Open Norway: Plasma cell aimed treatment with daratumumab in ME/CFS - Haukeland University Hospital

Yann04 said:
So IIRC the RCT is double blinded? Does that mean there is no way to have results until the trial has completed?

Oh 3 years of patience…
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When we discussed this before there was some speculation of preliminary results in 2027 if they got enough funding, its either in this thread or the pilot one but can't look now.

I think it was speculation about the recruitment being so long because they can't enroll as many patients simultaneously without full funding? That sort of thing.

I want this done right but late 2028 is an age away...
 
Yann04 said:
So IIRC the RCT is double blinded? Does that mean there is no way to have results until the trial has completed?

Oh 3 years of patience…
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Someone could publish interim results if they wanted to, but I don't think that is too realistic or purposeful here. It seems to be forgotten that this trial wil be fairly small: 66 patients. That might be small enough to just wait things out. We're only speaking about a handful of patients.

We've seen elsewhere that patient recuitment is probably the hardest part. Getting 66 patients that fit the bill will be hard enough. Could be quite tricky.

Quite possibly we might get a glimpse of what the results look like through the usual channels before 3 years.
 
Sasha said:
This is like a more consequential and hellish version of Netflix, where you watch a really great series and then realise that Season 2 won't come out for two more years.
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Yes, I used to be a big A Song of Ice and Fire fan (Game of Thrones books, for the less nerdy among us) and waiting for a treatment feels like waiting for The Winds of Winter to be published (the last book came out like 14 years ago)...except I can't live my life while I wait.
 
V.R.T. said:
Although it says 2030 completion on the clinical trial site are we really sure it's going to take that long? Seems ages for a relatively small phase 2.
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Utsikt said:
The planned followup for phase 2 is 72 weeks. Recruitment is uprn until Dec’26, but participants will never be a bottle neck (they could probably do 10x).

My guess is that the results will be out in 2027 if they get the funding sorted.
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Found the conversation
 
EndME said:
Quite possibly we might get a glimpse of what the results look like through the usual channels before 3 years.
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How? This is in the study design (linked from the english fundraising page)
The study will be conducted with a double-blind design, so that neither the patient nor the therapist knows which treatment the individual patient receives. The pharmacy has a coded list of the treatments, and this will be opened after the study is complete, i.e. when the last patient has been to the last visit after 72 weeks.
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It’s great there’s so much interest, hope and positivity about this trial. I know it’s hard but we should try to base discussion in what is known about the trial.
 
EndME said:
We've seen elsewhere that patient recuitment is probably the hardest part. Getting 66 patients that fit the bill will be hard enough.
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I'm just reading the published paper on the pilot study, which had 10 moderate-to-severe PwME, and it says that recruitment lasted from June 2022 to December 2023. That's 18 months to recruit 10 people! And now they need 66!

Any sense of how recruitment is going? It would be awful if slow recruitment dragged this thing out.
 
hotblack said:
How? This is in the study design (oinked from the english fundraising page)
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Because from the time you know how the roughly results look like (once everything is done), until publication often takes several months. Often you present your results at conferences or to colleagues before that and will already have a feel for the results before that once the first analyses have been run. I'm suggesting the possiblitiy of getting an idea of the results before publication.

Beyond that there might be an intermin analysis, as discussed above.
 
Sasha said:
Any sense of how recruitment is going? It would be awful if slow recruitment dragged this thing out.
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I don't think there is information on that. Recruitment taking somewhat longer is possibly a good sign. Any ME/CFS trial that is not based on an already existing clinical cohort that fills up quickly is a probably a red flag for recruitment not being rigorous enough. Let's not forget that the study with the most rigorous recruiment criteria to have been conducted on ME/CFS, the intramural study, failed to get even 20 patients (they had some additional criteria that needed to be fulfilled but the recuitment criteria here are also more stringent than elsewhere).

F&M managed to recruit close to 200 people for their RTX trials, but of course the criteria are now a lot tighter then back then. I guess one could take a look at those trials to see how many could roughly fit the Daratumumab criteria (the NK condition would have to be guessed).
 
EndME said:
I'm suggesting the possiblitiy of getting an idea of the results before publication.
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Got you. Sorry I’d misunderstood and assumed you meant before study completion and unblinding. My mistake and crossed wires with other posts.

On recruitment, is it that or funding that would be the limiting factor here I wonder? I guess it’s difficult to know for sure.
 
hotblack said:
On recruitment, is it that or funding that would be the limiting factor here I wonder? I guess it’s difficult to know for sure.
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I think that's hard to know, for example even given all information you don't know how rigorously the assessment of diagnosis will be handled. How detailed do the medical files have to be, what medical examinations are a must, do you exclude all people with autoimmune diseases, what about psychiatric conditions, how detailed will the medical examination with clinical examination at Haukeland be etc...
 
Trish said:
Aren't we getting ahead of ourselves? I thought this was a phase 2 trial. We need to wait for the results of at least one full scale double blind controlled trial before we start talking about roll out. Remember how long it took for the series of trials of rituximab.
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They're calling it a phase 2/3 trial in one of their documents. I think if the effect size is strong it might be good enough?
 
66 people in RCT feels like enough to tell us if this really works. What I fear is its not big enough to move the needle with the NHS, NICE and other bodies and they will want something more substantial. Going bigger isn't warranted yet but its 3 years for this trial and then another 3 years for the next big one assuming a government will fund it, might be years of fighting for that trial. Maybe in between it might be available at great cost privately but many ME patients after a life of being impoverished can't afford that.

I feel like a effectiveness trial like this needs to report faster as its not going to be the one that gets it approved as the treatment in the world, its too small.
 
Sasha said:
I'm just reading the published paper on the pilot study, which had 10 moderate-to-severe PwME, and it says that recruitment lasted from June 2022 to December 2023. That's 18 months to recruit 10 people! And now they need 66!

Any sense of how recruitment is going? It would be awful if slow recruitment dragged this thing out.
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Well the difference is back then they were coming off the back of a cyclo and ritux trial. Now the results from the pilot study are out, people are going to want to sign up.
 
V.R.T. said:
When we discussed this before there was some speculation of preliminary results in 2027 if they got enough funding, its either in this thread or the pilot one but can't look now.

I think it was speculation about the recruitment being so long because they can't enroll as many patients simultaneously without full funding? That sort of thing.

I want this done right but late 2028 is an age away...
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Kari Sørland, the study coordinator, says that they are aiming for 2029.

They have already started the trial, so I wonder how much the timeline depends on the funding. They have previously said they’ll get it done eventually (regardless of the funding situation), so 2029 might be a worst case scenario?

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Det kommer til å gå over, sa legene til Sigrid Danielsen (40) da hun var 23 år. Hun er fortsatt syk. Nå gir ny forskning håp.
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Utsikt said:
Kari Sørland, the study coordinator, says that they are aiming for 2029.

They have already started the trial, so I wonder how much the timeline depends on the funding. They have previously said they’ll get it done eventually (regardless of the funding situation), so 2029 might be a worst case scenario?
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I really hope that's the worst-case scenario! I wonder what the rate-limiting factor is - money, or recruitment?
 
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