Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

The Impact of Myasthenia Gravis in the Real World

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04176211
Recruitment Status : Recruiting
First Posted : November 25, 2019
Last Update Posted : October 7, 2021
Sponsor:
Collaborator:
argenx
Information provided by (Responsible Party):
Vitaccess Ltd

Tracking Information
First Submitted Date November 20, 2019
First Posted Date November 25, 2019
Last Update Posted Date October 7, 2021
Actual Study Start Date December 12, 2019
Estimated Primary Completion Date November 30, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 29, 2021)
  • 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.
Original Primary Outcome Measures
 (submitted: November 22, 2019)
  • 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: Monthly 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: Monthly 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: Monthly 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: Monthly 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.
Change History
Current Secondary Outcome Measures
 (submitted: April 21, 2021)
  • Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Dyspnea Characteristics score [ Time Frame: Once per six months throughout 2 year study duration ]
    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.
  • Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score [ Time Frame: Once per six months throughout 2 year study duration ]
    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.
  • 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 ]
    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.
Original Secondary Outcome Measures
 (submitted: November 22, 2019)
  • Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Dyspnea Characteristics score [ Time Frame: Monthly throughout 2 year study duration ]
    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.
  • Change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score [ Time Frame: Monthly throughout 2 year study duration ]
    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.
  • Change in Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance short form 6a change [ Time Frame: Monthly throughout 2 year study duration ]
    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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title The Impact of Myasthenia Gravis in the Real World
Official Title The Impact of Myasthenia Gravis in the Real World
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 will be collected from people with MG using the MyRealWorld™ RWE platform , developed by the healthcare digital research company, Vitaccess. Participants will download a 'bring your own device' (BYOD) study app, MyRealWorld™ MG, onto their smartphones, which they will use to enter data and complete surveys about their MG.
Study Type Observational [Patient Registry]
Study Design Observational Model: Ecologic or Community
Time Perspective: Prospective
Target Follow-Up Duration 2 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Adults (age >18 years) with any stage and any subtype of myasthenia gravis disease
Condition Myasthenia Gravis
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Berrih-Aknin S, Claeys KG, Law N, Mantegazza R, Murai H, Saccà 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: November 22, 2019)
2000
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 31, 2022
Estimated Primary Completion Date November 30, 2021   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Mark JW Larkin, PhD +33 645 900 348 mark.larkin@vitaccess.com
Listed Location Countries United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number NCT04176211
Other Study ID Numbers 5105-08-2019
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Plan Description: Aggregated data may be available via application.
Responsible Party Vitaccess Ltd
Study Sponsor Vitaccess Ltd
Collaborators argenx
Investigators
Principal Investigator: Mark JW Larkin, PhD Vitaccess Ltd
PRS Account Vitaccess Ltd
Verification Date September 2021