Page 16 - CF Roundtable - Winter/Spring 2026

FOCUS TOPIC: CF CLINICAL TRIALS By: Laura Mentch
INTERESTED IN CLINICAL
RESEARCH FOR CF?
WONDER WHAT’S INVOLVED?
Tobi and Pulmozyme ushered me into my CF life. At my first
clinic visit I was introduced to new medications, treatments,
and instructions. I left clinic that June day thinking about the
new treatments and worrying about the cost of it all without
a thought about what it took for these meds to come into
my hands. In time I began to understand that progress in CF
care was the result of the efforts of many: parents who were
persistent in seeking help for their children and raised so
much money to make it happen, scientists and doctors who
asked questions and moved research forward, and those
with CF who volunteered for studies of new drugs. It has
taken me time to appreciate all of this since I was handed
that first box of Tobi—gifts from the legions of people in the
CF community who have come before me.
When I began connecting with other adults online, I learned
a lot about living with CF. My new friends talked about
research studies, new meds on the horizon, and the pipeline.
Pipeline? In clinic I asked about research studies and learned
that travel was a barrier; I live 700 miles and two states away
from my CF center. When study sponsors began to pay travel
expenses for CF patients in research studies I wanted to be
involved.
Finding a Clinical Trial
Participation in a clinical research study may begin with
an invitation from someone on the CF team. Another way
to learn about research studies is checking out these two
websites that describe current studies for CF and give the
contact information for participating centers:
•
•
The Cystic Fibrosis Foundations clinical trials finder:
https://apps.cff.org/trials/finder
Clinical Trial Connect (CTC) at Emily’s Entourage https://
www.emilysentourage.org/ctc
After finding an interesting or promising study the next step
is to contact the research site and speak with a research
coordinator about the study. When patients meet the criteria
for the study, they can be scheduled for a screening visit to
assess if the study is appropriate for the participant. The
clinical research coordinator becomes the main contact
going forward.
Screening for a Study
At the screening visit the purpose of the study is explained.
Details such as possible risks and benefits of participating
in the study, the timing for each study visit, the plan for
administering the study drug/placebo, assessments such as
blood tests and other clinical procedures, and compensation
are spelled out clearly. The informed consent document will
address common questions like: How long will this study
take? Does everyone get the new drug or treatment? Are
different doses of the study drug being tried in this study?
Will I know if I am getting the study drug? What happens
if I decide I want to stop the study? There will be time to
ask questions and address concerns before signing the
informed consent. Typical tests that will be done at this
visit are spirometry and bloodwork but there may also be
additional specific tests for different studies. The next hurdle
is assessment of the screening visit and approval - the green
light to begin the study. Delays can happen, such as the time
my fecal elastase test was hung up in a hurricane!
Participating in Clinical Research
Like many of our relationships in CF care, clinical research
is a partnership. The study team includes the company
sponsoring the study (sponsor), one or more clinical
research coordinators, a principal investigator (PI), most
often a doctor at the CF center, and the research subject, the
CF patient. Our shared efforts continue over weeks, months,
or years for the active and follow-up phases of the study.
An important step in preparing for a study is aligning the visits
with everyone’s schedule and making sure the necessary
spaces in the research center, the study drug, and equipment
are ready when needed for each visit. Research coordinators
really earn their title. Participants who travel for the study
may work with a travel agency to arrange flights, car rentals,
and lodging with expenses paid for by the sponsor. Preparing
to travel to my center begins the process of a study visit—
packing all the things I need for one or more nights away
from home then usually heading to the airport the day before
I need to be there. A delayed or cancelled flight can throw a
wrench into a carefully planned study day.
For me, each study visit has begun with the CF quality-of-life
questionnaire. Most studies include spirometry and blood
16 CF Roundtable Winter/Spring 2026