CNM-AU8 Trial

[JC2024]

Hello All,

Just last week I was contacted by my neurologist and asked if I wanted to take part of a phase 2 trial of CNM-AU8 conducted by
Department of Neurology, Neuromuscular Division
Northwestern University Feinberg School of Medicine
Of course I said yes. I'm 4 days into it now keeping my fingers crossed.
I'll keep you all updated.

Happy Holidays to all

 

[JAM]

PLEASE make sure you check back in. We must live close. I’m right over the border of Wisconsin in Kenosha area. Is your Neuro at Northwestern?

 

[JAM]

Are you sure it’s Ph2? I thought Clene was planning a Ph3 in the New Year?

 

[JC2024]

Yes my Neuro is with Northwestern. I pretty sure they said phase 2 and they were waiting for funding and grants to start phase 3. When they do start phase 3 they said that I am now a candidate. I'm about 50 miles west of Chicago

 

[lgelb]

Looks like they are running a couple of EAPs, with Northwestern as one of the sites.

 

[JC2024]

I just wanted to clarify the trial that I am in is EAP04. Sorry for the confusion.

 

[JC2024]

Also I found this information about the EAP's here
ALS Expanded Access Protocols (EAP)
Hope its okay to post this

 

[ahl39]

Just want to give everyone a heads up. The availability listed on the Neals website is out of date. A lot of the listd "available" sites are actually all filled up. I know synapticure just finished screening their last patient this month and duke says they are not accepting new outside patients. The research coordinator I talked to says a lot of sites are saving spots for their existing patients, which makes sense.

Anyways, point is I would hurry and try to contact these sites as soon as possible. Even though there are like 300+ spots the eligibility criteria are pretty generous and there are a lot of ALS patients.

 

[JAM]

I don’t understand why someone would be in a clinical trial of this if there is EAP? Assuming you had a choice between the 2, this is confusing to me. As is a AA drug running at the same time as a Ph3 trial - if that were to happen (hypothetically) why would anyone do the PH 3 if they could get the drug thru AA? I’m surely missing something

 

[ahl39]

They would screen you first. If you are still eligible for the trial they won't let you get on the EAP. It would not be your choice.

Otherwise yes everyone would want to get on expanded access which is longer period and guaranteed drug instead of possible placebo.

 

[lgelb]

The EAP is for PALS not eligible for trials. Once they are in the EAP, they are asked not to start any new ALS treatments even if eligible.

 

[JAM]

That’s the part I didn’t understand. Their choice, not yours. Is that for AA drugs too?

 

[lgelb]

EAP and post-marketing confirmatory trial results are still publishable and become part of the evidence base for registration or continued approval (remember Relyvrio failed its post-marketing confirmatory trial and tofersen is in one right now) so was withdrawn by prior agreement).

Adding treatments mid-stream skews the data. That is true whether the drug/treatment is on the Accelerated Approval (AA) pathway or not.

 

[JAM]

But why would someone go into a Ph3 trial if the sane drug is offered via AA, if the trial has a chance of placebo? That’s what I dont understand.

 

[Nikki J]

For relyvrio once it was approved they didn’t enroll in North America so the trial participants were in Europe where they couldn’t otherwise get it

For Qalsody the confirmatory trial is in Sod1 carriers who also wouldn’t have access either. They are observed after enrollment and get monthly nfls. When it rises a certain amount they go into a placebo controlled phase. If anyone gets symptomatic ALS in either phase they get qalsody open label. Incidentally the only reason this was possible was the data from the Miami prefals study that showed a sharp nfl rise in sod 1 carriers in the 6 months before clinical onset. There is a lot to thank dr Benatar for who pioneered carrier research with this study ( still ongoing) but also the sod1 carriers who provided the data that was used. Sadly most if not at all passed away before ATLAS started. A number of them were my friends