Open What questions should a PwME ask if they're considering entering a treatment trial? (specifically Lindus Health ReMEdi trial)

josepdelafuente

josepdelafuente

Senior Member (Voting Rights)
Hello,

I've done the eligibility questionnaire for the Lindus Health ReMEdi trial.
(some info here - https://www.lindushealth.com/news/l...rial-to-advance-me-cfs-research-and-treatment)
Apparently I'm eligible, according to the results of the questionnaire,
I now have a "screening call" with one of their nurses booked on Monday 8th September.

In another thread, we had some discussion on what the drug was (some people think it's most likely Amifampridine, but I'm not sure if we've managed to confirm this yet).

I'm wondering what questions I should ask during this screening call. I need to work out whether I want to do the trial or not.
I suppose one question might be - what is the drug? And another might be - why is it being trialled?

What other questions would pwME suggest asking in general, about clinical trials that we might be considering enrolling on, for potential ME interventions?
 
josepdelafuente said:
I suppose one question might be - what is the drug?
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I already asked, and they said they're not planning to tell people.

josepdelafuente said:
And another might be - why is it being trialled?
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Yep—they should be able to explain what mechanism they're targeting, what the biological rationale is for trialling the drug.

Other things about the drug might be knowing the full side effects profile (I'd want that as a written leaflet), whether there are any potential drug interactions (they probably won't say but it's worth asking), and whether it's a dose response trial.

I'd also expect a full description of the trial methods, selection criteria and outcome measures.
 
Some things I would want to know.
Name of drug
What it's already used for
Side effects
Number of doses and length of trial
Will they be trying different doses and will there be a control group getting a placebo
What patient reported outcome measures will they use?
What happens if I have bad side effects
Why is it thought to be useful for ME/CFS
Will my primary health care provider be informed, and who will pay if I need treatment for side effects including hospital treatment
Do any of the outcome measures assess PEM
Is there a reporting system for harms
How will effectiveness be assessed.
What is the primary outcome measure, and what secondary measures, what level of change will be considered clinically effective.
How many participants
Will outcomes reported take into account drop outs and deterioration
 
Others have covered most things. It may not be necessary to know the name of the drug (I’d want to, but many people are prescribed things without knowing much from the name) but understanding what it should do (mechanism) and potential side effects as well as what support is in place for those who experience them and adverse events must surely be essential for giving informed consent.

An argument against prompting with information on side effects may be that this encourages people to report them, but that shouldn’t matter if properly blinded and intervention and control groups are given the same information.
 
hotblack said:
It may not be necessary to know the name of the drug (I’d want to, but many people are prescribed things without knowing much from the name)
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But people aren't prescribed drugs until those drugs have undergone safety trials and trials for the condition that they're being prescribed for (unless they're being prescribed off-label, which is in a minority of cases). Also, the doctor prescribing them should be putting that patient first, whereas the patient in a clinical trial for a drug is arguably not being put first by the clinicians involved - there may be a profit/career motive instead or as well. People taking prescriptions are in a very different situation from people in trials.
 
Sasha said:
People taking prescriptions are in a very different situation from people in trials.
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Absolutely agree @Sasha and I may have phrased things poorly. I was trying to make a point that it’s often not the name of something which informs people, it’s the information about the effects of the medication (in conjunction with trust in the person prescribing or running the trial). So at the opposite end of the scale, saying ‘here’s the name of the drug, good luck’ would also not IMHO be good enough for informed consent.

Edit: and I suppose that a surprisingly large number of people just aren’t interested.
 
Given people with ME/CFS often respond badly to medication, anyone taking a new drug should be able to investigate what they might be taking if allocated to the treatment arm.

Obviously this pre knowledge shouldn't be a problem for those already enrolled in the trial as long as it is appropriately double blinded. However, there is a risk of it introducing selection bias into the study if there is a pattern as to people not participating or withdrawing once they know what drug is involved. One way to at least monitor this might be to say they will let people know the name of the drug once they have registered, but before they start the actual treatment.

Not telling people what the drug is at all does not avoid any selection bias, as not knowing might selectively put off those people with ME/CFS who have previously experienced adverse drug reactions, resulting in a cohort less likely to experience adverse reactions than the norm for people with ME/CFS.
 
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hotblack said:
I was trying to make a point that it’s often not the name of something which informs people
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That's true, but having a name unlocks the potential for them to access reams of information about it. Not all of which will be useful, or even reliable!—but there often is well grounded info from health agencies, disease charities, and organisations like the British National Formulary. It can help fill out the picture.

If it were amifampridine, though, a lot of that information could be missing anyway. It's apparently been in and out of licence and is only prescribed for small groups of people with rare muscle diseases.
 
I think in this case it's a Phase 2 trial, which from memory is an early stage trial, probably not with a control group and probably aimed at determining whether pwME tolerate the drug, drop out rates, and whether enough report some improvement to justify a bigger double blind controlled trial.
 
Trish said:
I think in this case it's a Phase 2 trial, which from memory is an early stage trial, probably not with a control group and probably aimed at determining whether pwME tolerate the drug, drop out rates, and whether enough report some improvement to justify a bigger double blind controlled trial.
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That would possibly be a good broad question, what are the aims of the trial?
 
josepdelafuente said:
(some info here - https://www.lindushealth.com/news/l...rial-to-advance-me-cfs-research-and-treatment)
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characterized by extreme fatigue, decreased cognitive function (“brain fog”), and other symptoms related to pain and dizziness
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No mention of PEM.

Participants will be required to wear the Oura ring for continuous remote monitoring and use the GripAble device for hand grip testing.
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OK - good to see some objective measures.

will evaluate the efficacy of a therapeutic with proven success to treat symptoms of other fatigue-related conditions.
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Anyway, looking at their 2024 portfolio - I don't know if it is exhaustive - in the "under development" section I also noted Pitolisant, which is an H3 inverse agonist acting as a CNS stimulant, FDA/EMA approved for narcolepsy and obstructive sleep apnoea. See our 2019 thread. No idea if that's a candidate, but we have a recent thread on possible overlaps with narcolepsy.
 
They've sent me an email which links to this participant information sheet - https://lindus-study.s3.eu-west-2.a...on%20Sheet_v2.0%2023Sep2024_Clean.docx%20.pdf

I googled PHOE-01 (the name Lindus give the medication, in their information sheet), and that led me to this NHS Health Research Authority page about the trial - https://www.hra.nhs.uk/planning-and...lication-summaries/research-summaries/remedi/

Where it says "Currently there is no clinical evidence with the effectiveness of the drug called PHOE-01, so in order to fully understand the clinical value of treating moderate ME/CFS, we plan to conduct this clinical trial in an adult population of patients suffering from ME/CFS and willing to take part in the trial. PHOE-01 is an oral tablet that has been licensed for the symptomatic treatment of patients with Lambert-Eaton myasthenic syndrome (LEMS)."

Don't know if that narrows things down at all for us.

The NHS page on LEMS (https://www.nhs.uk/conditions/lambert-eaton-myasthenic-syndrome/) says the following re specific medications that are used to treat LEMS:

"medicine to help nerve signals reach the muscles – commonly used medicines include 3,4-diaminopyridine and pyridostigmine.
medicine to reduce the activity of the immune system (immunosuppressants) – commonly used medicines include steroid tablets (such as prednisolone), azathioprine and methotrexate"

Interestingly no mention of amifampridine there.
 
josepdelafuente said:
"medicine to help nerve signals reach the muscles – commonly used medicines include 3,4-diaminopyridine and pyridostigmine.
medicine to reduce the activity of the immune system (immunosuppressants) – commonly used medicines include steroid tablets (such as prednisolone), azathioprine and methotrexate"

Interestingly no mention of amifampridine there.
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I think amifampridine is 3,4-diaminopyridine. It sound very much as if that is what PHOE-01 is.
 
josepdelafuente said:
I googled PHOE-01 (the name Lindus give the medication, in their information sheet), and that led me to this NHS Health Research Authority page about the trial - https://www.hra.nhs.uk/planning-and...lication-summaries/research-summaries/remedi/
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That looks like this may have been from an earlier application as the date is 2024 and the Research Ethics Committee gave an ‘Unfavourable Opinion’ and possibly asked for further information. I can’t find another or updated application though so not sure what to make of it, but presumably they had to get to ethics approval to run this? It would be interesting to know why they got an unfavourable opinion and what they did to resolve it though.

There’s a big document about what that all means linked from this page.

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