Clinical trials are tests of drugs or medical devices to
determine if they are safe to be used on humans and whether they are
effective. The government requires clinical trials be conducted and
evaluated before drugs are approved for human use. Before any drug or
device can be administered or used by anyone, the sponsor must provide
the FDA with the results of laboratory and animal research. Once
approval is given the clinical trial process can begin. Trials are
usually done in three parts or "phases".
A Phase One Trial is the beginning of the testing process. The
drug or device is tested on a small number of healthy volunteers. In
CF, sometimes a waiver is given so that the testing is done on people
who have CF, but are generally fairly healthy. The results of a Phase
One trial show what happens to the drug in a human, and whether side
effects occur or change as the amount of the dosage is increased. A
Phase One trial usually takes from six to twelve months.
A Phase Two Trial is one done to determine if a drug that was
proven safe in Phase One is also effective. In a Phase Two trial, people
who have the disease that is being studied are given the drug to test
if it has any effect on the disease. This phase is done over several
months and another factor is added. The drug is "randomized", meaning
that some people get the drug and others get a placebo that looks just
like the study drug but has no active ingredients. In this way no one,
even the clinicians studying the drug, know if the patients are getting
the actual drug and so the results are not biased. A Phase Two trial
determines the short-term safety and effectiveness of the drug or
A Phase Three Trial is a much larger study done with many more
people and for a much longer time. The purpose of a Phase Three trial
is to determine further safety, effectiveness, and proper dosages of the
Another very important part of clinical trials is that they are
performed on people who have agreed to participate. This concept is
called "Informed Consent". The voluntary consent of human subjects is
absolutely essential. Any clinical investigation involving human
subjects is required to obtain an informed consent from the subject
prior to the study.
The consent is an actual document designed to give potential subjects
all the information they need in order to decide about whether to
participate in a trial. It is meant to ensure that they understand all
aspects of what is being asked of them, and that there can be discussion
between the subject and the investigator. The Informed Consent must be
signed before the subject can participate in the clinical trial.
Clinical Trials: Why Volunteer for Research?
By Zoe Davies, RN, MS, PNP
The only way we can develop better treatments and, hopefully a cure
for CF, is through good clinical research. Clinical trials are
scientific investigations involving people with CF to answer specific
questions about a new treatment or a new way of using an old treatment.
Medication trials test a specific use for a specific medication.
During clinical trials, researchers closely evaluate the safety and
efficacy of particular therapies in study volunteers under very
controlled circumstances. This process can last years, but it is
important that each new drug and/or device undergo very careful scrutiny
to provide patients with medications and treatments that are safe as
well as effective.
To get FDA approval of new medications for use in CF, they need to be
tested in people with the illness. Therefore, it is imperative that
individuals with CF participate in the clinical research process. Each
and every volunteer provides important information that cannot be
gathered without their contribution. We need to encourage all patients
with CF to become actively involved with this process so we can continue
to find new and better ways to care for them.
On a more personal level, patients who participate in clinical trials
are followed very closely, and have thorough and frequent physical
exams and laboratory evaluations. This in itself has the potential to
improve their health. Many participants find that they have increased
adherence with all types of therapies that continues on after the
The reason for volunteering for research are as varied as the people
who volunteer, but one quote from an adult with CF was really striking.
He said, "What if the people before us had not wanted to try new
things?" " We would only be living to age six instead of well into
adulthood." Clinical trials are for everyone. Check our clinical trials page
now to see if you are eligible for any of the current trials, or call
650-498-5315 and leave your name number so we can call you when future
studies become available.
Be Part of the Cure: CF Research Team at Stanford
Research Participant Testimonials
Having the opportunity to participate in research studies has
been such a blessing. Throughout my seventeen years of life, I’ve been
in many studies.
Whether it’s just filling out a quick survey, giving blood work or a
sputum culture, or having to do months of test- ing out a new medicine,
whenever I am asked to be in a study, my answer is always yes. The only
way new drugs can get FDA approved and get out onto the market is for it
to go through the clinical trials process. Clinical trials can’t happen
and new medicines that can potentially im- prove the health of cystic
fibrosis patients can’t be made available without “people
participation”. That’s why I say yes to every study I am eligible and
asked to be a part of.
Living with a life threatening disease is hard work. I can go from
100% lung function one week to 80% the next. I am compliant with my
daily treatments but that doesn’t guarantee that a new bacteria won’t
enter my lungs. When
Members of the CF Parent Advisory Council are working to identify
areas in which improvements can be made to better support patients with
CFRD. If you have suggestions, or want to share your experience, we want
to hear from you. Either go to the CF Center website
(http://cfcenter.stanford.edu), where there is a link on the homepage to
leave anonymous feedback, or email Siri Vaeth, Council Lead Parent, at email@example.com. The input of Center families is encouraged.
I’m asked to test out a new drug, I feel like a kid on
Christmas. Even though I don’t know if I am getting placebo or the real
deal, it’s still just as exciting knowing that I could be receiving a
medicine that could possibly change my life. If given the opportunity to
change someone’s life forever, wouldn’t you do it? Participating in
research studies is the best decision I have ever made and I hope that
maybe just one person will read this and change their mind about be- ing
a part of changing and improving lives.--Julie Phillips
My son Brian was in his first research study before he turned 2. Not
only did I want him to benefit from using TOBl at a young age, I wanted
to contribute back to the CF community. It is through studies that new
therapies are developed, and these therapies add years to Brian’s life.
By being in a research study, Brian can pass that same gift on to
Brian is now 14 and continues to participate in studies when he’s
eligible. For each study, coordinators explain the process not only to
me, but also to Brian. The coordinators then patiently answer our
questions until we are completely satisfied.
When concerned about possible risk to Brian’s health, his doctors and
coordinators always address our concerns. The care givers want the best
for their patients; they immediately end a trial at slightest
indication of harm. Brian is regularly reminded that he is free to end
the study at any time.
Research coordinators work alongside doctors to keep all patients in
the best of health. Working together, we move closer to our common goal,
finding a cure for cystic fibrosis. ---Linda Burks
Being in a clinical trial is like getting to be a scientist. You get
to see some cutting-edge medical equipment and procedures, and you can
ask all the questions you want about it (and get all the answers!). They
explain everything that’s going to happen so there are no surprises.
It’s great to feel like you are helping others with CF (or maybe even
you, someday) because of your participation. Volunteers like us make
clinical trials possible. —Laura Steuer
Volunteering for a clinical trial is a great experience. The staff
answered all my questions and explained everything that was going to
happen. I was never worried or concerned because their support made each
step easy. The science and technology underlying potential new
treatments for CF is just phenomenal; it’s kind of thrilling to think
that I was part of it. And, above all, it’s tremendously satisfying to
know that someday many other patients with CF (or even myself) may
benefit from these treatments. —Anonymous
"Our son has CF. We will to do everything we can to help find a cure
for this illness. By participating in these studies, we hope the doctors
get a little bit more understanding of CF and get one step closer to
the cure. Not only does it feel good to help with these studies but we
also felt assured our son was getting extra medical attention by the
constant check ups and monitoring. The staff was always perfect in
explaining what the process was and always accommodated our schedule
where they could. If we ever had any questions or concerns, we were able
to contact the staff directly - and really at anytime of the day or
night. We have been part of 3 studies now and we are always open for
more. Again, anything to help find a cure...."--Anonymous
Milla, C & Spano, J. Defining the Clinical Utility of the Lung Clearance Index, Are We There Yet? American Journal of Respiratory and Critical Care Medicine, 2021, 203, 8 937-938. doi: 10.1164/rccm.202010-3899ED on November 12, 2020. PMID: 33181036
Marmor, M., Zirbes, J., Davies, Z., & Mohabir, P. 2020. How is CF Diagnosed? (Chapter 2) in Y Sher, A Georgiopoulos, T. Stern.(Ed). Facing Cystic Fibrosis (pp 9-17). United States: The Massachusetts General Hospital Psychiatry Academy.
Malkovskiy, AV, Yacob , AA, Dunn, CE Zirbes, JM, Ryan, SP, Bollyky, PL, Rajadas,J, & Milla, CE. Salivary thiocyanate as a biomarker of cystic fibrosis transmembrane regulator function. Analytical Chemistry,2019; 91, 12, 7929-7934. https://doi.org/10.1021/acs.analchem.9b01800
Spano, JM, Ryan, SP & Milla, CE. Longitudinal assessment of Lung Clearance Index as a marker of disease progression in a cohort of children with cystic fibrosis and normal spirometry. Abstract, ePoster European Respiratory Society Virtual Congress 9/7-9/9/2020.
Zirbes, JM, Alvarez, D., Ryan SP., & Milla, CE. Lack of Correlation between patient reported outcomes (PROs) and lung clearance index (LCI) among cystic fibrosis children with normal spirometry. Abstract, ePoster and Oral Presentation at the 42nd European Cystic Fibrosis Conference, Liverpool, United Kingdom. June 2019.
Comparison of Acute Effects of Conventional High
Frequency Chest Oscillation (HFCWO) and Hand Held Percussor (Electro-flo
5000) for Airway Clearance in Cystic Fibrosis Patients.
A Comparison of the Acute Effects of Two Hand Held
Percussive Devices-The Electro-Flo 5000 and The G5 Flimm-Fighter, for
Airway Clearance in Patients
Development of an enhanced Beta-adrenergic Sweat Secretion Rate assay as a Cystic Fibrosis Biomarker
Davies, Z., Dunn, C., Fong, K., Ryan, S., Yacob, A., Zirbes, J., Milla, C.
Clinical Trials: Frequently Asked Questions
What is an informed consent?
Informed consent is a process of questions and answers that begins with our initial contact and continues through out the study. It is also a document that you sign stating that you have been informed about the trial and that you freely consent to participate. The consent contains the following information: The purpose of the trial. Which procedures will be performed. The number of visits. The risks associated with the study drug. Who to contact with questions. Your rights as a study participant.
How long will my participation last?
Your participation will vary depending on the trial you are interested in. Some studies are completed in one day, but typically they will last a couple of months.
Is there always a benefit to participating in a clinical trial?
No, often the only benefit is to future Cystic Fibrosis patients.
What happens if I think the study drug is making me sick?
Your health and well being is our primary concern. If the research team agrees that your health is being compromised we will withdraw you from the study, but we would ask that you to return for one final study visit.
In general what procedures can I anticipate while participating in a study?
Most often you will have your vital signs taken (heart rate, respiratory rate, height, weight, temperature and pulse oximetry check), a physical exam, blood work and usually spirometry.
While participating in a study what is my responsibility?
You need to show up for all study visits on time, comply with study procedures, and notify study staff of changes in your health and medications as soon as possible.
When I come to a study visit who will see me?
Usually you will be seen during your visit by the study coordinators.
Do I need to tell my primary physician that I am participating in a clinical trial?
Yes, if you like we will contact your doctor at the beginning of the study to discuss your participation.
What happens if I get sick while I'm enrolled in a clinical trial?
Contact your study coordinator immediately. The coordinators are on call 24 hours a day and we will direct your care through the research team.
If I participate in a study will I receive study drug for sure?
No, study subjects are most often randomly assigned to groups (study drug vs. inactive agent). The exact sequence for assignment to study drug is specified in the informed consent. Usually the study subject, the coordinators and the rest of the research team does not know which participants are getting study drug.
Is it OK for me to drop out of a study after I enroll?
Yes, you can always drop out of a study but the team will ask why you want to leave. Remember, you have made a commitment to participate.
Do I get paid to participate in a clinical trial?
Most often you are reimbursed for your time and inconvenience. Each study is a bit different. This information is clearly stated in the informed consent.
Who do I contact if I am interested in future clinical trial participation?
You should call one of the study coordinators:
Jackie Zirbes (650)721-1132
Lani Demchak (650)725-1087
In order to reach our goals of better treatments and ultimately a cure for Cystic Fibrosis, we need your help. You ask: "why me?" we reply: "why not you?" Together we can work to attain our goals.