Abstract Details

Title Remotely Monitored Ambulatory Activity Correlates with Disability in Progressive MS: A Baseline Analysis from the SPI2 Phase 3 Clinical Trial of MD1003 (High Dose Pharmaceutical Grade Biotin)

Topic Multiple Sclerosis

Presentation(s) S54 - Multiple Sclerosis: Disease Progression, Disease-modifying Therapy, and Clinical Considerations (4:06 PM-4:18 PM)

Poster/Presentation
Number 004

Objective Assess the relationship between ambulatory activity and clinical disability/MRI measures in progressive MS patients in the SPI2 study.

Background Remote ambulatory activity monitoring has good face validity and ample validation against research grade accelerometers and is proposed as a new outcome measure for clinical trials.^1,2 This measure is an exploratory endpoint of SPI2, a phase 3 trial investigating the efficacy and safety of MD1003 (high dose Pharmaceutical grade Biotin) in primary and secondary progressive MS (PPMS, SPMS).

Design/Methods Remote monitoring of ambulation in patients receiving either MD1003 100 mg tid or placebo was collected using a Fitbit Flex®. Average daily step count from the first 30 days (STEPS) was used for the baseline activity measure. LASSO subset selection and Least Squares regression models were used to examine the relationship between STEPS and demographic variables (sex, age, disease duration, onset age, disease course), clinical disability measures (EDSS, T25FW: timed 25-foot walk, SDMT: Symbol Digit Modalities Test), a quality of life composite measure (MSQoL-29 [PHCS/MHCS: Physical/Mental Health Composite Score]), and MRI metrics (TCA: upper cervical cord area, GMV: grey matter volume, NBV: normalized brain volume, THV: thalamic volume, T1L/T2L: T1/T2 lesion volumes).

Results As of April 23rd, 2019, there were 492 enrolled patients with full STEPS data drawn from 90 global centers; 262 were women, 311 had SPMS. Median EDSS was 6.0 (IQR 4.5-6.0). Mean STEPS over the first month was 3,699 (SD: 2,962). STEPS correlated with EDSS (p<0.0001), T25FW (p<0.0001), PHCS-29 (p<0.0001), SDMT (p=0.001), and MHCS-29 (p=0.006). STEPS correlated with TCA (p=0.0014), NBV (p=0.0003), GMV (p=0.004), T1L (p=0.04); THV and T2L were not significant.

Conclusions STEPS correlated with clinical measures, cognitive function, volumetric MRI measures, and quality of life at baseline in SPI2. Remote monitoring of ambulation (Fitbit) may be a surrogate for MS disability in clinical trials.

Authors/Disclosures
Valerie A. Block, DPTSc (University of California, San Francisco) PRESENTER	The institution of Dr. Block has received research support from National MS Society.
Amber M. Alexander	Amber M. Alexander has nothing to disclose.
Nico Papinutto	No disclosure on file
	No disclosure on file
	No disclosure on file
Jeffrey M. Gelfand, MD, MAS, FAAN (University of California, San Francisco)	Dr. Gelfand has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Arialys. Dr. Gelfand has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ventyx Bio. An immediate family member of Dr. Gelfand has received personal compensation in the range of $50,000-$99,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Headache: The Journal of Head and Face Pain. The institution of Dr. Gelfand has received research support from Genentech/Roche. The institution of Dr. Gelfand has received research support from Vigil Neurosciences. An immediate family member of Dr. Gelfand has received publishing royalties from a publication relating to health care. Dr. Gelfand has received publishing royalties from a publication relating to health care. Dr. Gelfand has received publishing royalties from a publication relating to health care. Dr. Gelfand has a non-compensated relationship as a Trial Steering Committee Chairperson and member with Roche / Genentech that is relevant to AAN interests or activities.
Roland G. Henry, PhD (University of California, San Francisco)	Dr. Henry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MEDDAY. Dr. Henry has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Henry has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Henry has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sanofi/Genzyme.
Frederic Sedel	No disclosure on file
Bruce A. Cree, MD, PhD, MAS, FAAN (UCSF, Multiple Sclerosis Center)	The institution of Dr. Cree has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. The institution of Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TG Therapeutics. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Autobahn. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avotres. The institution of Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Horizon. Dr. Cree has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Neuron23. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston Pharma. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Hexal/Sandoz. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kyverna. Dr. Cree has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunic AG. The institution of Dr. Cree has received research support from Genentech. The institution of Dr. Cree has received research support from Kyverna. Dr. Cree has received publishing royalties from a publication relating to health care.

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