CHAPTER 9  ENROLLING IN A CLINICAL TRIAL

DECIDING WHETHER OR NOT TO ENROLL IN A CLINICAL TRIAL

Let’s say that someone is wondering if he or she should enter a clinical trial. There is never a perfect answer but here are a few things to think about.

One of the key questions to ask when considering a clinical trial is what benefit can you reasonably expect from the currently accepted treatment for your disease?

If the response rate for the currently accepted treatment is very good there may be less incentive to enroll in a clinical trial than if the response rate is poor.

Let’s assume you’re interested in exploring clinical trials and you’re sitting with your physician with five different clinical trials that you’re eligible for. Let’s look at some of the discussion points.

It’s probably not true that all clinical trials carry the same probability of success. Some trials are merely testing slightly different chemotherapeutic agents while others are testing radically new technologies. The possibility of a major breakthrough may be higher in the latter trials.

Obviously, by definition, all clinical trials entail risk. There may be unexpected side effects which haven’t yet shown up in previous studies. And, there is no guarantee that the experimental treatments will be effective.

By and large, you’ll probably see a number of trials including phase 1, 2, and 3 trials in front of you.  And, although there are no hard and fast rules about this, there are some generalities.

Phase 1 trials of course are the earliest trials so that if you were particularly interested in a treatment then this would be the earliest time that you would be able to get it. Remember, of course, that the evidence which shows that this new treatment is promising is usually based on animal studies.

Traditionally, in phase 1 studies, extremely ill patients who have failed all other therapies are enlisted. The main goal of the study is to determine if the new therapy is safe in humans and what would be the maximum safe dose to give to patients.

Also, phase 1 studies tend to be small in number and therefore take place at fewer locations.

If you were considering a phase 1 study there would be a few other issues for you to consider.

You need to inquire about the study design. For example, in some studies, groups are randomized so that one group receives a low dose of the new therapy, a second group receives a medium dose, a third group receives a higher dose, etc.

If you really wanted the therapy you may want to be in the group which receives the highest dose. Knowing what group you’ll be in could determine whether or not you would like to enroll in the study.

Also phase 1 trials tend to be short. So the question arises what happens if you take the experimental treatment and, instead of experiencing side effects, you experience great improvement. Although this is statistically not that common in Phase 1 trials it can happen.

Again, study designs are different. In some studies, you would not be able to receive more treatment after the trial ends, and in other studies you would be able to continue to receive treatment.

Some people consider phase 2 trials the best ones to get into. In most phase 2 trials everybody receives the drug so that you don’t have to be worried about randomization. And, you won’t have to worry about receiving an extremely low (and perhaps ineffective) dose like the earliest patients in phase 1 trials.

And, of course, you have the phase 1 data to look at in terms of patient response if there was any.

In phase 3 trials there is the risk of being randomized into an arm of the study that you would prefer not to be in.

For phase 3 studies you have the advantage of looking at the results of the phase 2 studies. You should be looking to see what kind of positive response patients experienced and, if possible, how long the response lasted for.

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