Things to ask before participating in a trial

[spectda]

Participating in the right trial can sometimes be great opportunity, although it is important to understand the constraints and drawbacks as well as the benefits.

I am grateful to have been included in a boceprevir trial and would still have made the same decision to participate if I had it to do over. At the same time I have learned a lot about what I should have asked prior to tx and what I gained and what I gave up from not treating privately.

I thought people might list the kind of questions they would ask of the trial sponsors before making a decision to participate. I think it would be helpful for anyone considering the trial option.

- Dave

[270217-1b]

you are doing an awesome job of getting this information into one place for us non-savvy users.  Thank you!  I have an appointment for a study on Tuesday and am trying to know what to ask.  This is VERY helpful as I haven't a clue.

[willbb]

Beat the Beast...excellent question for them...all trials to my knowledge are doing these tests .as part of their info.gathering.,however , Ask for  it in writing at the start ..."the date you will receive this valuable information.

Will

[BeatTheBeast]

Can I get a commitment to receive any sequencing (population and clonal if available) results done on my virus in the case of failure?  (Push strongly for this given the importance of understanding resistance as best one can.)

[coeric]

how often will i need to come into the clinic?  how long will the appointments take?  can i schedule the appointments to suit me schedule?

[spectda]

please add anything that would be helpful for someone to ask pre-trial of their doctor
-Dave

[Michigan57]

I just cut and pasted everything I could gather from the above posts about clinical trials.  I am calling a Hepatologist today and hoping that I end up in a clinical trial for monetary reasons since I don't have the greatest insurance.  Thanks for all of your input and I've added this thread to my watch list so I can update myself with any new information you add.  You guys rock!  Thanks!

[spectda]

Being an investigator in a trial puts the treating doctor in the usual situation of having conflicting allegiances to their patients and the trial sponsor. Although trials have fairly strict guidelines, many decisions are still left to the discretion of the doctor. When considering a trial I would also consider the doctor's personal approach to treatment.

In the trial I participated in and because of my own response the doctors discretion greatly influenced the balance of dose reduction/increase vs rescue drugs. This of course influenced my chance of success as it did for many others in the same trial.

[spectda]

Consider having an IL28B test done privately before participating in a trial that includes interferon and there is a possibility of getting a placebo rather then the study drug.

[foofighter]

Hey, I get that "chimp in a mirror" stare all the time these days. In spite of a negative trial experience I have continued on in two follow-up studies (one on mutations!!!) thinking it may get me some brownie points with Merck so that someday I might gain access to my records.

Last visit with the trial doc when I asked yet again, "Any news on mutational info for me?" he told me, "Oh no, they are busy marketing now, they won't have time to get any info to trial participants."

This is the same Doc that said to me in the fall, that he thought that the drug company not providing the data to me was reprehensible behavior.

Now that the whole team has gone over the trial consent form line by line, and found that it doesn't really say they will release it, he seems to have forgotten he is the one that said they would.

I contacted the IRB (listed in every trial consent form) to see if I could get at least some of my info and this is the last response from him:
+++++++++++++++++++++++++++++
Information confidentiality is generally described in detail in consent
forms and study protocols. Information access/ownership raises many
different issues. For example, a recent study on Huntington disease (a
genetic neurologic illness) handled this in a convuluted way that for many
reasons is optimal: the study tested subjects for the genetic fingerprint
of the illness, said that this would NOT be shared with the subjects, then
offered a free genetic test (the same test, but to be ordered in clinic and
to appear in the subject's medical record) for subjects that wanted it. I
give you this as an example of the complexitity in this area. To convince
you why this was a good plan is more than I need to get into.

I think I can be your advocate in requesting information (though not as
effective as Dr. Pain, who knows the people involved, which is why I have
let him start.) If you want me to request something after you have heard
from him--test results or a clarification of their policy--I will be happy
to. But I can bring the regulatory weight of my IRB down on the sponsor if
they have made a mistake, and I am not seeing that here. My request would
be a request.
+++++++++++++++++++++++++
I haven't gotten back to the IRB yet. I guess you practically need a lawyer to parse exactly what the info means. Remember, your doctor may not completely understand what it means either. I don't think my doc lied to me, he thought we would get the info. Now that he knows he made a mistake, he won't admit it. Just the monkey face.

And he is GROUCHY now that I have gone to the IRB. His last remark to me, was "Why do you care about mutations anyway, it's a moot point."

I gave him the monkey face and said,  "What are you talking about?" He said, "Interferon makes you too sick, you cannot treat again."

Could have knocked me over with a feather. First time I heard that come out of anyone's mouth. Guess he is gonna get me back for going around him.

So I high-stepped it down to medical records to say I wanted NOTHING from Dr. Pain released to anyone but me. Can you imagine? It could be a liability thing for my hepatologist, or maybe the insurance co could use it as an excuse to not pay to treat me. Oy!

A bummer of an experience in my situation. Just like everything with this bug, every situation is different.

[spectda]

I am doing a half assed job of trying to make the information from all of us more concise. Collectively perhaps we can create a document that would be easy for someone considering a trial to reference. If anyone feels so inclined to put this together I think it would be very valuable.
-Dave



What access do I have to lab results during the course of my trial participation? At what intervals are the labs done, do they always include viral load results, when are the viral load results disclosed?

Will I receive hard copies of lab results, and how are they transmitted to me?

Are adjunctive drugs such as epo, GCSF (Neupogen) etc allowed or provided during this trial?  Is there use encouraged rather then dose reduction?

What are the parameters for intervention in the event I develop cytopenias such as hemolytic anemia, neutropenia, etc.

I understand genetic sequencing is generally conducted as part of HCV drug trials to study viral mutation. Will this data be made available to me, and if so, at what time?

What are the statistical odds I will receive placebo in this trial?  I found this very important. I had assumed that the chance of being randomized into different arms of a trial was always divided equally but this is not always the case.

For those patients that don’t respond fully to the trial (SVR) because they received the placebo will the study drug be made available to them at a later date (roll over program)? (I assume if this is the case it would be after the trial is unblinded which is normally after all results from the last participant have been collected.)

In the event I don’t achieve SVR through this study, how will this adversely affect future HCV therapy? For instance, will I be prohibited from future participation in other DAA clinical trial due to drug resistance, etc?

Who will directly manage my care throughout my trial participation; Physician, NP, PA, RN, etc

When will the study be unblinded?

What are the response parameters for determining whether treatment will continue, how long it will continue for, or if it will stop?

[willing]

useful list. Not sure if already covered but understanding the  discontinuation rules seems important. At what point and why  would you boot me out?

The fact that drug cos can collect blood/tissue from you and not release your test results seems to violate a fundamental lab-rat bill of rights. Anyone know whether this has ever been challenged in court?

[Susan_1a_CC]

I am currently in a trial and in addition to the good points above I would add this: what precisely is the information that will become available when the study is unblinded?  In my case this was at week 12.  I was given the result of viral load for that week but no results for earlier weeks, and also, I won't get another VL until the end of study.  I had expected to receive all possible numbers - not so.  Had I realized this, I would have had my primary doc run tests at key times in order to make my most informed decisions about continuation in a trial.  What I have received is copies of basic labs - CBC, chemistry, le's.

Will participation in this trial limit my future participation in trials and how so?

Also, will I be seen by a physician or a PA?  Was surprised that even though my GI is running the study, I haven't been seen by same.  The physical exam given at set points is hardly thorough.

I'll reiterate that the trial isn't for the participant first, it is for the study drug.  Read the "perspective" carefully and take the time to digest it.  Also, I agree that when one is trying to determine the efficacy of a drug it is only logical to pick your "healthiest" candidates in order to limit variables.

[SteveOC]

Regretably I hardly asked any questions at all and just went with the flow. In hindsight, armed with what I now know, I would of asked dozens of specific, indepth questions covering the whole spectrum rather than the generic ones I posed.
   I'm certain I would have still participated at the time but it was actually experiencing the Tx sides and lack of financial planning that has concerned me the most about how trials are conducted with your average John Q Public. From what I have read and been told a sizable percentage of trials/studies are not the least bit transparent in many cases. Yes I signed the disclosure which I have read numerous times but it desribes the trial protocol only.
My point is to ask many questions and don't settle for half-a$$ answers and then make the best, most informed decision in your best interest.
My chief concern is ability and availablility to participate in future non-trial settings where curing me will be the objective rather than to study me.....

[RobertBeWell]

I suspect it's probably they don't want to deal with any complications, and perhaps someone with more liver damage would fall into that category. They want patients who are likely to complete the trial, so they can gather the required data. If someone quits, it's a waste of time for study purposes - or certainly not as good from a clinical standpoint.

Of course, they do want the best results possible for their trial, but I think that's just gravy.

But in the end, isn't what works best for "good patients" also going to work best for "tough patients" ???

And as several others have mentioned, the primary goal of a drug trial is often not to cure patients. The goal of a drug trial is to collect data. If someone gets cured, great.

My study lists several goals. None of them say anything about cure.

[willbb]

  Unc....Yes they want to "cook the books" for the obvious reason.The best customers,so to speak have the best chance of success therefore best shot at getting the drug approved.

That is why I have maintained all along the success  pecentages of these trial results.while surely impressive,the true test on success will be when the "not so best customer" buys the product.

Will

[uncledudeness]

i wasted one year trying to get into clinical trials. at the end of the year, and 3 rejections, my HEPA told me that i was too late as a Stage 3 maybe 4 to be considered for most trials. no parting gifts or parking validation ...i asked him why he didn't tell me that prior to applying....anyone ever see a chimp stare at a mirror?

i was then told that most trial sponsors are looking for candidates that have a higher degree of responding to the test meds. his phrase was "cook the books".

in the clinical trial criteria section, they don't list this as an exclusion. i was told by BMS that that is proprietary information.....sure it is....

[FlGuy]

It would be good to know the Phase of the trial.  The goal of a trial is not necessarily to "cure" people.

In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.

In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

[Bill1954]

Hi Dave-

Here are a few questions I thought of for someone earlier that is considering trial; good thread, by the way. Hopefully we can get a dialogue going here, and other people will contribute their thoughts too. I never took part in a trial; these are questions I’d have asked in advance if I was thinking of it myself:

What access do I have to lab results during the course of my trial participation?

Will I receive hard copies of lab results, and how are they transmitted to me?

Are adjunctive drugs such as epo, GCSF (Neupogen) etc allowed or provided during this trial?

What are the parameters for intervention in the event I develop cytopenia such as hemolytic anemia, neutropenia, etc.

I understand genetic sequencing is generally conducted as part of HCV drug trials to study viral mutation. Will this data be made available to me, and if so, at what time?

What are the statistical odds I will receive placebo in this trial?

For those patients that don’t respond fully to the trial (SVR) will the study drug be made available to them at a later date (roll over program)?

--Bill

[spectda]

Thanks Will,

Without the rescue drugs I would not have gotten past the first couple of months. Its a very important question which many of us missed because we did not know enough about tx to ask it.
- Dave

[willbb]

  Great post Dave...As you know I was in a trial also and did ask most of the questions I wanted answered prior ,however  there was one I neglected to ask...

  1)  If no rescue drugs are  given ...what action is taken in regards to decreasing Hgb.,whites,platlets etc.  and at what levels do they intercede?

There are many more questions to be answered  but that is the one I missed.

WILL