- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04176211
The Impact of Myasthenia Gravis in the Real World
Study Overview
Status
Conditions
Detailed Description
Study Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Mark JW Larkin, PhD
- Phone Number: +33 645 900 348
- Email: mark.larkin@vitaccess.com
Study Locations
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Oxford, United Kingdom, OX4 4GA
- Recruiting
- Vitaccess Ltd
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Contact:
- Mark JW Larkin, PhD
- Phone Number: +33 645 900 348
- Email: mark.larkin@vitaccess.com
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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
Study Plan
How is the study designed?
Design Details
- Observational Models: Ecologic or Community
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Change in EuroQol 5-dimension 5-level (EQ-5D-5L) score
Time Frame: Monthly throughout 2 year study duration
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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
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Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score
Time Frame: Four times per six months throughout 2 year study duration
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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.
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Four times per six months throughout 2 year study duration
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Change in Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL15r) score
Time Frame: Once per six months throughout 2 year study duration
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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.
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Once per six months throughout 2 year study duration
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Change in Hospital Anxiety and Depression Scale (HADS) score
Time Frame: Once per six months throughout 2 year study duration
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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
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Once per six months throughout 2 year study duration
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Change in Health Utilities Index III (HUI3) score
Time Frame: Once per 12 months throughout 2 year study duration
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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.
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Once per 12 months throughout 2 year study duration
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COVID-19 survey results
Time Frame: Monthly throughout 2 year study duration
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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.
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Monthly throughout 2 year study duration
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Change in Single Simple Question (SSQ) and Patient Acceptable Symptom States (PASS) scores
Time Frame: Four times per six months throughout 2 year study duration
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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.
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Four times per six months throughout 2 year study duration
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Change in EuroQol 5-dimension 3-level (EQ-5D-3L) score
Time Frame: Monthly for three months for participants from the US and Italy only
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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.
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Monthly for three months for participants from the US and Italy only
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Dyspnea Characteristics score
Time Frame: Once per six months throughout 2 year study duration
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The PROMIS sleep disturbance measure assesses self-reported frequency, severity, and duration of dyspnea (shortness of breath) over the last 7 days using either a 0-10 numeric rating scale or 0-4 rating scale to assess dyspnea characteristics.Each item is evaluated individually according to the numeric rating scale.
No total score is calculated for this instrument.
Higher scores on items indicate more severe symptoms.
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Once per six months throughout 2 year study duration
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Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score
Time Frame: Once per six months throughout 2 year study duration
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The FACIT-Fatigue measure is a generic 13-item survey which assesses fatigue levels and impact over the past 7 days.
The level of fatigue is measured on a 4-point Likert scale (4 = not at all fatigue to 0 = very much fatigued).
Scores range from 0-52.
The higher the score, the better the quality of life.
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Once per six months throughout 2 year study duration
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Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance short form 6a change
Time Frame: Once per six months throughout 2 year study duration
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The PROMIS sleep disturbance measure assesses self-reported perceptions of sleep quality, sleep depth, and restoration associated with sleep over the last 7 days using a 5-point scale.
Scores range from 6-30.
Higher scores represent worse sleep disturbance.
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Once per six months throughout 2 year study duration
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Mark JW Larkin, PhD, Vitaccess Ltd
Publications and helpful links
General Publications
- 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.
- 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.
- 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.
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Immune System Diseases
- Neoplasms
- Autoimmune Diseases of the Nervous System
- Autoimmune Diseases
- Neoplasms by Site
- Neurologic Manifestations
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Nervous System Neoplasms
- Paraneoplastic Syndromes, Nervous System
- Paraneoplastic Syndromes
- Neuromuscular Junction Diseases
- Muscle Weakness
- Myasthenia Gravis
Other Study ID Numbers
- 5105-08-2019
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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