Selection process for clinical trials

[Conductor71]

So we have a paucity of PWP for clinical trials...ha! I am applying for NIH GDNF trials and apparently I have a lot of company, but there are only 28 openings. Looks like maybe we are just more selective.

Anyway does anyone know what happens when more people qualify for a trial than there are open slots? Do they try to balance the trial so being a woman might be an advantage but being youngest may hurt chances? Anyone know or have experience with this?

Laura

[lurkingforacure]

Originally Posted by Conductor71

So we have a paucity of PWP for clinical trials...ha! I am applying for NIH GDNF trials and apparently I have a lot of company, but there are only 28 openings. Looks like maybe we are just more selective.

Anyway does anyone know what happens when more people qualify for a trial than there are open slots? Do they try to balance the trial so being a woman might be an advantage but being youngest may hurt chances? Anyone know or have experience with this?

Laura

No experience but will hazard a guess that they will favor picking PWP that increase the odds of a successful outcome. We've all read the stories of how drug companies "cherry pick" those patients most likely to make their experimental drug/protocol/procedure a success.

Case in point: we were invited to participate in a trial. Excellent health sans PD, young onset, very proactive in terms of exercise, etc. I would think this is particularly important if the trial involves any kind of surgery. Who wouldn't want to stack the deck with the "healthiest" PWP?

To be fair, I get that if a lot of participants have had PD for decades, this could make the results harder to read, and increase the risks of adverse events, but the reality is that most PWP have had it for a very long time. So trying to just pick young onsetters, or those who are newly dx'd, etc., does not really represent the PD population.

These are just my thoughts, as I said, no personal experience.

[lab rat]

I have applied for three clinical trials. I have a low white blood cell count but other than PD am healthy. I take nothing but Azilect.

Since they were testing new drugs, they all turned me down.

[Conductor71]

Originally Posted by lab rat

I have applied for three clinical trials. I have a low white blood cell count but other than PD am healthy. I take nothing but Azilect.

Since they were testing new drugs, they all turned me down.

Lab Rat,

Welcome to the NT crew. See this just gets me. I honestly think that it is now fair to question the low participation lament of the research establishment. It is beginning to seem like an excuse. What will they have to say given all the new MJFF recruits?

The very fact that the NIH will turn people away for GDNF while many other trials close speaks volumes. It says we are a bright group who choose what has the most potential to make a significant impact on our condition. It says enough with the "me too" drugs.

I remember first getting the diagnosis; I naively thought I would make an ideal research participant because my family has a history of tremor. Nope. Reject! You must have a parent diagnosed with PD!?! Ummm okay. What about auto-recessive forms of the disease?

Just serves how much we need patient based research. This might also skew Placebo Effect in us. Think about it; if a person is fortunate enough to make it into a highly competitive, desirable trial, aren't they then more likely to feel a benefit from a placebo? We can't know, but it seems plausible.

It is crazy that they can be so choosy. Isn't that selection bias?

Laura

[Dianna_Wood]

I enrolled in MJF trials list and was recently enrolled in a genetic research study. I enrolled when they first started up.

Dianna

[Songfellow]

Some of it might at first look seem fishy but I feel better about it than I once did.

There is a company here that is in the business of running trials and reporting back to whichever drug company had retained them.

In my experience, the study doctor makes the decision of who the most eligible candidates are. I've never gotten the impression that a drug company was "rigging" the results beyond their initial study criteria. I even have doubts that they could even if they wanted to.

As a final note, I'm pretty sure that all of those nasty side effects that are reported aren't welcome news to a drug company!

Steve

[lab rat]

The way the study doctors explained the problem to me is:

1) Without biomarkers, they are not sure if the study participant has PD. (This is especially true with recently diagnosed applicants)

2)Things like an unexplained low white blood cell count bring up safety and liability concerns.

3)Some applicants UPDRS (motor) score normally fluctuate too much to detect a clinically important difference due to a study medication.

4)A lot of study participants that think they are getting the placebo drop out.