Development of real-world motor outcome measures in ataxia-telangiectasia

Active Project Grant Awarded Last action

Award ID
R01NS134597
Recipient
Awarding agency
Obligated
$3,176,292

Award facts

Completion date 58% complete
Place of performance
Boston, MA 02114, USA

Award details come from USAspending.gov.

Description

As the agency reported it.

Abstract
Objective, sensitive, and clinically meaningful assessments of disease severity are needed in ataxia- telangiectasia (a-t) and other neurological disorders to support drug development efforts, facilitate clinical care, and provide caregivers and patients with real-time information about disease status. This need has become even more critical as the number of disease-modifying therapies under evaluation rapidly expands. Daily fluctuation in symptoms, superimposed changes related to child development, and variable participation in task-based assessments pose challenges to precisely measuring disease severity. Passive and continuous (i.e., task-free) collection of movement data at-home using consumer-grade wearable sensors, combined with motor control theory-inspired signal processing and machine learning methods, provides an opportunity to produce reliable, interpretable, and meaningful motor measures that can sensitively capture disease change. We have recently made substantial preliminary progress in creating algorithms to extract and characterize movement building blocks called "submovements" from a single wrist or ankle sensor worn continuously at home. These sensor-based features show strong potential as novel disease measures: they demonstrate high reliability, correlate with clinician assessments of motor severity and caregiver-reported motor function, and show potential to sensitively quantify disease progression. By passively measuring everyday activity, the information obtained can be more ecologically valid and comprehensive than task- specific measurements and is applicable in young children as well in older, non-ambulatory individuals. We propose to further develop and expand this technology over a wide age range and in a longitudinal cohort of a- t and age- and gender-matched control participants to establish reliability, age relationships, and sensitivity to disease change. Additional clinical, caregiver-reported, and molecular data will be obtained alongside wrist and ankle sensor data to increase the interpretability and clinical meaningfulness of the measures and move toward clinical trial-ready outcome measures. The overall goal of this project is to develop accessible technologies that produce highly sensitive, meaningful, and ecologically-valid measures of motor function for use in clinical trials and clinical practice in a- t as well as other pediatric and adult neurodegenerative disorders.

Transactions

Action Obligated Date
Amendment $624,776
Amendment $584,256
Amendment $584,256
Amendment $691,502
Original award $691,502

Subawards

Subrecipient Amount Awarded
University Of Massachusetts The research team led by Dr. Lee will collaborate with Dr. Gupta’s research group to develop meaningful, interpretable, and reliable wrist sensor-based models… $43,665
The Children's Hospital Corporation Dr. Yu will serve as a Co-PI in this grant. He and his team will incorporate the wearable sensor technologies developed in this project into n-of-1 clinical… $56,832
President And Fellows Of Harvard College 1. Maintain and continually improve the usability and efficiency of software for collecting and analyzing data on motor performance in the dominant arm. 2.… $50,770
The Broad Institute, Inc. Dr. Philippakis will contribute 5% of his time collaborating with the team and providing expertise on machine learning modeling, co-mentoring the postdoctoral… $50,356
Ataxia Telangiectasia Children's Project, Inc. The Ataxia Telangiectasia Children's Project (ATCP) will arrange for blood draws and biomarker assays for A-T patients and controls in order to measure… $124,233

Quick answers

Who received award R01NS134597?

R01NS134597 was awarded to The General Hospital Corporation by National Institute of Neurological Disorders and Stroke (National Institutes of Health) on December 15, 2023.

How much is award R01NS134597 worth?

$3,176,292 has been obligated on R01NS134597.

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