Myasthenia GravisNCT04176211
The Impact of Myasthenia Gravis in the Real World
An observational study of Myasthenia Gravis, run by Vitaccess Ltd at 1 site in United Kingdom.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- How many people
- 2,847 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 12 Dec 2019to 31 May 2024
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults (age >18 years) with any stage and any subtype of myasthenia gravis disease
Who may be able to join
- 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
Who cannot join
- No specific exclusion criteria
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- 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
- Medical subject headings
- Myasthenia Gravis, Neuromuscular Diseases, Neuromuscular Junction Diseases, Nervous System Diseases, Autoimmune Diseases, Autoimmune Diseases of the Nervous System
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
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.
Measured: Once per six months throughout 2 year study duration
Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score
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.
Measured: Once per six months throughout 2 year study duration
Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance short form 6a change
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.
Measured: Once per six months throughout 2 year study duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2019
- Study start12 Dec 2019
- Primary completion31 May 2024
- Study completion31 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Ecologic or community
- Time perspective
- Prospective
- Enrolment
- 2,847 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Vitaccess Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark JW Larkin, PhD, Principal investigator, Vitaccess Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04176211
- Sponsor's protocol ID
- 5105-08-2019
- Status last verified
- May 2024
- First submitted
- 20 Nov 2019
- Last updated
- 3 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Aggregated data may be available via application.
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.
PubMed 34285010Dewilde 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.
PubMed 36720569Berrih-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.
PubMed 37169499Janssen 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.
PubMed 39068745
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Impact of Myasthenia Gravis in the Real World
- ClinicalTrials.gov ID
- NCT04176211
- Acronym / sponsor ID
- 5105-08-2019
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 2,847 participants (actual)
- Lead sponsor
- Vitaccess LtdIndustry (pharma, biotech, devices)
- Study start
- 12 Dec 2019 (actual)
- Primary completion
- 31 May 2024 (actual)
- Study completion
- 31 May 2024 (actual)
- First posted
- 25 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Ecologic or community
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults (age >18 years) with any stage and any subtype of myasthenia gravis disease
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- No specific exclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in EuroQol 5-dimension 5-level (EQ-5D-5L) score
Time frame: Monthly throughout 2 year study duration
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.
Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score
Time frame: Four times per six months throughout 2 year study duration
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.
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
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.
Change in Hospital Anxiety and Depression Scale (HADS) score
Time frame: Once per six months throughout 2 year study duration
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
Change in Health Utilities Index III (HUI3) score
Time frame: Once per 12 months throughout 2 year study duration
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.
COVID-19 survey results
Time frame: Monthly throughout 2 year study duration
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.
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
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.
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
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.
3 secondary outcomes
Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Dyspnea Characteristics score
Time frame: Once per six months throughout 2 year study duration
Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score
Time frame: Once per six months throughout 2 year study duration
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2024.
- Background: PubMed 34285010Berrih-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.
- Results publication: PubMed 36720569Dewilde 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.
- Results publication: PubMed 37169499Berrih-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.
- Derived from this study: PubMed 39068745Janssen 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.
- Individual participant dataSharing planned: No. Aggregated data may be available via application.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark JW Larkin, PhD principal investigator
Vitaccess Ltd
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myasthenia Gravis, run by Vitaccess Ltd at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in EuroQol 5-dimension 5-level (EQ-5D-5L) score (time frame: Monthly throughout 2 year study duration). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04176211, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2024.