Establishing walking-related digital biomarkers in rare childhood onset progressive neuromuscular disorders

Active Project Grant Awarded Last action

Award ID
R01HD112908
Recipient
Awarding agency
Obligated
$2,141,219

Award facts

Completion date 63% complete
Place of performance
New York, NY 10032, USA

Award details come from USAspending.gov.

Description

As the agency reported it.

Abstract
Individuals with childhood onset, genetically determined neuromuscular disorders (NMD) experience progressive neuromuscular degeneration resulting in weakness that affects ambulatory function. Genetic testing and consensus-derived diagnosis guidelines have led to an increasing prevalence of neuromuscular diseases approaching other more common neurological conditions. Recent therapeutic approaches in duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) have resulted in disease modifying therapies commercially available or in clinical trials with the best outcomes with early treatment. While results from novel drug therapies are promising, symptoms of weakness and impaired function persists. Patients report changes in motor function in real-world settings, but these changes often elude standard in-clinic examinations. There is a compelling need to develop more sensitive, quantitative assessments that focus on ambulatory function in real- life settings. Digital mobility outcomes (dmos) use wearable technology for continuous remote monitoring in a person's own environment. Consumer-grade activity trackers monitor healthy and clinical cohorts, but with limited accuracy. Research grade trackers and foot-worn gait monitoring devices measure mobility-related volume (step count, distance, duration of walking bouts, etc.), but can only capture a minimal set of stride-by-stride real-life gait parameters (e.g., stride time, length, velocity) and they cannot measure kinetic parameters. We argue that the modest accuracy and granularity of current wearable devices hamper the objective characterization of subtle but clinically meaningful changes. Our recent findings suggest that novel machine-learning (ML)-based abstraction models may map noisy signals from foot-worn sensors (namely, instrumented insoles developed by the project team) into accurate and clinically relevant spatiotemporal and kinetic gait parameters. These gait parameters derived from instrumented insoles, which we refer to as digital mobility outcomes (dmos), may serve as functional biomarkers to detect changes in real world function. The purpose of this research is twofold: (1) to identify disease-specific walking-related digital biomarkers of disease severity, and (2) to determine the insole-derived dmos that are most sensitive to longitudinal changes in ambulatory function and best able to predict 12-month changes in ambulatory function in DMD and SMA.

Transactions

Action Obligated Date
Amendment $501,402
Amendment $496,385
Amendment $571,716
Original award $571,716

Subawards

Subrecipient Amount Awarded
The Trustees Of The Stevens Institute Of Technology Dr. Damiano Zanotto will be responsible for the engineering-related aspects of the research activities. Zanotto will be assisted by a postdoctoral associate and… $205,177
The Leland Stanford Junior University Dr. John Day will be responsible for participant recruitment, enrollment, and data collection of 30 participants at Stanford University. He/she will confirm… $25,535
ST Jude Children's Research Hospital Inc. The subawardees will be Joshua Burns, PhD and Richard Finkel, MD at St. Jude Children's Research Hospital on the National Institutes of Health grant titled… $34,661
The Children's Hospital Corporation Drs. Amy Pasternak and Maria Fragala Pinkham will be responsible for be responsible for participant recruitment, enrollment, data collection and data entry of… $33,358
The Leland Stanford Junior University Dr. John Day will be responsible for participant recruitment, enrollment, and data collection of 30 participants at Stanford University. He/she will confirm… $30,450
The Leland Stanford Junior University Board of Trustees of the Leland Stanford Junior University will support “Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive… $28,373
The Trustees Of The Stevens Institute Of Technology Stevens Institute of Technology will support “Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders.”… $222,677
The Trustees Of The Stevens Institute Of Technology Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders Statement of Work Stevens Institute of Technology… $221,444
The Leland Stanford Junior University Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders Statement of Work Clinical Site Dr. John Day will be… $28,872
The Children's Hospital Corporation Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders Statement of Work Clinical Site Drs. Amy Pasternak… $28,585

Quick answers

Who received award R01HD112908?

R01HD112908 was awarded to The Trustees Of Columbia University In The City Of New York by National Institute of Child Health and Human Development (National Institutes of Health) on May 1, 2024.

How much is award R01HD112908 worth?

$2,141,219 has been obligated on R01HD112908.

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