2024 American Academy of Neurology Abstract Website

Edouard Berling¹, David Orlikowski², Guillaume Nicolas³, Hélène Prigent³, Ghislain N’Dah Sekou³, Loïc Carment⁴, Clarissa Gorin⁴, Lissandra Klaeyle⁴, Dellini Ravindra⁴, Emma Touré Cuq⁴, Alizé Vives⁴, Martin Keller⁵, Emrah Aras⁶, Saad Zinaï⁴, Pascal Laforêt³
¹Nord-Est/Ile-de-France Neuromuscular Reference Center, UMR 1179, CIC 1429, Neurology Department, Raymond-Poincaré Hospital, Garches and FHU PHENIX, UVSQ Paris-Saclay University, France, ²Nord-Est/Ile-de-France Neuromuscular Reference Center, UMR 1179, CIC 1429, Intensive Care Unit, Raymond-Poincaré Hospital, Garches and FHU PHENIX, UVSQ Paris-Saclay University, France, ³Nord-Est/Ile-de-France Neuromuscular Reference Center, UMR 1179, Neurology Department, Raymond-Poincaré Hospital, Garches, FHU PHENIX, UVSQ Paris-Saclay University, France, ⁴Ad Scientiam, Paris, France, ⁵Alexion GmbH, ⁶Alexion Pharmaceuticals Inc.

Objective:

To describe usability validation results and subsequent investigational study designed to validate the clinical performance of a novel smartphone-based device intended for patients with generalized myasthenia gravis (gMG).

Background:

gMG is an antibody-mediated autoimmune disease that causes weakness of the bulbar, ocular, and skeletal limb muscles. Its fluctuating symptoms hamper adequate monitoring for patients and clinicians.

To address this challenge, ME&MGTM, a Software as a Medical Device, was developed to enable patients to self-monitor in their daily environment by collecting objective digital biomarkers on main gMG symptoms (ptosis, dysarthria, respiratory capacity, upper and lower limb weakness), while communicating the results to clinicians.

Design/Methods:

26 gMG patients were recruited from the USA and France to participate in a 90-minute evaluation following usability engineering methods to validate the ME&MG^TM interface (mobile application).

The DOMYA clinical investigation, a prospective, low-interventional, open-label, international study (USA, France), was designed to validate the clinical performance and safety of ME&MG^TM. It will enroll 94 gMG adult patients with positive anti-acetylcholine receptor antibodies. The primary objective will measure the association between ME&MG^TM scores measured at home, with Quantitative Myasthenia Gravis (QMG) corresponding subscores measured in the clinic using a minimum significant correlation coefficient of 0.65. Secondary objectives will confirm analytical performance, adherence and safety.

Results:

ME&MG^TM was perceived as very easy to use (mean=9.2/10, standard deviation (SD)=0.90) with a success rate of over 96% for each tested feature.

After a study duration of 30 months, final results of the DOMYA study will be analyzed and made available in 2025.

Conclusions:

Through this usability validation evaluation, we demonstrated that ME&MG^TM is a safe and usable device.

The DOMYA clinical study aims to demonstrate ME&MG^TM clinical performance, enabling its registration as a medical device in Europe and the USA.