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Sent: Thursday, July 17, 2008 3:49 PM
Subject: Re: [FAPG] Ediison A-0001 Phase I Trial Participants
I'd like to follow Paul's excellent comments with some information FARA has obtained about the A0001 trial. This information reinforces Paul's points about the format of this trial as compared with the Idebenone trial and may help answer some of your questions.
The phase I trial of A0001, as stated in the release, is in healthy volunteers. It is being conducted by a Contract Research Organization (CRO) that was chosen by Penwest Pharmaceuticals and with which FARA has no dealings. This is, by the way, the more typical way phase I trials are conducted (by CROs in healthy volunteers that they have on their rolls). I believe phase I of the Idebenone trial was conducted, atypically, in FA patients primarily because Idebenone had already been "in a lot of people." Idebenone had been tried at low doses in a number of other diseases such as Parkinson's, Huntington's and Alzheimer's, and had been taken by a lot of healthy people as an anti-aging or brain-function aid. So, the FDA agreed the Idebenone phase I could be in FA patients. Similarly, the iron chelator Deferiprone and EPO have both been "in a lot of people" with other indications, so the phase I work, or pilot studies, in those two drugs have been conducted in FA patients.
A0001, however, has not been "in a lot of people" so the FDA suggested beginning with the typical phase I approach - in healthy volunteers. FARA has not seen the protocol for this phase I study of A0001 and would not usually need to see it. My understanding is, though, that carrier status would usually disqualify a potential participant. I believe healthy participants are usually young students with no involvement at all with the disease in question. CROs normally recruit the healthy volunteers very quickly from their own rolls and ads and have no trouble doing so. This A0001 phase I trial, for example, is to use only 60 healthy volunteers and 10 of them were dosed on the very first day (last Friday).
As Paul states, phase I of this trial, like all phase I trials, will focus on safety. Like the phase I of Idebenone, it will look at safety in a dose-escalating manner and will attempt to identify the maximum tolerated dose. Data from this phase I trial will be used to establish safety of A0001 in humans and will help instruct selection of the best doses to use in the phase II in FA patients.
We will ALL be VERY much involved in the phase II of the trial, of course, and FARA will let everybody know about timing and inclusion/exclusion criteria, etc. We are in constant contact with the drug companies involved, on a daily basis, and will be working very hard to accelerate their timelines. FARA's Scientific Advisors and Board of Directors meet at the end of next week and we will review, based on reports we are now receiving from all the drug companies, the schedules and timelines for all the clinical trials currently underway and coming up. Of course, we will let everybody know the results and how you can help.
In preparation for all these clinical trials, please make sure all FA patients are signed up in the FARA Patient Registry and for FARA's electronic bulletins and newsletters.
Hope this helps.
Warm regards to all,
(Ron & Raychel; Keith-22-FA; Byron-24-clear; Stuart-19-carrier)
Please register in the FARA Patient Registry at http://www.curefa.org/registry/
and for e-news at http://visitor.constantcontact.com/email.jsp?m=1101190303489
From: Paul Konanz
Subject: [FAPG] Edison A-0001 Phase I Trial Participants
Reminder on the Edison A-0001Phase I Trial
It is wonderful news that one more possible FA treatment is starting FDA trials, with the announcement of the Edison A-0001 Phase I trial.
One note is that this Phase I trial is to completely focus on safety in human subjects using non-FA-involved participants. In the press release
it says, "The phase I clinical trial just initiated by Penwest Pharmaceuticals will be conducted in healthy volunteers...".
For sure this means no FA'ers will be involved and very probably no carriers (parents, siblings, etc) either. I have little information
on the protocol at this time (this trial is not on the NIH trial page). Looking at the Idebenone Phase I trial as an example can give you a feel
for what is involved in a safety-testing-only trial. That trial was split into an A and B sections, with A being a single dose to volunteers with
close scrutiny for some time after the dose, and B was several doses up to 75mg/Kg/day for one month with the same close scrutiny.
DO NOTE FOR IDEBENONE THEY USED FAers.
They aren't with the A-0001, according to the press release. If you are interested, the Idebenone Phase IB trial description can be found here,
So, please don't try to volunteer for this A-0001 trial if you think you have one or more bad FA-related genes. Several of the FA
organizations have received many inquiries but have little information because they are not involved and we don't qualify anyway. :-)
These organizations did comment how they appreciated the interest and willingness of the families to so quickly contact them!
It demonstrated how closely tied in to advances in research we are.
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