Phase 3 AMX0035

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Familial ALS is an exclusion for the short open label trial? That seems odd to me.
 
It is a very small trial so they can be picky.
 
Why such a small trial?
 
Because it is designed specifically to look at how the body of a PALS takes up/disseminates the drug, not efficacy/safety as does the larger Phase 3 trial. It will augment the Phase 3 trial in the approval application process.
 
Nikki, My PALS wants to know if he could possibly do this through Emory and would he have to go to Emory. Right now we do Zoom visits with Emory because of the Covid and the distance (100 miles).
 
You are going to have to make some site visits. It doesn’t say how often. It also doesn’t list trial sites but would not be surprised if Emory was one. There is contact information in the listing both email and phone. You could also ask at Emory. I don’t know their setup but you could start with your nurse and they could tell you who to ask if they don’t know. At mgh there is a nurse who is the initial contact for trials but then there are coordinators for each trial.

I would pursue this in your place. Kristina was an early participant in the first trial and I know she believed it helped
 
Thank you Nikki, We will send an email off to Emory right this minute. Emory staff love to communicate by email and I am sure they will respond in the morning! By the way PALS is doing a little research study with MGH via zoom...study is done with a speech pathologist..no drugs are involved.
 
Good for him! I wonder if it is the one I am doing it is with a post doc Ana Luiza? I think there is another study as well

good luck with the trial
 
Emory has responded and they say PALS was just diagnosed in April but had symptoms/other diagnosis longer than 2 years so does not qualify for study. MD is discussing a supplement in AMX0035 that is available over the counter that other PALS are taking. PALS has ordered this supplement.
 
Yes that is in the description. Not uncommon unfortunately. Many of us take TUDCA some also have successfully got prescriptions for the other element You could ask about that too. It is expensive. Some have got insurance to pay. Others have gone to a compounding pharmacy
 
I was just yesterday informed that Phase III Trial of AMX0035 is expected to start within a month in Europe (not certain, but expected).

I was asked by my doctor would I consider participating. However, I don't know what would be the best decision --> if I choose to participate I can't start with TUDCA now and there's a chance of getting a placebo during the trial. So a full year without any meaningful therapy. Alternatively, I could ignore the trial and just take TUDCA on my own, perhaps with ibudilast (ketas) if I can obtain it. I'm in an early stage of ALS. Does anyone have a suggestion what to do?

As for the 2nd component of AMX0035 (SPB), although it is available in NL I can't get a prescription from my doctor for it (as it is not proven for ALS). So without prescription is both unavailable and too expensive for me.
 
I am signed up for this trial as of thursday 🤞
 
Amylyx has now formally requested review. The FDA has two months to reply as to whether it will entertain a New Drug Application, and, if so, whether under an accelerated schedule or not, at this time.

Also, dosing has begun in the big trial (PHOENIX).
 
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