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NIH grant 1U01FD009005-01: Digital Biomarkers of Motor Function as Drug Development Tools in Charcot-Marie-Tooth Disease
Institute: {'code': 'FD', 'abbreviation': 'FDA', 'name': 'Food and Drug Administration', 'admin_org_id': '12500799', 'admin_funding_url': ''}. PI: Mazen Dimachkie. Award: $1100000.
Start: 2026-09-25T00:00:00.
ABSTRACT
Charcot-Marie-Tooth (CMT) disease is a group of inherited disorders that cause nerve damage, primarily
affecting the peripheral nerves, which are responsible for movement and sensation in the arms and legs. CMT
is characterized by progressive muscle weakness and atrophy, particularly in the feet, legs, hands and arms.
Clinical measures of CMT, largely based on quantitative variations on the neurological exam, have suboptimal
sensitivity to change due to the slowly progressive nature of the disease and relevance to clinical meaning in
patients’ lives. More direct measures of physical activity, ambulation and hand function are not always assessed
in the clinical setting, and rarely quantified. Such tasks require a consistent reproducible protocol and are highly
susceptible to trial-to-trial variability, often serving as a poor reflection of natural ability and function.
BioSensics has partnered with the Hereditary Neuropathy Foundation to explore the use of its wearable-based
digital health technologies in assessing lower and upper limb function in CMT. Our study with 47 adult
participants with CMT established the feasibility and initial clinical validity of using wearable sensors for at-home
monitoring of motor function and disease severity in CMT. In addition, Elpida Therapeutics has partnered with
BioSensics to use its wearable sensors to collect exploratory outcomes related to physical activity and hand
function in its natural history study in CMT type 4J, a rare genetic subtype of CMT, and the upcoming gene
therapy clinical trials.
In this project, we will conduct an 18-month multi-site clinical validation study for the biomarkers of motor function
in CMT1A, which accounts for over 50% of all CMT cases worldwide. CMT1A represents the most prevalent and
clinically well-characterized demyelinating form of CMT and is the primary focus of nearly all current industry-
sponsored therapeutic development programs, making it an optimal initial population for digital motor biomarker
development. The primary goal of the project is to qualify digital measures of motor function for CMT1A by
working with the FDA Innovative Science and Technology Approaches for New Drugs (ISTAND) Program.
BioSensics has already entered this pathway: the Letter of Intent for COU1 below was submitted to the ISTAND
Program in May 2026 and determined reviewable by FDA (DDT-IST-000073). This project generates the clinical
validation evidence that will support its Qualification Plan. Context of Use: COU1) Wearable sensor-derived foot
drop metrics as longitudinal outcome measures in adult CMT1A patients; COU2) Wearable sensor-derived
ambulation metrics passively acquired during activities of daily living as longitudinal outcome measures in adult
CMT1A patients; COU3) Wearable sensor-derived upper limb use metrics passively acquired during activities of
daily living as longitudinal outcome measures in adult CMT1A patients. Moreover, as recommended by the FDA
ISTAND Program, we will interview participants at baseline, month 6, and month 18 using the Technology
Acceptance Model to examine perceived usefulness, perceived ease of use, and attitude toward use, and will
ask participants whether they prefer at-home sensor monitoring or in-clinic strength testing.document fetched 2026-10-04 02:43