The Impact of Myasthenia Gravis in the Real World

NCT04176211 | Completed DMC

• 1 other identifier: 5105-08-2019
• Study Type: observational
• Target: 2,847
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (8)

• Change in EuroQol 5-dimension 5-level (EQ-5D-5L) score

  The EQ-5D-5L comprises two parts - the EQ-5D-5L descriptive system and the EQ Visual Analogue Scale (EQ VAS). The descriptive system comprises 5 dimensions (mobility, self care, usual activities, pain/discomfort, and anxiety/depression), each with 5 levels (no problems, slight problems, moderate problems, severe problems, and extreme problems - i.e., higher scores represent worse health). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state. The EQ VAS records the respondent's self-rated health on vertical, visual analogue scale with endpoints labelled 'the best health you can imagine' and 'the worst health you can imagine'. Higher scores represent better self-perceived health.

  Monthly throughout 2 year study duration

• Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score

  The MG-ADL is a myasthenia gravis-specific 8-item scale, which assesses the severity of the following symptoms: talking, chewing, swallowing, breathing, impairment of ability to brush teeth/comb hair, impairment of ability to rise from a chair, double vision, and eyelid droop. Each of the 8 items has 4 response options; 0 (Mild), 1 (Moderate), 2 (Mild) and 3 (Severe). The MG-ADL total score can be calculated by totalling all 8 items out of a maximum score of 24. A higher score indicates severe impairment.

  Four times per six months throughout 2 year study duration

• Change in Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL15r) score

  The MG-QOL15r is a myasthenia gravis-specific health-related quality of life survey, which assesses the impact of myasthenia gravis over the past few weeks on the following domains: emotions, physical health, self-care, social life, and impact on role. Each of the 15 items has three response options: 0 (Not at all), 1 (Somewhat), and 2 (Very much). The MG-QoL15r total score can be calculated by totalling all 15 items out a maximum score of 30. A higher score indicates severe impairment.

  Once per six months throughout 2 year study duration

• Change in Hospital Anxiety and Depression Scale (HADS) score

  The HADS is a frequently used self-rating scale developed to assess psychological distress in non-psychiatric patients. It consists of two subscales, Anxiety and Depression. The instrument comprises 14 items, 7 for the anxiety subscale and 7 for the depression subscale. Each item is scored on a 4-point response scale, scale wording changes for each item with each coding response corresponding with greater impairment or severity. Domain scores can be calculated by totalling all 7 items out of a maximum score of 21. High scores indicate greater severity of that impairment

  Once per six months throughout 2 year study duration

• Change in Health Utilities Index III (HUI3) score

  The HUI3 is a generic 8-item survey, which provides descriptive evidence on multiple dimensions of health status, a score for each dimension of health, and a health-related quality of life score for overall health.Health dimensions include vision, hearing, speech, ambulation/mobility, pain, dexterity, emotion and cognition. Each dimension has five or six response options. It has recall periods of either 'usual health', the past week, two weeks, or four weeks. Scores on individual items are combined to given a combined health state which can then be converted to health utilities. Lower health utilities represent worse health.

  Once per 12 months throughout 2 year study duration

• COVID-19 survey results

  This is a bespoke survey designed to understand the impact of COVID-19 on study participants. It includes questions about whether participants have had COVID, and the consequences if so, whether they have been vaccinated, and how the wider pandemic has affected their MG treatment and quality of life.

  Monthly throughout 2 year study duration

• Change in Single Simple Question (SSQ) and Patient Acceptable Symptom States (PASS) scores

  The SSQ is a single question that asks respondents what percentage of normal they feel with respect to their MG. The PASS is a single question that asks respondents whether they would consider their current disease state to be satisfactory if they had to stay in it for the next few months. The SSQ and PASS will be presented to participants at the same time, as a question pair.

  Four times per six months throughout 2 year study duration

• Change in EuroQol 5-dimension 3-level (EQ-5D-3L) score

  The EQ-5D-3L is a three-level version of the EQ-5D-5L. It includes the same dimensions as the EQ-5D-5L, but each has only three levels: no problems, some problems, and extreme problems. The EQ VAS is unchanged from the EQ-5D-5L version.

  Monthly for three months for participants from the US and Italy only

Secondary Outcomes (3)

• Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Dyspnea Characteristics score

  Once per six months throughout 2 year study duration

• Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score

  Once per six months throughout 2 year study duration

• Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance short form 6a change

  Once per six months throughout 2 year study duration

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults (age \>18 years) with any stage and any subtype of myasthenia gravis disease

You may qualify if:

• 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

Sponsors & Collaborators

• Vitaccess Ltd: lead

Study Sites (1)

Vitaccess Ltd

Oxford, OX4 4GA, United Kingdom

Location

Related Publications (4)

• 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.

  PMID: 34285010 BACKGROUND

• 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.

  PMID: 36720569 RESULT

• 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.

  PMID: 37169499 RESULT

• Janssen MF, Dewilde S, Wolfe GI, Muppidi S, Phillips G. Psychometric properties of MG-ADL items and MG-ADL score: An assessment of distributional characteristics, validity and factor structure in two large datasets. J Neurol Sci. 2024 Aug 15;463:123135. doi: 10.1016/j.jns.2024.123135. Epub 2024 Jul 22.

  PMID: 39068745 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis; Neuromuscular Diseases; Neuromuscular Junction Diseases; Nervous System Diseases; Autoimmune Diseases; Autoimmune Diseases of the Nervous System

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Immune System Diseases

Study Officials

• Mark JW Larkin, PhD

  Vitaccess Ltd

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: ECOLOGIC OR COMMUNITY
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2019
• First Posted: November 25, 2019
• Study Start: December 12, 2019
• Primary Completion: May 31, 2024
• Study Completion: May 31, 2024
• Last Updated: June 3, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)