UNISUS PAH Clinical Trial - phaware® interview 461
Mar 26, 2024
SPECIAL EDITION
Jonathan Hudson is a Global Trial Leader in Pulmonary Hypertension
at Janssen Research & Development and is accompanied by
colleague Josephine Garcia-Ferrer, Ph.D and Medical Manager.
Jonathan and Josephine discuss Pulmonary Arterial Hypertension
(PAH), the importance of clinical trials, what they entail,
and share information on Janssen’s
UNISUS Study for adults with PAH.
Learn more about UNISUS Study with the
Janssen Global Trail Finder.
Steve Van
Wormer:
Hello, and
welcome to, I’m Aware That I’m Rare, the phaware® podcast. I’m your
host, Steve Van Wormer, from Phaware Global Association. Today, I
am joined by two Study Team Members from Janssen Research &
Development, a pharmaceutical company dedicated to Pulmonary
Hypertension research. Jonathan Hudson is a Global Trial Leader and
Josephine Garcia-Ferrer is a Medical Manager. They are overseeing
the
UNISUS clinical trial — evaluating an investigational medicine
in patients with Pulmonary Arterial Hypertension, or PAH. Jonathan
and Josephine... welcome...
Jonathan
Hudson:
Thank you for
the introduction, Steve. Great to be here today speaking with an
organization dedicated to patients in the PH community.
Josephine
Garcia-Ferrer:
I feel the same, Jonathan.
Steve:
First off,
Jonathan, could you tell our listeners a bit more about Janssen
Research & Development and Janssen’s commitment to the PH
space?
Jonathan:
Sure, for
nearly a century, we have fearlessly confronted the world’s most
devastating and complex diseases. We are proud to be a part of
Johnson & Johnson, one of the largest and most broadly-based
healthcare companies. Backed by the size and reach of Johnson &
Johnson, we are transforming lives and reinventing healthcare. As
part of Johnson & Johnson, we tackle society’s most pressing health
challenges, connecting big ideas to the resources it takes to make
them a reality and advancing access to good health for all. We seek
medical breakthroughs wherever they occur, leveraging internal
expertise and embracing external science to bring solutions to
people who need them. We envision a world where people in every
community, of all walks of life, have access to necessary medicines
and health solutions. We are a leader across our medical areas of
focus with a pioneering history of firsts. Expanding and enhancing
the quality of people’s lives remains our greatest reward. We are
committed to transforming pulmonary arterial hypertension into a
long-term, manageable condition so that patients can live a full
life. We have made PH a ‘therapeutic area of focus’ and are here to
help advance research for those affected by PH.
Steve:
Well, thanks
for that background. Now, before we discuss clinical research and
the UNISUS study, would you be able to tell us a bit more about
Pulmonary Arterial Hypertension?
Jonathan:
Certainly.
There are five groups of PH, as defined by the World Health
Organization (WHO). All are severe diseases characterized by high
blood pressure in the lungs. Our focus has been on a specific, rare
form of PH (WHO Group 1) called pulmonary arterial hypertension
(PAH), but our research covers all groups of PH. PAH (WHO Group 1)
is a rare, progressive, and relentless disease, where the pressure
in the blood vessels of the lungs is elevated, resulting in stress
on the heart. Despite recent advances, PAH remains a severely
debilitating condition that leads to heart disease and early death.
Early diagnosis and treatment are critical to helping improve life
expectancy, but PAH is difficult to diagnose and there is currently
no cure. PAH causes significant physical, social, and emotional
burdens amongst affected patients and their caregivers. Over the
last 20 years, the number of medications for the management of PAH
has increased, which has helped to improve the lives of patients.
However, even with these new advancements, we still have a lot of
work to do to improve the lives of our patients. We’re working to
transform PAH into a long- term, manageable condition to help
improve the lives of patients. Janssen’s aim is to help increase
the mainstream awareness around PAH so that no patient goes
undiagnosed and there’s no delay in diagnosis.
Steve:
Thanks,
Jonathan. We are very familiar with PAH here at phaware®. I’d love
to hear more about clinical trials in general, and their role in
bringing a therapy to market. Could you elaborate on that a little
bit?
Jonathan:
Sure, I’d be
happy to. The complex, global healthcare landscape continues to
present new challenges, new diseases, and new dynamics. Healthcare
systems are shifting toward delivering improved outcomes, enhancing
the patient experience, and providing value-based care. Clinical
research is medical research involving human participation. So,
clinical trials are the primary way that researchers find out if a
new medication, which we refer to as investigational medicine, is
safe and effective. Clinical trials are conducted by doctors and
researchers. Using what they learn from studies, regulatory
agencies can decide if an investigational medication for a specific
condition could one day be approved and made available to the
public. Before the regulatory agencies, like the U.S. Food and Drug
Administration, or FDA, can approve a clinical trial to begin,
potential treatments must be studied in laboratory animals
first.
Steve:
I
see. And for those who might be unfamiliar, could you breakdown the
different phases of clinical research, and let us know what phase
the UNISUS study is in?
Jonathan:
Yes. We have a
deep responsibility to the people we serve; we work tirelessly to
advance and improve how medicines are discovered, developed, and
made. Phase I is the first study in humans. These are small studies
with 20–100 participants, mostly healthy volunteers. The main
purpose for phase I studies is to evaluate the safety of the study
medication at various doses. During phase I, researchers evaluate
how the study medication is absorbed by the body and removed from
the body, while also determining any potential side effects of the
study medication. If a study meets its endpoints in phase 1, it
will then move to phase II with the doses selected. Phase II
studies are small studies with around 100-500 participants, who are
patients with a disease being investigated. Phase II studies
typically assess safety and efficacy, which is whether the study
medication works for a particular disease. Then we get to phase III
studies, which is what the UNISUS Study is. Phase III studies are
large studies with around 500 or more participants. These are the
main studies for final approval by health authorities. The main
objectives for phase III studies to investigate are as follows: to
further evaluate safety and efficacy (whether the study medication
works for a particular disease), and how the study medication
compares to already existing standard therapies. The next phase is
phase IV, which take place AFTER a study medication has been
approved by regulatory authorities for prescription use or public
use. The goal of phase IV studies is to evaluate safety and side
effects during real world use and assess risks and benefits over a
longer period of time.
Steve:
Could you go over the benefits and risks associated with trial
participation?
Jonathan:
While it is
possible that a patient’s PAH could improve as a participant in the
study, that cannot be guaranteed. However, participation in
research may help people with PAH in the future. Your consent to
participate in the UNISUS Study is optional, and you can withdraw
your consent at any time. It is possible that patients could
experience one or more side effects during this study. Before a
patient begins, however, the study staff will review any potential
risks or side effects and ensure that the patient is a good fit for
the clinical trial. In clinical trials, patients are closely
monitored throughout. Study researchers design protocols that
explain the study in detail, and the protocol must be followed
exactly as written. An independent ethics committee or
institutional review board carefully reviews study protocols before
approving studies at a clinical site.
Steve:
I see. Could you share a bit more about why the study is being
conducted?
Jonathan:
The UNISUS
study is a research study for adults with Pulmonary Arterial
Hypertension. While there has been progress in the development of
medications for PAH, there is still a need for medications that can
help treat and slow the progression of the disease. In this study,
doctors want to learn more about a higher dose of an
investigational medication for adults with PAH when compared to a
lower dose. If a patient is determined to be eligible to
participate, they will be randomly assigned to receive either the
higher dose investigational medication, or lower dose. The patient,
the study doctor, and the study staff will not know what medication
the patient is assigned. However, in the event of an emergency,
that information can be provided to your study doctor so he or she
may take the right actions for the participant. Additionally, an
independent committee, including PAH experts, is aware of what
medication the patients receive during the study and monitor their
safety. Patients will take their assigned medication once a day
while in the study, which could last between 3–6 years. During the
study, patients are asked to attend regular study visits to
evaluate their health, and also to participate in phone calls from
the study doctor.
Steve:
What else can a study participant expect during clinical trials for
PAH?
Jonathan:
Clinical
trials aim to show how a study medication affects the participant’s
health condition over a given timeframe. To assess how the study
medication is working, the clinical trial team evaluates the
participant through various assessments. For PAH, teams can monitor
the change in PAH symptoms, like shortness of breath, swollen
extremities, chest pain, tiredness, and dizziness, along with PAH
tests like heart rate, blood pressure, WHO Functional Class, and
6-minute walk test values. These are also known as risk
assessments. These evaluations are used alongside the patient
reported data, like how you feel and how your symptoms are, to
provide healthcare professionals with a better understanding of
your PAH. We also conduct standard safety lab assessments to ensure
the well-being of our patients. I’m sure the PAH community is
familiar with these tests, as they’re fairly standard diagnostic
tests for PAH, but I would like to take a moment to have Josephine
elaborate on them.
Steve:
Sure, Jonathan, I’m sure we’d all love a refresher. Thank you for
explaining, Josephine.
Josephine:
Not a problem
at all, Steve. The World Health Organization, abbreviated as WHO,
standardized a test to measure how severe a patient’s pulmonary
hypertension symptoms are. This is called the WHO Functional Class.
They created 4 classes, class I, class II, class III, and class IV.
Class I is defined as symptom free when physically active or
resting, more generally speaking, the least progressed version of
pulmonary hypertension. Class II individuals have no symptoms at
rest, but normal activities such as grocery shopping or going up
stairs can cause some discomfort and shortness of breath. Class III
may also have no symptoms at rest, but are more limited when
performing and completing normal chores around the house. And
finally, class IV will have symptoms at rest and severe symptoms
with activity. Normally, a patient’s doctor can assign them to one
of these classes and monitor their PAH through the different
definitions.
Steve:
Can you also
explain a bit about the methodology behind the 6-minute walk tests?
Why are they such an important piece of a patient’s treatment
regimen?
Josephine:
Yes, of
course. The 6-minute walk test was developed by the American
Thoracic Society. To keep it simple, the test is designed exactly
as it sounds. It works to measure the distance an individual can
walk in 6 minutes. A site staff member will explain how this is set
up and performed during an individual’s site visit. As an
individual’s PH progresses, the distance they can walk for 6
minutes will decrease. This is one of the tests we use to measure
disease progression, we also use other assessments like Jonathan
had mentioned. A patient’s heart rate, blood pressure, WHO
Functional Class, among other things, are considered when
evaluating disease progression.
Steve:
Very straight forward. Thanks!
Josephine:
I
thought so too. There are also other ways we can measure PAH
progression, through things that are naturally occurring in
everyone’s body, like peptides, prostaglandins, and molecules. PAH
can cause imbalances in these compounds, which can lead to strain
on the heart and lungs in PAH patients. But, that’s enough about
the science behind PAH. I’ll now pass it back to Jonathan to review
a bit more about the UNISUS Study.
Steve:
Thank you, Josephine. Jonathan, can you tell us where this study is
being conducted?
Jonathan:
The study is
being conducted worldwide in about 40 different countries, at more
than 200 study sites. There are currently 42 sites in the US and 4
sites in Canada. The study is looking to enroll around 900 patients
with PAH and are WHO FC II to IV. We are driven by our belief that
“patients are waiting” and there is no time to waste. We do our
best to select sites that best serve the patient population. We
take pride in selecting our study sites for our
patients.
Steve:
Just to be clear... What are study “sites?”
Jonathan:
Study sites
are institutions where clinical research can take place. We
personally vet each possible study site to ensure that they’re a
good fit for our clinical trial. We advocate for sustainable
healthcare systems that value innovation and are accessible to
people around the world. A lot of research goes into selecting and
opening study sites, so we know that they’ll be a good fit for our
patients and study goals. We support our participating sites by
ensuring healthcare professionals and patients have access to the
information, programs, and support they need to achieve the best
results possible.
Steve:
Can you tell us a little bit more about the investigational
medication?
Jonathan:
We
work to ensure broad and timely access to medicines through the
unique collaborations with governments, healthcare agencies, and
others. The lower dose of the investigational medication has been
approved for other indications by the U.S. Food and Drug
Administration and the European Medicines Agency (EMA), as well as
by health authorities in other countries. As part of the UNISUS
study, a higher dose of the study medication is now being evaluated
by doctors in people with PAH to study safety and
efficacy.
Steve:
And who is eligible to be in the UNISUS study?
Jonathan:
To
qualify for the UNISUS study, participants must be 18 years of age
or older and must have been diagnosed with PAH. More specifically,
participants must have a World Health Organization functional class
of II, III or IV. Additional criteria apply, but those are the main
requirements. The study site would conduct additional tests and
assessments to determine if a patient is eligible. Once a potential
patient reaches out to a study site, the site will usually ask for
medical records if they’re not already established in their care,
and work to set up a more detailed consenting and screening visit.
Each site will take the time to work with an individual to ensure
the clinical trial is a good fit for them. We work with patients
through the entire process to ensure the best possible
experience.
Steve:
Are there costs for patients when joining a clinical
trial?
Jonathan:
All
study-required visits, tests, and medications will be provided at
no cost. Reimbursement for study- required travel is also
available.
Steve:
What else can you tell us about the study design of
UNISUS?
Jonathan:
The study was
designed with 2 parts. The first part is the double-blind period
which we referenced earlier where neither the patent nor doctor
know which dose of the study medication they are receiving. If the
double-blind part of the study meets its endpoints, then patients
that complete the 1st part will be enrolled into the 2nd part,
which is an open-labelled part of the study that allows for
patients to take the higher dose of the study medication for up to
2 years. If study endpoints are met, and if the study medication is
approved for use, participants would be allowed to take the study
medication following their participation in the study.
Steve:
So, how can patients learn more about the study?
Jonathan:
If
interested in learning more about the UNISUS study or perhaps
participating, patents can search for the UNISUS study on
clinicaltrials.gov or visit Janssen’s own Global Trial Finder on
the Janssen website. We suggest using “PAH” as a keyword when
searching, and then select the correct study for your age group.
Please talk to your treating physician before considering if
participation is right for you.
Steve:
Great, a big
thank you to you and Josephine for taking the time to review the
UNISUS Study. It’s nice to provide our listeners with some more
information on PAH in clinical trials. It was great to hear from
you today. Thanks a lot, Jonathan and Josephine!
Jonathan:
Thank you for
having us, Steve. It was great to share information on the UNISUS
Study and help educate on clinical trials for PAH too.
Josephine:
Thank you for having us, Steve.
Steve:
And thank you, listener, for joining
us here today on I'm Aware That I'm Rare,
the phaware® podcast. You can also learn more about pulmonary
hypertension clinical trials on our website
at www.phaware.global/clinicaltrials.
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