Without getting too far out in front of this (the trial is in the planning stages and its design is not final)...

Different trials have different objectives...this particular one is looking to identify and validate biomarkers of disease progression. It is not an interventional trial testing some particular therapeutic.

So, basically, you want to learn as much as you can about the natural history of the disease and see if there are biological features that correlate with the clinical features of the disease. In an ideal world, if we could identify people at risk for the disease (pre-diagnosis) we would follow large groups of them and collect all sorts of data. Well, we can't identify large groups of at-risk people (at least not yet but that's another goal). And, we can't afford the cost to follow them for long periods of time. So, is there an alternative strategy that can inform us and is cost-effective? Experts generally agree that the greatest rate of change in clinical features (and hopefully biological ones too--that's what were trying to capture) is taking place at the time when a patient goes from asymptomatic to symptomatic so, that's the window we are after.

So, the best we can do today is find people right at diagnosis and start to follow them for several years...it is still common for patients not to go on medication immediately but it is recognized that they will need to at some point. To the extent such a group of people can be followed in large enough numbers, they could provide extraordinary insights. The expectation, again for the purpose of this study, would be to recruit such newly diagnosed pwp and collect clinical, biological, and imaging data (some of which will be before meds and much after) as well as evaluate some of the emerging biomarker candidates using these biological samples, once collected.

My hope and expectation is that we will have much more to say on this anticipated study in the coming months. Please realize that I know that by sharing some of our intentions (in the discussion/ examples of challenges for patient recruitment into trials) I am generating further questions on a new tact. I am limited, however, it what I can share as I pointed out before that the study is still in the planning stages. We are working diligently to finalize the design and funding commitments for the trial and look forward to sharing more soon.

Debi

After the precept/cep1347 trial ended, they started a new trial - postcept - to follow 500 of the original 800 trial participants.

When we entered the precept trial, one of the conditions was that we be "de novo." And the decision to follow us after the trial ended was made because scientists considered us a large 'control group' of pwp which they'd never had before. We've each had 3 SPECT scans, and I think maybe are on schedule for a 4th SPECT scan at Yale.

As a trial looking for biomarkers for PD-- which ran with postcept, my husband participated in PROBE. They'll compare him to me. (My husband consented to give many vials of blood for research -{around 7, I think...})

"PROBE will test three biomarkers in PD subjects and controls to determine their feasibility and potential utility as markers of risk and prognosis for PD. This is a case control study, in which PD subjects will be compared to neurologically healthy controls and disease controls (MSA and PSP). The blood biomarker samples will be drawn once to evaluate blood alpha-synuclein levels as well as collection of lymphocyte mass for array analysis. Olfaction will be measured using the UPSIT for all subjects. The UPSIT will be conducted as part of PostCEPT for PD subjects and will only be repeated in this study for PD subjects in not done within 6 months. Control subjects may also choose to submit a blood specimen for processing and storage at the Coriell Institute for Medical Research, a research resource supported by the NINDS Human Genetics Resource Center.

Follow-up of the PD population over a 3-year period will allow us to evaluate the prognosis for important motor aspects of PD that will occur frequently in this cohort. These complications of PD include motor complications, postural instability, and non-motor impairment such as cognitive decline."
(from clinicaltrials.gov - permission to post from this government site)

Thanks Debi!
Debi wrote:
“My hope and expectation is that we will have much more to say on this anticipated study in the coming months. Please realize that I know that by sharing some of our intentions (in the discussion/ examples of challenges for patient recruitment into trials) I am generating further questions on a new tact. I am limited, however, it what I can share as I pointed out before that the study is still in the planning stages. We are working diligently to finalize the design and funding commitments for the trial and look forward to sharing more soon.”

This thread has been a remarkable example of the type of communication that should be taking place during the planning stages of every clinical trial. If sponsors are having recruitment problems – why don’t more of them talk to patients? Browsing back through this thread they would have learned about issues such as, lack of trust in pharma (just open a newspaper any day), need for communication and education, concerns about placebo controls and sham surgery, difficulty with transportation to trial centers, feelings of being used by academics, difficulty recruiiting early stage PWP, etc.

And most importantly as paula wrote – they need to better understand what PD does to people
“paula wrote “Industry and medical community do need to know the illness much better than they do. Just switching a med is hard work and can cause us to lose something that may not come back. We feel much more and much worse than what you see.”

Debi, we encourage you to share this information with those planning the study, and we look forward to getting more information about the study from you. Maybe it will help solve some potential recruitment problems before they begin.

What would be even better would be for all new studies to include some patients in the planning stages from the very beginning. We are only asking to be a part of the process that hopefully will bring us all to a cure --- soon.
linda

Rick, am curious--what is "whitton's Ex 4 trial? I must have missed reading something important. madelyn

Madelyn-
It is a fast-moving trial that Tom Isaac's group is funding and is the most hopeful thing I have seen. Here is an earlier thread
http://neurotalk.psychcentral.com/sh...hlight=exendin