Phase 3 AMX0035

GXTrex

Distinguished member
Joined
Dec 2, 2020
Messages
136
Reason
Loved one DX
Diagnosis
12/2020
Country
US
State
NY
City
Long Island
Familial ALS is an exclusion for the short open label trial? That seems odd to me.
 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
10,859
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
It is a very small trial so they can be picky.
 

BlsdMama

Active member
Joined
Dec 5, 2016
Messages
80
Reason
PALS
Diagnosis
05/2017
Country
US
State
IA
City
Cedar Rapids
Why such a small trial?
 

lgelb

Moderator
Forum Supporter
Joined
Nov 5, 2009
Messages
10,420
Reason
Lost a loved one
Diagnosis
00/0000
Country
US
State
WA
City
Seattle
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.
 

Mary2

Distinguished member
Joined
May 1, 2021
Messages
103
Reason
CALS
Diagnosis
04/2021
Country
US
State
GA
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).
 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
10,859
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
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
 

Mary2

Distinguished member
Joined
May 1, 2021
Messages
103
Reason
CALS
Diagnosis
04/2021
Country
US
State
GA
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.
 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
10,859
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
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
 

Mary2

Distinguished member
Joined
May 1, 2021
Messages
103
Reason
CALS
Diagnosis
04/2021
Country
US
State
GA
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.
 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
10,859
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
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
 

smarinkov

New member
Joined
Sep 15, 2021
Messages
5
Reason
PALS
Diagnosis
09/2021
Country
NL
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.
 
Top