CNM-AU8 Trial

[Mg8]

my understanding, I am in an EAP, is that other clinical trials do not want participants that are on the CLENE product. However, there is no restriction by CLENE on the use of other treatments. This based on a presentation by CLENE in the past month or so, on everything ALS, I believe.

 

[JAM]

That’s interesting that Clene is okay with other treatments while in their trial. How will they know which (if any) is having effects?

 

[Nikki J]

If they are going to allow other experimental treatments in their phase 3 subjects it could potentially invalidate their data. They were not allowed in the Healey phase. It is normal to allow approved drugs- standard of care so currently riluzole and radicava for most

 

[SMJgal]

Hello JC2024 and friends,
I have limb onset ALS that began in my hands. Diagnosed 9/2022; symptoms since 1/2021. I have been on Riluzole and Radicava since diagnosed. I have been on Au8 for 7 months now. My ALSFRS has plateaued during that time, but I know the strength of my legs and diaphragm continue to lessen. Is it helping? So difficult to know. This is the first time I have plateaued in an otherwise steady decline, so that is encouraging.
I will keep you updated. And thank you for this forum and everyone’s inputs; they are invaluable.

 

[JAM]

SMJ gal I hope you continue to plateau or even better to improve! Question: if you know your legs & diaphragm are getting weaker how does ALFSR score plateau? Is that not part of the score?

 

[Nikki J]

JAM the frs is not very sensitive. An example is walking you can be walk with assist with a cane or afo and still walk a good distance. Or you can struggle a few steps with great difficulty with a walker and a helper. All technically score the same There are other examples in the measures if you think about it.

 

[JAM]

We don’t know his score and no one really discusses it with us. I’m not sure what we would do with the info anyway .

 

[lgelb]

You can calculate it yourself if you care to. It is based on your answers, not clinical exam.

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)

The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) stratifies severity of ALS, including respiratory function.

www.mdcalc.com

 

[JAM]

I did look at it the problem is on a couple areas you may be between 2 numbers - not directly in either or overlapping . Still it was very interesting to see the categories & questions.

 

[lgelb]

No one considers it a great scale. There is a paper out fairly recently that proposes a number of alternatives.