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ME/CFS patient launches ME/FM CoQ10/placebo DIY online RCT on Mendus - anyone can take part at home

Discussion in 'Active Clinical Studies' started by Sasha, Aug 12, 2015.

  1. Sasha

    Sasha Fine, thank you

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    Very interesting! A PWME is running a randomised, placebo-controlled trial of CoQ10 using the online patient-resource Mendus.

    Just had a quick skim and it looks pretty well-designed.

    A good template for patient-generated science that could lead to more definitive work by researchers if things pan out.

    Great development!

    http://www.mendus.org/q10-study-home.html
     
  2. A.B.

    A.B. Senior Member

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    It's not a real placebo controlled study in my opinion. The blinding is left to the participant. It's easy to accidentally break the blinding under these conditions.

    And there is nothing stopping unscrupulous individuals from submitting false information.
     
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  3. Sasha

    Sasha Fine, thank you

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    It's a real placebo-controlled study if people stick to the protocol - and if they don't, I hope they'd have the honesty to say so. People don't have to choose to placebo-control if they don't want: it's optional, so if your placebo concealment fails then you could just revert to CoQ10 only.

    Yes, it depends on people being honest and people not being weird and stupid enough to submit false information. But as a means of producing results that are suggestive of an effect and worthy of confirmation under independently controlled conditions, I think it's a great idea.

    And when you consider that the PACE trial was completely unblinded and that the study investigators abandoned their planned analysis and have refused repeated FOI requests for their data, I think this CoQ10 trial stands up pretty well to that £5 million, university-conducted trial in comparison.
     
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  4. A.B.

    A.B. Senior Member

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    The organizer could sell a package containing Q10 and placebos which are identifiable only through a randomly generated number. Participants could submit the results only for these numbers. Only the organizer would know which numbers correspond to what. If the placebos and Q10 are indistinguishable using human senses, this would give a blinding as good as professional study.

    The initial investment for purchasing the Q10 and placebos and shipping them could be crowdsourced, perhaps via kickstarter.com.
     
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  5. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    I am afraid this is no good Sasha. In fact I would be seriously suspicious that it is a promotional gambit. You cannot remotely believe trials done this way. I am afraid human nature is far more fickle than you suppose. Trials like this are not just a waste of time, they produce disinformation. A.B.s suggestion would work - you need a manufacturer to produce identical capsules and a code, the key to which is stored in a brown envelope held by a third party. You do not crack the code until all results are in. If we are to get anywhere with decent research in ME we need to throw this sort of thing in the bin.
     
  6. Sasha

    Sasha Fine, thank you

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    I agree that having a manufacturer produce identical capsules and a central party handling the randomisation would be ideal: but I don't know that a patient can organise that and provide capsules without incurring some sort of legal liability or having to go through some sort of ethics committee process.

    I think we need to build on this idea of patient-led trials rather than ditch it. There's such a dearth of research money, and until the trials come through of interventions such as rtx, we're stuck with what we can get over the counter. I do wonder if there's a way for patients to do this well, with the tools we have available to us.

    Not too late to contact the organiser...
     
  7. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    If these are non-prescribable supplements I don't think there are legal issues about providing them. All the patients would need to do would be to approach a manufacturer together asking for real and dummy tablets to be made available. The idea of patients setting up a trial seems perfectly reasonable. But it needs to be done properly. The design as it stands looks disingenuous to me - sounding like a proper controlled trial but being worse than useless. I wonder what the motives are.

    I agree that patient led trials should be encouraged, but only if they real trials rather than marketing. It could easily be done well. If companies really want their products tested they can help by providing coded pills. I doubt they will because they will know that then the results are quite likely to show no effect.
     
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  8. Valentijn

    Valentijn Senior Member

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    Could a patient do that for all of the participants? Basically just remove the supplement from its original capsule and put it in a new one? And the placebo in identical capsules of course.
     
  9. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    I wouldn't recommend that. If people are going to eat the capsules you don't want someone else fiddling with them without using professional levels of cleanliness.
     
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  10. geraldt52

    geraldt52 Senior Member

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    When you think about the ease with which tests such as this could be done, it does seem to suggest that the manufacturers already know the answer, and aren't that eager to see the test done.
     
    panckage likes this.
  11. Jenny TipsforME

    Jenny TipsforME Senior Member

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    I haven't taken part in this study but I've done the Diet Study (see https://tipsforme.wordpress.com/2015/06/17/mendus/ for a write up of my experience) and I've just done the d-ribose study.

    Josh who organises the studies seems very genuine. Any of us can suggest new studies and help organise improved protocol. This is a voluntary, patient led thing with a lot of potential. It is also fairly new and some things will need to be worked out as we go along. I'm not sure how realistic it is to centrally supply the placebos etc, probably one person would have to volunteer and just do that role.

    It is probably important to place this within the Quantified Self movement. Usually we read research papers in order to find out what could be useful for us as an individual. The rigour of the protocol and stats are important because they help me assess how likely the results are to apply to me. I'd want to see large scale, double blinded placebo trials if I wasn't a participant in the trial. However, with Mendus I do the trial myself and see my own stats. I know if I kept the placebo or not. This is really valuable personalised information and it then doesn't matter to me too much what the results were for the other people.

    On the other hand, Mendus also has the potential to produce respected science, with the support of the ME community. The main advantages are not having to wait for research and it doesn't require funding. Think of how slow official research is. I think Nancy Klimas said it usually takes 3 years from an idea to getting funding (if you get funding), the research may take 3 years, 2 years more to publish findings. It seems to take about a month for Mendus ideas to get going, maybe a month to complete. In 2 months you go from idea to personal results. I'd choose that over 7-8 years waiting for results that may not apply to me...
     
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  12. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    I am sure the organiser is genuine in intentions. The problem is that they may not appreciate the bias problems that can arise. These problems are the main reason why studies are expensive - you need a lot of careful planning. 'Personal results' are fine for the individual but they are no help to others because they cannot be generalised. So there is no point in doing a trial at all if all you get is personal results - each person can do that themselves (which I am all in favour of). Organising a trial is only worthwhile if you can get unbiased statistical results that can be used to advise others.

    The idea of trying to get trials up and running without having to put in grant applications for funding is of course fine. When I did the first rituximab study in RA I had no funding. And people may not be aware that Drs Fluge and Mella had no outside funding for their double blind rituximab study. They just got on and did it. For things like oral supplements it would be very easy to set up reliable controlled studies without funding and there is no reason why patients should not run such studies. But if they are not done in a reliable fashion they are likely just to add to the mass of misleading information we now have.
     
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  13. Jenny TipsforME

    Jenny TipsforME Senior Member

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    I think the issue is more working out sustainable practicalities for organising placebos etc within this setup. For example I've been involved in trying to find a placebo for d-ribose. I used stevia but it didn't work well for a few reasons related to the stevia itself (not the blinding).

    You can see here http://www.researchgate.net/profile/Joshua_Grant that Josh is someone who would be unlikely to miss the relevance of bias etc in designing research.
     
  14. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    I was initially very sceptical about motives as indicated in my first couple of posts but as indicated in my last post I am happy to take your word for Josh being genuine about trying to get good research set up. His RG page indicates that he has experience in neuroscience. The trouble is that I am afraid that does not necessarily make him 'unlikely to miss the relevance of bias' or at least to miss just how huge a problem it is for ME trials specifically. If you think about it most of the academic clinicians who should be the absolute experts in trial design, whether of CBT or anti-virals, seem to have screwed up on trial design in the end.

    Human nature is such that whenever there is a possibility of people cheating without meaning to in science, whether researchers or patients, they will cheat without meaning to. This is not a criticism of anyone. It is part of the instinct of being helpful. It is just a disaster for research design. I am sure Josh is aware of the problem in the lab but the way it pans out in clinical trials has a lot more layers of complexity. That isn't to say that the problem cannot be solved. Organising a placebo for a capsule should be straightforward - you can use something completely inert. University Hospital pharmacies do it all the time.

    If this is a genuine attempt to get good controlled trials done cheaply with patient involvement I am 100% for it and would be happy to advise free of charge, but if it is not bullet proof it will be a waste of everyone's time.
     
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