Clinical trial NCT04176211: The Impact of Myasthenia Gravis in the Real World
Official title: The Impact of Myasthenia Gravis in the Real World
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myasthenia Gravis
Keywords: Myasthenia Gravis Disease
Neuromuscular Diseases
Neuromuscular Junction Diseases
Nervous System Diseases
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Ocular Myasthenia Gravis
Generalized Myasthenia Gravis
Myasthenia Gravis, Generalized
Lead sponsor: Vitaccess Ltd
Sponsor class: INDUSTRY
First posted: 2019-11-25
Enrollment: 2847
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adults (age \>18 years) with any stage and any subtype of myasthenia gravis disease

Brief summary: An international, non-interventional, prospective, community recruited, longitudinal, lifestyle study which aims to collect Real-World Evidence (RWE) from people living with myasthenia gravis (MG) in order to map the disease's natural history, its burden on patients and their families, its treatment, as well as its medical, social and pharmacoeconomic impact.

Detailed description: Data was collected from people with MG using the MyRealWorld™ RWE platform , developed by the healthcare digital research company, Vitaccess. Participants downloaded a 'bring your own device' (BYOD) study app, MyRealWorld™ MG, onto their smartphones, which they used to enter data and complete surveys about their MG.

Eligibility criteria: Inclusion Criteria:

* Adults (aged 18 years and over) with any stage and any subtype of myasthenia gravis disease
* Resident in Belgium, Canada, France, Germany, Italy, Japan, Spain, the UK, the USA, or Denmark

Exclusion Criteria:

* No specific exclusion criteria

Primary outcomes:
measure: Change in EuroQol 5-dimension 5-level (EQ-5D-5L) score
timeFrame: Monthly throughout 2 year study duration
measure: Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score
timeFrame: Four times per six months throughout 2 year study duration
measure: Change in Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL15r) score
timeFrame: Once per six months throughout 2 year study duration
measure: Change in Hospital Anxiety and Depression Scale (HADS) score
timeFrame: Once per six months throughout 2 year study duration
measure: Change in Health Utilities Index III (HUI3) score
timeFrame: Once per 12 months throughout 2 year study duration
measure: COVID-19 survey results
timeFrame: Monthly throughout 2 year study duration
measure: Change in Single Simple Question (SSQ) and Patient Acceptable Symptom States (PASS) scores
timeFrame: Four times per six months throughout 2 year study duration
measure: Change in EuroQol 5-dimension 3-level (EQ-5D-3L) score
timeFrame: Monthly for three months for participants from the US and Italy only
Secondary outcomes:
measure: Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Dyspnea Characteristics score
timeFrame: Once per six months throughout 2 year study duration
measure: Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score
timeFrame: Once per six months throughout 2 year study duration
measure: Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance short form 6a change
timeFrame: Once per six months throughout 2 year study duration
Other outcomes:
Locations:
city: Oxford
country: United Kingdom
facility: Vitaccess Ltd
Study officials:
affiliation: Vitaccess Ltd
name: Mark JW Larkin, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Berrih-Aknin S, Claeys KG, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen MF, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S. Patient-reportedimpact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG). BMJ Open. 2021 Jul 20;11(7):e048198. doi: 10.1136/bmjopen-2020-048198.
citation: Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys KG, Muppidi S, Mantegazza R, Sacca F, Meisel A, Bassez G, Murai H, Janssen MF. Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG. BMJ Open. 2023 Jan 31;13(1):e066445. doi: 10.1136/bmjopen-2022-066445.
citation: Berrih-Aknin S, Palace J, Meisel A, Claeys KG, Muppidi S, Sacca F, Amini F, Larkin M, Quinn C, Beauchamp J, Philips G, De Ruyck F, Ramirez J, Paci S. Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG). BMJ Open. 2023 May 11;13(5):e068104. doi: 10.1136/bmjopen-2022-068104.