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Abstract Details

Feasibility of at-home motor assessments using wireless technology in patients with myelopathy: year 1 follow up of CYGNET, an observational study of patients with adrenomyeloneuropathy
Aging, Dementia, Cognitive, and Behavioral Neurology
P6 - Poster Session 6 (5:30 PM-6:30 PM)
7-006

AMN is a rare neurodegenerative disorder of the brain and spinal cord affecting adults with mutations in the ABCD1 gene. Quantification of AMN disease progression is challenging, owing to phenotypic and disease progression variability. One solution to address this is to measure traditional functional motor tasks more frequently using wireless motion sensors during clinic and remote visits. Here, we assess the feasibility and test-retest reliability of motion sensors over 1 year, and relate it to standard in-clinic measurements.

To quantify change in symptoms over time using home-based gait and mobility assessments from a prospective natural history study of patients with adrenomyeloneuropathy (AMN).

CYGNET (NCT05008874) is an ongoing prospective, multicenter, observational study that will enroll up to 80 male patients with AMN and follow them for ≥2 years with annual clinic visits and quarterly remote visits. Gait and balance assessments include postural body sway parameters and traditional functional motor tasks. In clinic, standard methods are used to calculate time and distance. Wireless motion sensors enable quantification of the temporal–spatial components of gait assessments both in the clinic and at home.

Over 50 patients between 18–69 years of age with Expanded Disability Status Score 1-6.5 have been enrolled. Correlation between wireless sensor and operator-assessed mobility measures collected at baseline clinic visit ranged from 0.746 (Timed Up-and-Go) to 0.998 (2-Minute Walk Test). In-clinic and home data collected over 1 year utilizing wireless technology will be presented for the first 20 patients enrolled.

Use of wireless motion sensors can provide an objective and sensitive assessment of gait, stability, balance and functional abilities of an individual both in their natural and the clinical environment. These data will help inform the design of future clinical trials evaluating potential therapeutics, including gene therapy for AMN.

Funding: This study was sponsored by SwanBio Therapeutics.

Authors/Disclosures
Christopher D. Stephen, MB ChB FRCP SM (Massachusetts General Hospital)
PRESENTER
Dr. Stephen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SwanBio Therapeutics. The institution of Dr. Stephen has received research support from Sanofi. Dr. Stephen has received research support from National Institutes of Health.
Caroline Bergner, MD (University hospital Leipzig) The institution of Dr. Bergner has received research support from Deutsche Forschungsgemeinschaft .
Wolfgang Koehler (University of Leipzig Medical Center) Wolfgang Koehler has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Minoryx. Wolfgang Koehler has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vigil. Wolfgang Koehler has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for United Leukodystrophy Foundation. Wolfgang Koehler has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for European Leukodystrophy Association. The institution of Wolfgang Koehler has received research support from Minoryx. The institution of Wolfgang Koehler has received research support from Vigil. The institution of Wolfgang Koehler has received research support from SwanBio. The institution of Wolfgang Koehler has received research support from Alexion.
Joshua Bonkowsky, MD, PhD Dr. Bonkowsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Denali. Dr. Bonkowsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurogene. Dr. Bonkowsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Passage Bio. Dr. Bonkowsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Autobahn. Dr. Bonkowsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Bonkowsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Bonkowsky has stock in Orchard. The institution of Dr. Bonkowsky has received research support from NIH. The institution of Dr. Bonkowsky has received research support from ELA. An immediate family member of Dr. Bonkowsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Bonkowsky has received publishing royalties from a publication relating to health care. Dr. Bonkowsky has received publishing royalties from a publication relating to health care.
Asif M. Paker, MD, MPH Dr. Paker has received personal compensation for serving as an employee of SwanBio Therapeutics.
Florian Eichler, MD (Massachusetts General Hospital) An immediate family member of Dr. Eichler has received personal compensation for serving as an employee of UpToDate. Dr. Eichler has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for SwanBio Therapeutics. Dr. Eichler has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Eichler has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Origin Biosciences. Dr. Eichler has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Orchard Therapeutics. Dr. Eichler has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Autobahn Therapeutics. Dr. Eichler has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Bluebird Bio. Dr. Eichler has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for UpToDate. Dr. Eichler has received stock or an ownership interest from SwanBio Therapeutics. The institution of Dr. Eichler has received research support from Bluebird Bio. The institution of Dr. Eichler has received research support from Minoryx Therpeutics. The institution of Dr. Eichler has received research support from Sio Therapeutics. Dr. Eichler has received intellectual property interests from a discovery or technology relating to health care.
Lauren Glenn (SwanBio) Mrs. Glenn has nothing to disclose.
Steven Zelenkofske, MD, DO (SwanBio Therapeutics Ltd.) Dr. Zelenkofske has received personal compensation for serving as an employee of SwanBio Therapeutics. Dr. Zelenkofske has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for DinaQor .
Marc Engelen, MD, PhD (Academic Medical Center) Dr. Engelen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Autobahn. Dr. Engelen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BlueBirdBio. Dr. Engelen has received personal compensation in the range of $0-$499 for serving as a Consultant for SwanBio. The institution of Dr. Engelen has received research support from SwanBio. The institution of Dr. Engelen has received research support from BlueBirdBio. The institution of Dr. Engelen has received research support from Autobahn. The institution of Dr. Engelen has received research support from Minoryx. The institution of Dr. Engelen has received research support from Netherlands Organization for Scientific Research.