Responding to Roxanne, and to get conversation
going again (it starts up every so often) about clinical trials, I'm
going to write some stuff. But I want to start by emphasizing that
it's different for everybody.
For starters, where are you at in your 'journey'
as we say?
Is your doc offering and/or recommending a
clinical trial at your institution? Is it a Phase 3? Is the
projected total enrollment high? Like in the hundreds if not more?
Typically, that means that there are two (or more) arms (variants),
one of which is the current standard of practice, i.e., what you'd
get it you didn't sign up for the trial. The other arm includes the
experimental drug or technique or schedule. You don't get to choose
which you're on. You're assigned at random to one or the other.
Sometimes the trial is double-blinded, which means you don't know
which arm you're on and neither does your doctor. Other times that
In principle that's the best kind of trial to be
on, because it implies that there's lots of evidence supporting the
new approach, as well as lots of evidence for its safety. But guess
what: about half the time, it turns out not to be any better than
the old approach, the standard of practice. Rarely does it turn out
to be significantly worse. So whichever arm you get assigned to, you
can feel that you're getting good care. And you can take some
satisfaction in furthering knowledge that will help other women down
I want to add one thing: I would be inclined to
prefer a study that includes some kind of analysis of the cancer
tissue, not just determining what type of cancer it is.
Remember, there are exceptions to every general
statement I'm making here, and if I went into them all, this would
I haven't been in any Phase 3 studies. Why not: I
wasn't offered one in my first-line treatment, nor after my
first-line treatment was completed and I was NED (no evidence of
disease), nor at my first recurrence, seven months later. Those are
the landmarks when you're most likely to be eligible for a Phase 3
study. I knew nothing about clinical trials, and nobody told me
anything. I wasn't on this listserv.
Most trials are Phase 1. These are studies
designed to assess safety. And by the way, they might also see
whether it helps anybody. Eligibility for a Phase 1 is much less
restricted than for a Phase 3. Phase 2 studies are done if a drug
makes it through Phase 1 with a good safety profile, and looks
promising enough to move forward with.
I've been mostly in Phase 1 studies (9 of them,
with some additional wrinkles), and one Phase 2. The Phase 2 was
ineffective, and that drug never made it to Phase 3. That often
happens. More of my Phase 1's were ineffective than effective, or I
was dropped early because of what were considered serious enough
Adverse Events (AE's). That ends up meaning that I didn't spend much
time in trials that weren't working for me, nor in trials that were
putting me at risk. I generally spent several months in the trials
that did work, staying until my cancer became resistant to the trial
But that's me. I don't get remissions any more. My
cancer finds a way around the treatment within a few to several
months and comes roaring back.
And that's why I have 10 clinical trials under my
belt, so to speak, and I think that's why I've just made my 8th
cancerversary. It makes sense for me to try to lengthen the list of
"options". If I buzz through the list of drugs FDA-approved for
ovarian cancer: Carbo, Taxol, Doxil, etc, some of them working, some
not - I get very quickly to the end of the list, and have gone
through it too quickly to go back on the ones that worked, because
my cancer's still resistant to them. It's generally thought that the
longer it's been since your cancer has seen a particular drug, the
better, especially platinum.
And it's more complicated than that, depending on
your medical status, your particular genetics, the genetics of your
cancer, drug combos, schedules, delivery methods (IV vs. IP .). But
the upshot is that the list isn't long, and the list of the ones
that have some likelihood of working is very short. So I'm motivated
to look for Phase 1 and 2 (if can could get them) clinical trials. R
asked, how do I look .
Occasionally the treating physician will recommend
one. That happened to me once. It probably happens more now than it
did in my early days.
My first move, when I suspect/know that what I'm
on isn't working, is to go to the National Cancer Institute
database. I go to clinical trials.gov. (note clinicaltrials.gov is
actually the FDAís database not the NCIís but I didnít want to
change her original text)
Searching is pretty straightforward if you
remember how to use AND's and OR's. You can restrict your search by
geographical location (city, state, zip code), by institution name,
condition, age group (e.g., NOT children), most things you can think
of. If you're not comfortable, your local librarian, among others,
will help you.
I evaluate whether I'm obviously excluded (more
and more these days, I am, often because of number of prior
treatments). And if I'm interested (more on that below) and not
obviously ineligible, I email &/or phone. Sometimes the contact
listed is at the company, and they'll refer you to the study site.
You hope you get to an experienced trial coordinator/nurse. This is
all assuming you're comfortable reading and evaluating descriptions
of clinical trials. If you're not, find someone who is to work with
you. Or put some time into it and develop your skills
What happens next varies, but if this trial
seriously looks appealing to you, or the trial coordinator tells you
about something else that looks appealing, it ends up with an
evaluation at the institution where this trial is running, and
recommendations for this and/or other trials at that place. It may
be worth a trip out of town if you have the means or insurance
coverage for travel, and it's an active research physician in an
institution with lots of clinical research going on.
The best place to look at first is, of course, the
major cancer treatment center where you're usually treated (or if
you're not, the easiest one to get to). Most cancer centers now have
Phase 1 departments, which could be called something like
"Developmental Therapeutics" or "Phase 1" . You make an appointment
and if it's your own institution, you don't need to go through
gathering all your records because the Phase 1 doc already has
access to them.
Here's an important point: the data base isn't up
to date. Even if there isn't a trial that looks good to you at your
own institution, go see the Phase 1 doc anyway. Maybe something is
about to start or has just started or a slot has just opened up.
Phase 1 slots are like Brigadoon. They appear and then disappear.
This is because of how Phase 1 trials are structured.
(I once consented at lunchtime, and learned after
lunch that the slot had been taken by someone at another
institution). Quite normal. (I bolded this because I think itís an
Same thing for other major cancer centers,
especially the big guns, if you can consider going there for
treatment (you fly a small plane, your cousin can put you up, you
used to live there and all your old friends still do .). Get
evaluated and then you will be made aware of what studies you might
qualify for. Of course, studies AT the National Cancer Institute, in
Bethesda, MD (Washington DC) have plane tickets paid for, at least
once you're enrolled in a study, plus some defrayment of other
Or, consider this a second opinion, see the famous
guy at Famous Cancer Center, and get her/his recommendations for
treatment plus referral to the Center's Phase 1 department. (Though
sometimes famous guy just evaluates you for his/her studies, so find
out whether there are other studies for which you might be eligible
and find out what you need to do to be considered for them, if they
Being seen at Phase 1 departments also helps get
you on their radar, acquaints you with the nurse/coordinators, with
the particular wrinkles of the institution (you know, there is only
ONE way to do things and it's the way WE do it. Different everywhere
you go.) Then you can call in the future to see whether/what studies
that may interest you are going on/about to open up. If time has
elapsed, you may need to be seen again to get on the waiting list.
There are waiting lists for drugs that are 'hot', like PARP-inhibitors.
Again, it isn't all represented in the data base, so talking to the
trial coordinator, who knows you, is a faster route to finding out
I keep saying "if it interests you". What I mean
is I look at whether it's something that there's buzz about, or
something that seems to be garnering interest in the literature, how
long it's been going on (i.e., not going on, like in 6 years they've
accrued only 4 of the 23 subjects they need), what the schedule of
treatment is (you can't fly there three times a week, etc), what the
side effect profile is, etc. Also where they are at.
Phase 1's enroll patients three at a time, if that
functions ok, then three more . until they arrive at the Maximum
Tolerated Dose (MTD). Then they enroll an "extension cohort", a
number of people whom they give the MTD to. All things being equal,
you'd prefer to be part of the extension cohort, just like you'd
prefer Phase 2 to Phase 1. Does this study look at biomarkers or do
genetic analyses or something besides just round up people who can
put a check mark next to "ovarian cancer" or even just "solid
tumor"? Studies on
patients with solid tumors (ovarian or lots of
other things) could be helpful to you, but all things being equal,
seems preferable to go for a study designed to address your type of
cancer, or people with characteristics you share (such as a
Consent: Signing consent (aka Informed Consent,
aka IC) doesn't obligate you to ANYTHING. It obligates the
investigators not to request that you do something the consent says
you don't need to. You are ALWAYS free to ditch the study. The PI
(principle investigator) is always free to drop you from the study
too. But the investigators WANT subjects. They'll only drop you if
they determine that the study endangers you, or it clearly isn't
working, or their funding is terminated . Of course, don't sign
consent if you don't intend to participate in the study!
Before you sign consent, an investigator will tell
you about the protocol (what they will and won't do). Listen and ask
questions. All things being equal (right, they never are), you'd
like a study that allows for dose reduction if needed, you'd like to
get a sense that the study will be conducted with serious regard for
your welfare. You need to make sure of what to do, whom to call in
case of need.
As always, you want to feel confident in the doc
and feel that you can communicate, understand and be listened to.
Phase 1 docs are usually personable. They can't afford to turn off
This is immensely long, though far from complete,
but I want to make sure to add: run the options past your own doc.
If you have other docs who know you, a super-brief email asking them
for their pick among 2 or 3 options (maybe 3 lines of text?).Ē