So, I'm in that Mayo Vancomycin study

My doctor confirmed that yesterday when I had a followup visit. Study will start from January and all cost covered.
I told her that I’m not a believer of Vancomycin in adult patients, but do agree it worked on some patients in certain ways for whatever reasons. She actually agrees on that. But I told her I’ll be very happy to be a trial subject to help clarify this topic.
Of course it will be a double blind study. So we don’t know what I will get. Well here being a scientist has its advantages. I have a friend running HPLC/MS. So he will tell me easily what I’m really get. If it’s Vanco, I’ll keep you guys updated. If it’s placebo, I’ll just quit.

Why not stay in the study of you get the placebo? You would be helping the rest of the PSC community get closer to an answer.


My first thought too - You don’t believe in Vanco. You know the importance of having to have a placebo - based on you being a Scientist. Yet… you won’t take a placebo Vanco?

I’m confused.

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Please consider continuing, even if you get placebo. Drop out must mess with percentage results. It’s only for a few months and it would be very helpful for all to get accurate results. Thanks!

Sorry for the confusion. I mean if placebo, I’ll still stay in the study but won’t take it, which won’t affect anything because it’s just placebo. More importantly, I’ll have chance to talk to those people directly involved in this study. I do have some thoughts about Vanco. Hopefully we can find some places to test them.

DHZ please don’t be selfish. I volunteered my time and dedicated 6 years of my life to getting a trial on vancomycin funded, a nearly full time effort. I helped write this Mayo grant and secured the donation of the vancomycin, at great personal expense and time. I did this because my daughter (dx with PSC/UC in 2012) has been successfully treated with vancomycin and I want others who might be responsive to benefit from this treatment. By seeking to learn what product you will get you destroy the purpose of a placebo trial – the patients receiving the drug should not know as well as those on placebo. You should opt to not learn to which group you are assigned or withdraw from the study. Here is the definition of a Placebo-controlled study from Wikipedia: Placebo-controlled studies are a way of testing a medical therapy in which, in addition to a group of subjects that receives the treatment to be evaluated, a separate group receives a sham treatment which is specifically designed to have no real effect. Placebos are most commonly used in trials, where subjects do not know whether they are receiving real or placebo treatment. Often, there is also a further “natural history” group that does not receive any treatment at all.

The purpose of the placebo group is to account for the [placebo effect], that is, effects from treatment that do not depend on the treatment itself. Such factors include knowing one is receiving a treatment, attention from health care professionals, and the expectations of a treatment’s effectiveness by those running the research study. Without a placebo group to compare against, it is not possible to know whether the treatment itself had any effect.

Patients frequently show improvement even when given a sham or “fake” treatment. Such intentionally inert treatments can take many forms, such as a pill containing only sugar, a surgery where nothing efficacious is actually done (just an incision and sometimes some minor touching or handling of the underlying structures), or a medical device (such as an ultrasound machine) that is not actually turned on. Also, due to the body’s natural healing ability and statistical effects such as regression to the mean, many patients will get better even when given no treatment at all. Thus, the relevant question when assessing a treatment is not “does the treatment work?” but “does the treatment work better than a placebo treatment, or no treatment at all?” As one early researcher wrote, "the first object of a therapeutic trial is to discover whether the patients who receive the treatment under investigation are cured more rapidly, more completely or more frequently, than they would have been without it. More broadly, the aim of a clinical trial is to determine what treatments, delivered in what circumstances, to which patients, in what conditions, are the most effective.

I understand your concern, but what I’m doing won’t hurt the project. As I have said in my previous post, I’ll stay in the trial even I’d be in the placebo group, and I’m not a even believer of vancomycin. And I’m not selfish either. I have participated in all research projects they asked me, even some of them I’m sure just a waste of time and efforts.
As to placebo group or double-blind design, it’s critical. But to a professional, it only matters to certain extent. My experience told me that most of the time, there is no absolute ‘blind’ design or ‘placebo’. Human being is too smart. A typical scenario is that if vanco works in PSC, most likely they, patients or doctors, will know because of the improved symptoms. Then, they are not blind any more. We can have more discussion about science if we have time, but I assure you that I know what’s best for this trial as a patient.

DHZ, if your friend running LCMS does not find evidence of vancomycin in your tablet, that doesn’t confirm that you have placebo. Before LCMS or HPLC methods in the pharmaceutical industry are used to test tablets, they are first validated with standards of the active ingredient, confirming that the method itself is capable of detecting the active ingredient. In your case, the presence of an ion with mass identical to that of vancomycin is conclusive evidence of its presence. But a negative response could mean that vanco isn’t there, or it could mean that your analyst friend didn’t have the right solvent system to properly extract this molecule from the tablet, or maybe doesn’t have the optimal instrument conditions for ionization of the molecule. You could end up thinking you have placebo and then not taking what is actually real vanco tablets, and screwing up the answer to the question about vancomycin for all the rest of us.
And finally, even if a study does not result in a cure, it is not a “waste of time”. even negative results play a part in the progression of knowledge about PSC.