TUCSON, Ariz.,
March 28, 2024 /PRNewswire/ --
Critical Path Institute's (C-Path) Duchenne Regulatory Science
Consortium (D-RSC) is excited to announce the launch of a
groundbreaking model-based Clinical Trial Simulator (CTS),
specifically designed to improve design of efficacy studies for
potential therapies for Duchenne muscular dystrophy (DMD). This
pioneering Drug Development Tool is set to positively impact the
medical research community by significantly optimizing clinical
trial design.
This Clinical Trial Simulator will
positively impact DMD research by significantly optimizing clinical
trial design.
DMD is a rare, fatal, X-linked, muscle wasting, and progressive
disease that predominantly affects boys but has been shown to
manifest in some female carriers. Research and development in this
area have been challenging due to the complexities associated with
the disease.
Developed by C-Path's Quantitative Medicine Program and
D-RSC, together with an extensive group of collaborators, this
simulator utilizes advanced models of the longitudinal changes in
the velocity at which individuals can complete specified, timed
functional tests. These tests, which include the supine-stand,
4-stair climb, 10-meter walk/run test, or 30-foot walk/run test,
are frequently used as clinical trial endpoints. Additionally, the
tool models longitudinal changes in forced vital capacity and the
North Star Ambulatory Assessment total score, providing a
comprehensive approach to trial design for simulation. A key
feature of the DMD Clinical Trial Simulator is its incorporation of
relevant sources of variability such as baseline severity at study
start, age, steroid use, genetic mutation and study type. This
ensures that the simulations are as realistic and informative as
possible, paving the way for more effective and efficient clinical
trial designs.
In November 2022, D-RSC received a Letter of Support from
the European Medicines Agency, and the Clinical Trial Simulator is
currently being reviewed by the U.S. Food and Drug Administration
(FDA).
"The launch of the DMD Clinical Trial Simulator represents
a significant step forward in our fight against Duchenne muscular
dystrophy," said Ramona
Belfiore-Oshan, Ph.D., Executive Director for D-RSC. "By
using quantitative methodologies that allow the improvement of
trial design in silico, we can accelerate the development of
effective therapies for DMD and provide a learning platform for
application of model informed drug development in other rare
diseases."
"The DMD Clinical Trial Simulator is poised to become a
valuable resource for researchers and pharmaceutical companies
involved in DMD research," commented C-Path CEO Klaus Romero, M.D. M.S., FCP. "Its ability to
simulate various scenarios and outcomes will not only improve the
accuracy of clinical trials but also reduce the time and resources
required to bring new therapies to market."
The CTS will be made publicly available on c-path.org,
here, and hosted on the Rare Disease Cures Accelerator-Data and
Analytics Platform (RDCA-DAP®), C-Path's designated
analytics hub for data analysis and generation of drug development
tools across multiple rare diseases.
"We advance the RDCA-DAP platform as a place for innovation
for rare disease drug development and are excited to offer open
access to C-Path's drug development tools on RDCA-DAP. Granting
access to the D-RSC Clinical Trial Simulator through RDCA-DAP
creates a unique opportunity for innovative trial designs in DMD,"
said Alexandre Bétourné, Pharm.D., Ph.D., Executive Director,
RDCA-DAP.
About D-RSC:
D-RSC (Duchenne Regulatory Science Consortium) is comprised of a
team of experts dedicated to advancing the understanding and
treatment of Duchenne muscular dystrophy through innovative
research and technology solutions.
About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit
established in 2005 as a public-private partnership, in response to
the FDA's Critical Path Initiative. C-Path's mission is to
lead collaborations that advance better treatments for people
worldwide. Globally recognized as a pioneer in accelerating drug
development, C-Path has established numerous international
consortia, programs and initiatives that currently include more
than 1,600 scientists and representatives from government and
regulatory agencies, academia, patient organizations, disease
foundations and pharmaceutical and biotech companies. With
dedicated team members located throughout the world, C-Path's
global headquarters is in Tucson,
Arizona and C-Path's Europe
subsidiary is headquartered in Amsterdam,
Netherlands. For more information, visit c-path.org.
FDA Acknowledgement
Contacts:
Roxan Triolo Olivas
C-Path
520.954.1634
rolivas@c-path.org
Kissy Black
C-Path
615.310.1894
kblack@c-path.org
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SOURCE Critical Path Institute