take a larger dose and improve her quality of life."
Patient II
"My daughter was in this study as well. I saw overall improvements. She did not have any seizures, she was awake and alert,
she walked independently which she has not done for several years. Her overall health improved. Stamina, walking, seizures all improved while on NNZ medication. After just a few days of coming off NNZ my daughter went right back to all of her problems. She's having seizures, head is back to hanging down, she does not want to walk without full support from me, her overall health has declined and gone back to the way it was prior to taking NNZ."
Patient III
"Just wanted to update everyone about Patient III's NNZ-2566 Study. I do believe she is on the actual Drug. Patient III has a Baclafen Pump to control her tone/spasticity and she is due for a pump refill in 9 days, usually by this time she gets very tight and has pronounced leaning from her C-Curve. None of that is happening now as a matter of fact her tone is amazingly better throughout her body and that is without a PT for the last 3 months too. If the drug does nothing more than this for her I would have her take it in a second. Her constipation is better could even say she is in the arena of normal. She is using her lips to take food from a Fork/Spoon as opposed to using her teeth. Her motor planning also is better."
Patient IV
"My daughter Patient IV participated in this test at Baylor College of Medicine in Houston late last year. She was in the 35mg/kg group. Since it is a double blinded test we did not know is she received placebo or NNZ-2566. However, her behavior during the test changed enough that we believe she did. Having been off the study for two months she has reverted to her former behavior."
"Her behaviour changed noticeably. The hand wringing, mouthing, hyerpervenitlating, etc. all stopped. She (was) incredibly calm through all the travel and testing, like we’ve not seen before. A few weeks after the tests were over Patient IV’s former behaviour returned. "
Happy holidays, all!
8's
(20min delay)