Project Summary
To improve the efficiency, convenience and quality of ALS clinical trials, we need better biomarkers for
assessing amyotrophic lateral sclerosis (ALS) progression and response to therapy. All recent ALS trials
require patients to visit an ALS clinical trials center every 2-3 months at which time assessments are obtained,
including limb strength, vital capacity, and the ALS functional rating scale-revised (ALSFRS-R) score.
However, such infrequent assessments mean that the estimation of the rate of decline for a given patient--the
major outcome of interest in clinical trials--has great uncertainty. Moreover, visits to such centers are a major
burden for the patients and caregivers; many patients must withdraw as they become increasingly debilitated
during the study. It also means that many potentially eligible people cannot participate because they lack
convenient transportation or simply live too far away from a participating tertiary care center. One approach for
both improving our assessments and increasing convenience and encouraging broader enrollment is to
perform biomarker assessment on a more frequent basis at home. Given the advent of the Internet, Skype®,
and wireless technologies, there has been interest in collecting data at home by caregivers or patients. While
safety assessments, medication reconciliation, and blood draws will still be needed, these can be performed by
local trained health professionals (e.g. nurses and medical assistants) and consent can be obtained
electronically, making it possible for the next generation of clinical trials in ALS and other neuromuscular
disorders to take place, to a great extent, remotely. Moreover, such procedures, once in place, could extend
beyond clinical trials, to assist in the routine management of patients, to monitor therapy effectiveness,
including the need for dose adjustment or therapy discontinuation. Myolex, Inc, has as its major focus, the
development and refinement of electrical impedance myography (EIM) technology for clinical use. Building on
a successful Phase 2 SBIR study that demonstrated the power of EIM to assess ALS progression, here we
propose to design and develop a device for at-home medical monitoring. Our focus is on ALS, but this device
could be readily used in other disorders, including muscular dystrophy, myositis, and deconditioning. Our
specific aims of this proposed Phase 2B study include: 1. To develop a user-friendly at-home device (the
mSelfTM) for highly reliable, clinical-trial quality muscle data collection; 2. To develop dedicated software and
backend information technology (IT) infrastructure to ensure ease of use, ongoing feedback to the user, and
secure data transfer and storage. 3. To complete a clinical study to assess disease progression with the
mSelfTM and its ability to track disease versus standard measures. With the completion of this work, we will be
well positioned to seek FDA-approval and will seek to establish this system as a biomarker in ALS and other
neuromuscular conditions.
