Hi All,
I too have been following this thread with great interest. I could have sworn that I read this is a small patient group - 6-20 patients and in Canada. What are the possibilities of entering a clinical trial in a foreign country? Also, isn't phase 1 typically about determining levels of toxicity more than therapeutic benefit which is more of a phase 2 thing. More Brian can expound a little on this.

Hi, Gary --

You are correct both on the size/location and the focus of the study.

As for being able to participate in a "foreign" study, there is no easy answer. It really is a study by study determination. Suffice it to say that there are many, many cross-border studies.

This one, given its size, would probably require either moving to Canada or, perhaps, getting your doctor registered as a study investigator. His/her affiliated hospital may balk at that for a single patient.

As for the focus of the study, yes it is an MTD study and has been designed to give statistical picture of the maximum tolerable dose. It is not designed, from a statistical perspective, to provide insight into therapeutic value.

That being said, to say that it deals in toxicity only is somewhat misleading. If it were designed ONLY to determine toxicity, the eligbility criteria would be healthy individuals. Here they are looking for participants from the patient population that they eventually hope will be therapeutically served. Thus, they must have shown some evidence of efficacy (even if it were only anecdotal) otherwise the ethics committee overseeing the study design would not have approved the design. This of course may not hold true in, say, Belarus . . . .

I am not an MD or even a researcher. I have had a good deal of exposure over the past couple of years to a wide variety of drug studies and study designs including transnational studies, however.