Page 20 - CF Roundtable - Winter/Spring 2026

FOCUS TOPIC: CF CLINICAL TRIALS By: Mariah Caise
PARTICIPATING IN
CF CLINICAL TRIALS:
WEIGHING THE PROS AND CONS
Clinical trials are so important for
the advancement of medicine. While
deciding whether to partake in one is
a personal decision, if I see a clinical
trial that I may qualify for I will always
go through the necessary screening. If
I end up qualifying, I ask the necessary
questions (mainly the potential
risks to me) and decide whether to
take part in it accordingly. I have no
problem being a test subject so that
these pharmaceutical companies that
have developed medications and/or
treatments can have the appropriate
data to ensure their safety or
effectiveness for generations to come.
While it is bittersweet that I had to
grow up in and out of the hospital due
to lack of treatment and medications
available, clinical trials continue to
elevate the quality of life for newer
generations. The newer generations,
hopefully, will not have to endure what
I (and many others) had to go through
with our cystic fibrosis care regimen.
I have been in various clinical trials for
cystic fibrosis throughout the years. I
participated in a phage clinical trial,
a clinical trial for a drug that was
for managing swelling in the lungs,
and a clinical trial that was simply
observational based on exercise.
The different trials are for different
purposes, but they all help assess
cystic fibrosis. The first two were drug-
related trials. In a drug trial you’re
given either a placebo or the actual
drug itself but you don’t know which
one you received until after the study
has ended. For the phage study I was
given a placebo but for the other study
I did receive the actual drug. I think it’s
cool that the test subjects don’t know
whether they are getting the placebo
or the actual drug. I feel like people
overall do better/try harder not knowing
because of the “placebo effect.” I think
some people do better on the placebo
because mentally they think they are
taking the drug in the trial or could be
taking it. In both studies my starting
lung function was not great; I was very
sick and literally wanted to try anything
at that point. In my head, nothing could
hurt. My lung function at the time of
those two drug trials was 30-40% and
the bacteria in my lungs had become
resistant to most medications, so I
was willing to try anything. My lung
function and chest x-rays were better
after the second drug clinical trial, so I
was hopeful it would become available
to me, but unfortunately the FDA never
approved it.
Another risk with clinical trials is
that you could do all that work and
testing for it only for the drug to not
be approved by the FDA. I remember
it being a definite bummer that the
drug never came to fruition because it
did help me. The exercise clinical trial
was purely observational, but I found it
interesting that even though there was
no drug involved, I found myself trying
to exercise more and take better care
of myself physically so I could perform
well for the categories they observed.
In that trial I had a six-minute walk test,
a test to determine how many squats I
could do in one minute, a glucose test,
lung function tests, and monitoring
of my blood pressure & oxygen levels
while doing the exercises. I loved being
a part of that study as the purpose was
to see how people with CF did after
exercising for a little bit compared to a
person who didn’t have CF.
As stated above, you have to decide
for yourself if you should participate
in clinical trials. For me, I look at the
purpose of the study and how long the
study is. The purpose of the study is
important because I need to know if it’s
worth it for me and most of the time it
is if it helps me and the CF community.
Short studies (a few months) are
mostly an automatic yes, and some
studies are for a year or two and those
are more of a time commitment. You
don’t know what life will throw your
20 CF Roundtable Winter/Spring 2026