Study examines if gene editing can help
people living with transthyretin amyloidosis with
cardiomyopathy (ATTR-CM)
WASHINGTON, May 2, 2024
/PRNewswire/ -- The first U.S. heart patient involved
in a study for a new gene-editing therapy directly infused inside
the body has successfully been enrolled at MedStar Washington
Hospital Center. The pivotal, international clinical trial is
evaluating the safety and efficacy of a gene editing treatment for
patients with transthyretin amyloidosis with cardiomyopathy
(ATTR-CM), an under-recognized cause of heart failure.
The clinical trial, known as MAGNITUDE, is a Phase 3,
multinational, multicenter, double-blind, placebo-controlled trial
that is evaluating the efficacy and safety of a single infusion of
a gene-editing therapy in patients with ATTR-CM. Transthyretin
amyloidosis is a protein disorder that typically affects the heart
by stiffening the muscle and making it harder for the heart to pump
blood throughout the body. The treatment is designed to prevent the
production of an abnormal form of a protein called transthyretin
(TTR) that causes the disease.
In ATTR, the TTR protein accumulates and builds up in different
parts of the body, particularly in the heart and nerves. The
buildup is known as amyloidosis. When TTR amyloidosis is in the
heart, it is known as ATTR-CM, a life-threatening condition.
Participants in the study will be assigned at random to receive
either the research medication called NTLA-2001 or a placebo, which
has no active ingredients. NTLA-2001 contains a gene-editing system
known as CRISPR/Cas9. It is designed to find and disable the TTR
gene in the liver where most TTR protein is produced. NTLA-2001 is
given as a one-time intravenous infusion.
"It is with great excitement that our center has enrolled the
first U.S. patient in the MAGNITUDE trial, a pivotal study
assessing a new avenue of treatment for human diseases,
specifically ATTR-CM," said Farooq
Sheikh, MD, medical director of the Advanced Heart Failure
Program at MedStar Washington Hospital Center and principal
investigator of the MAGNITUDE study. "The MedStar Health
Infiltrative Cardiomyopathy/Advanced Heart Failure Program is
passionate about improving the lives of our patients in the region
and beyond."
NTLA-2001 is the first investigational CRISPR therapy to be
administered intravenously to edit genes inside the human body.
Interim Phase 1 and 2 clinical trial data showed the administration
of NTLA-2001 led to consistent and long-lasting transthyretin (TTR)
protein reduction.
There are an estimated 50,000 people worldwide living with
hereditary ATTR amyloidosis and between 200,000 and 500,000 people
with wild-type ATTR amyloidosis, which typically impacts the heart,
leading to ATTR-CM. There is no known cure.
The MAGNITUDE study is expected to enroll approximately 765
participants worldwide and represents one of the more 175
cardiovascular research studies conducted at MedStar Health
annually. For more information about the study, visit
ClinicalTrials.gov or contact study coordinator Hellina Birru, MD, at
hellina.t.birru@medstar.net.
Learn more about our full research portfolio at
MedStarResearch.org.
Media Contact:
So Young
Pak
202-877-2748
soyoung.pak@medstar.net
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SOURCE MedStar Health