Contributions
Abstract: P1032
Type: Poster Sessions
Abstract Category: Clinical aspects of MS - Clinical assessment tools
Background: Passive ambulatory activity monitoring has been proposed as a new outcome measure for clinical trials (Block et al., J Neurol 2017). This type of monitoring estimates multiple activity parameters, including step count per epoch (daily or per minute). Passive ambulatory monitoring has good face validity and ample validation against research grade accelerometers. This measure is an exploratory endpoint of the pivotal SPI2 study, a randomised, double blind, placebo-controlled, phase 3 trial investigating the efficacy and safety of MD1003 (high dose Pharmaceutical grade Biotin) in patients (pts) with nonactive primary and secondary progressive MS (PPMS, SPMS).
Objective: To assess the feasibility and validity of remote activity monitoring as a measure of ambulatory disability in patients from the SPI2 study.
Methods: Remote monitoring of ambulatory activity in pts with PPMS/SPMS receiving either MD1003 300 mg/day or placebo will be collected using a Fitbit Flex® device between month (M) 0 and M15, and between M0 and M27 in the extension phase. The key variable will be the average daily step count (STEPS) over 1 week, recorded continuously in the natural environment as part of routine daily activity. Pts were trained by the investigational team on the setup, use, and maintenance of the device and asked to wear it as much as possible during the study. The daily goal on the device was initially set low to minimize the impact of motivation on ambulatory activity.
Results: As of December 2017, 67 active centres (USA: 34; Europe: 26; Canada: 7) had enrolled 242 pts, of which 188 had full STEPS data. In these 188 pts, median expanded disability status scale (EDSS) was 6.0 (interquartile range [IQR]: 5.0 - 6.5), 51.1% were female, 35.6% had PPMS, 64.4% had SPMS, and median disease duration (IQR) was 13 (7.0 - 18.0) years. Mean (SD) STEPS were 3,312 (2,498) for a mean of 24 out of 28 days (PPMS: 3,405 (2,349) STEPS; SPMS: 3,242 (2,612) STEPS). EDSS at the inclusion visit correlated with the step count averaged over 4 weeks (P< 0.001; r2=0.302). Updated baseline data will be presented.
Conclusion: When adjusted for timed 25-foot walk (T25FW), step counts for PPMS and SPMS were similar. Remote gait monitoring via Fitbit may be a good surrogate measure for ambulatory activity in clinical trials.
Disclosure: Bruce Cree has received personal compensation for consulting from Abbvie, Biogen, EMD Serono, GeNeuro, Novartis and Sanofi Genzyme.
Valerie Block has nothing to disclose.
Jeffrey Gelfand has received personal fees from medical-legal consulting.
Frédéric Sedel is an employee of MedDay Pharmaceuticals.
This study is sponsored by MedDay Pharmaceuticals.
Abstract: P1032
Type: Poster Sessions
Abstract Category: Clinical aspects of MS - Clinical assessment tools
Background: Passive ambulatory activity monitoring has been proposed as a new outcome measure for clinical trials (Block et al., J Neurol 2017). This type of monitoring estimates multiple activity parameters, including step count per epoch (daily or per minute). Passive ambulatory monitoring has good face validity and ample validation against research grade accelerometers. This measure is an exploratory endpoint of the pivotal SPI2 study, a randomised, double blind, placebo-controlled, phase 3 trial investigating the efficacy and safety of MD1003 (high dose Pharmaceutical grade Biotin) in patients (pts) with nonactive primary and secondary progressive MS (PPMS, SPMS).
Objective: To assess the feasibility and validity of remote activity monitoring as a measure of ambulatory disability in patients from the SPI2 study.
Methods: Remote monitoring of ambulatory activity in pts with PPMS/SPMS receiving either MD1003 300 mg/day or placebo will be collected using a Fitbit Flex® device between month (M) 0 and M15, and between M0 and M27 in the extension phase. The key variable will be the average daily step count (STEPS) over 1 week, recorded continuously in the natural environment as part of routine daily activity. Pts were trained by the investigational team on the setup, use, and maintenance of the device and asked to wear it as much as possible during the study. The daily goal on the device was initially set low to minimize the impact of motivation on ambulatory activity.
Results: As of December 2017, 67 active centres (USA: 34; Europe: 26; Canada: 7) had enrolled 242 pts, of which 188 had full STEPS data. In these 188 pts, median expanded disability status scale (EDSS) was 6.0 (interquartile range [IQR]: 5.0 - 6.5), 51.1% were female, 35.6% had PPMS, 64.4% had SPMS, and median disease duration (IQR) was 13 (7.0 - 18.0) years. Mean (SD) STEPS were 3,312 (2,498) for a mean of 24 out of 28 days (PPMS: 3,405 (2,349) STEPS; SPMS: 3,242 (2,612) STEPS). EDSS at the inclusion visit correlated with the step count averaged over 4 weeks (P< 0.001; r2=0.302). Updated baseline data will be presented.
Conclusion: When adjusted for timed 25-foot walk (T25FW), step counts for PPMS and SPMS were similar. Remote gait monitoring via Fitbit may be a good surrogate measure for ambulatory activity in clinical trials.
Disclosure: Bruce Cree has received personal compensation for consulting from Abbvie, Biogen, EMD Serono, GeNeuro, Novartis and Sanofi Genzyme.
Valerie Block has nothing to disclose.
Jeffrey Gelfand has received personal fees from medical-legal consulting.
Frédéric Sedel is an employee of MedDay Pharmaceuticals.
This study is sponsored by MedDay Pharmaceuticals.