Lead Investigator: Ramona Belfiore-Oshan, Critical Path Institute
Title of Proposal Research: Development of an Artificial Intelligence/Machine Learning (AI/ML) model-based clinical trial simulation platform to better characterize Loss of Ambulation in Duchenne Muscular Dystrophy (DMD)
Vivli Data Request: 10852
Funding Source: Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 56% funded by the FDA/HHS, totaling $23,740,424, and 44% funded by non-government source(s), totaling $18,881,611. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. ERDERA has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement N°101156595. Views and opinions expressed are those of the author(s) only and do not necessarily reflect those of the European Union or any other granting authority, who cannot be held responsible for them. D-RSC is partially funded by membership fees paid by the member companies, who pay an annual membership fee to be a part of the consortium.
Potential Conflicts of Interest: Dr. Belfiore-Oshan reports D-RSC is a non-profit consortium developing drug development tools that are suitable for regulatory uses. The consortium is supported by financial contributions of 12 drug companies, Parent Project Muscular Dystrophy and CureDuchenne, which contribute to the intellectual development of consortium projects. Companies are not permitted under the user agreement to discuss individual drug programs, and all results are made publicly available after submission to the regulatory authorities (FDA and EMA).
Summary of the Proposed Research:
This project aims to develop a new technology to build tools that improve drug development for Duchenne Muscular Dystrophy (DMD). DMD is a rare genetic condition that affects about 1 in every 3,500 male births worldwide. It causes muscle weakness and progressive loss of muscle function, leading to severe disability and significantly shortened life expectancy.
Currently, developing treatments for DMD is challenging, time-consuming, and expensive. This research seeks to address these challenges by creating tools that better predict how DMD progresses over time. These tools will also help researchers understand how safe and effective potential treatments might be, speeding up the development of new therapies and reducing costs.
The research involves designing a computer-based platform using Artificial Intelligence (AI) and Machine Learning (ML). These advanced technologies will analyze existing data to simulate how DMD progresses and how patients might respond to treatments.
The outcomes of this research could transform how treatments for DMD are developed, ultimately improving the lives of individuals living with this condition
Requested Studies:
A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.
Data Contributor: Cure Duchenne
Study ID: NCT01753804
Sponsor ID: PRO-DMD-01