Trial Outcomes & Findings for Myasthenia Gravis Inebilizumab Trial (NCT NCT04524273)

A total of 238 participants were enrolled in 18 countries, from 15 October 2020. This study is ongoing. Data cut-off date for primary analysis was 28 May 2024.

The study consisted of a Randomized Controlled Period (RCP) which lasted 52 weeks for the anti-AChR-Ab+ population and 26 weeks for the anti-MuSK-Ab+ population and an optional Open-label Period (OLP) of the duration of 3 years. This study also has a 2-year safety follow up period. The primary analysis was conducted when the last participants had the opportunity to complete Week 26 visit.

Myasthenia Gravis Inebilizumab Trial

MG-ADL score is an 8-item questionnaire that focuses on relevant symptoms and functional performance of activities of daily living over the previous 7 days. The MG-ADL score assesses disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items) impairment related to effects from MG. Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL scores is 0-24. A higher score represents more severe disease Outcome measure is reported for the overall population.

The QMG score is a validated outcome comprised of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item). Each item has a possible score of between 0 and 3 points. The total score range is 0-39 points, with higher score indicating more severe disease. Outcome measure is reported for the overall population.

MG-ADL score is an 8-item questionnaire that focuses on relevant symptoms and functional performance of activities of daily living over the previous 7 days. The MG-ADL score assesses disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items) impairment related to effects from MG. Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL scores is 0-24. A higher score represents more severe disease. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

The QMG score is a validated outcome comprised of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item). Each item has a possible score of between 0 and 3 points. The total score range is 0-39 points, with higher score indicating more severe disease. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

MG-ADL score is an 8-item questionnaire that focuses on relevant symptoms and functional performance of activities of daily living over the previous 7 days. The MG-ADL score assesses disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items) impairment related to effects from MG. Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL scores is 0-24. A higher score represents more severe disease. The protocol-allowed rescue therapy options included intravenous immunoglobulin (IVIg) and therapeutic plasma exchange (PLEX). Outcome measure is reported for the overall population.

MG-ADL score is an 8-item questionnaire that focuses on relevant symptoms and functional performance of activities of daily living over the previous 7 days. The MG-ADL score assesses disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items) impairment related to effects from MG. Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL scores is 0-24. A higher score represents more severe disease. The protocol-allowed rescue therapy options included IVIg and PLEX. Outcome measure is reported in Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

MG-ADL score is an 8-item questionnaire that focuses on relevant symptoms and functional performance of activities of daily living over the previous 7 days. The MG-ADL score assesses disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items) impairment related to effects from MG. Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL scores is 0-24. A higher score represents more severe disease. The protocol-allowed rescue therapy options included IVIg and PLEX. Outcome measure is reported for the Anti-AChR-Ab+ population.

The protocol-allowed rescue therapy options included corticosteroids, IVIg and PLEX. Outcome measure is reported for the Anti-AChR-Ab+ population.

MG-ADL score is an 8-item questionnaire that focuses on relevant symptoms and functional performance of activities of daily living over the previous 7 days. The MG-ADL score assesses disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items) impairment related to effects from MG. Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL scores is 0-24. A higher score represents more severe disease. Outcome measure is reported for the Anti-AChR-Ab+ population.

The QMG score is a validated outcome comprised of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item). Each item has a possible score of between 0 and 3 points. The total score range is 0-39 points, with higher score indicating more severe disease. Outcome measure is reported for the Anti-AChR-Ab+ population.

The MGC score consists of test items from MG-ADL score and the QMG score, with weighted response options. Scores range from 0-50, with higher scores indicating worse disease manifestations. Outcome measure is reported for the overall population.

The MGC score consists of test items from MG-ADL score and the QMG score, with weighted response options. Scores range from 0-50, with higher scores indicating worse disease manifestations. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

The MGC score consists of test items from MG-ADL score and the QMG score, with weighted response options. Scores range from 0-50, with higher scores indicating worse disease manifestations. Outcome measure is reported for the Anti-AChR-Ab+ population.

MGQOL-15r score is a validated, patient-scored instrument, which measures the impact of MG on health-related quality of life (HRQoL). The 15 items in the questionnaire evaluate mobility (9 items), symptoms (3 items), general contentment (1 item), and emotional well-being (2 items) domains. Each item is rated on a 3-point scale ranging from 0 ("not at all") to 2 ("very much") based on their experience "over the past few weeks." Items scores are summed to generate a total score ranging from 0-45, with higher score indicating worse HRQoL. Outcome measure is reported for the overall population.

MGQOL-15r score is a validated, patient-scored instrument, which measures the impact of MG on health-related quality of life (HRQoL). The 15 items in the questionnaire evaluate mobility (9 items), symptoms (3 items), general contentment (1 item), and emotional well-being (2 items) domains. Each item is rated on a 3-point scale ranging from 0 ("not at all") to 2 ("very much") based on their experience "over the past few weeks." Items scores are summed to generate a total score ranging from 0-45, with higher score indicating worse HRQoL. Outcome measure is reported in Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

MGQOL-15r score is a validated, patient-scored instrument, which measures the impact of MG on health-related quality of life (HRQoL). The 15 items in the questionnaire evaluate mobility (9 items), symptoms (3 items), general contentment (1 item), and emotional well-being (2 items) domains. Each item is rated on a 3-point scale ranging from 0 ("not at all") to 2 ("very much") based on their experience "over the past few weeks." Items scores are summed to generate a total score ranging from 0-45, with higher score indicating worse HRQoL. Outcome Measure is reported for the Anti-AChR-Ab+ population.

The self-report measure PGIC reflects a participant's belief about the efficacy of treatment. PGIC is a 7 point scale depicting a participant's rating of overall improvement. Participant rate their change as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse", Outcome measure is reported for the overall population.

The self-report measure PGIC reflects a participant's belief about the efficacy of treatment. PGIC is a 7 point scale depicting a participant's rating of overall improvement. Participants rate their change as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse". Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

The self-report measure PGIC score reflects a participant's belief about the efficacy of treatment. PGIC is a 7 point scale depicting a participant's rating of overall improvement. participants rate their change as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse". Outcome measure is reported for the Anti-AChR-Ab+ population.

An exacerbation was defined as one of the following: * Use of protocol defined rescue therapy, or * Myasthenic crisis, defined as worsening of myasthenic weakness requiring intubation or non-invasive ventilation to avoid intubation, except when these measures are employed during routine postoperative management, or * Significant symptomatic worsening to a score of 3 or a 2-point worsening from baseline on any one of the individual MG-ADL items other than double vision or eyelid droop. Outcome measure is reported for the overall population.

An exacerbation was defined as one of the following: * Use of protocol defined rescue therapy, or * Myasthenic crisis, defined as worsening of myasthenic weakness requiring intubation or non-invasive ventilation to avoid intubation, except when these measures are employed during routine postoperative management, or * Significant symptomatic worsening to a score of 3 or a 2-point worsening from baseline on any one of the individual MG-ADL items other than double vision or eyelid droop. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

An exacerbation was defined as one of the following: * Use of protocol defined rescue therapy, or * Myasthenic crisis, defined as worsening of myasthenic weakness requiring intubation or non-invasive ventilation to avoid intubation, except when these measures are employed during routine postoperative management, or * Significant symptomatic worsening to a score of 3 or a 2-point worsening from baseline on any one of the individual MG-ADL items other than double vision or eyelid droop. Outcome measure is reported for the Anti-AChR-Ab+ population.

MSE was defined as MG-ADL= 0 or 1. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK+ populations.

Outcome measure is reported for the overall population.

Outcome measure is reported in Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

Outcome measure is reported for the Anti-AChR-Ab+ population.

Outcome measure is reported for the overall population.

Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

Outcome measure is reported for the Anti-AChR-Ab+ population.

An AE is any untoward medical occurrence associated with the use of the IP, whether or not it is considered related. Clinically significant changes from baseline in clinical laboratory results, vital signs and ECGs were also reported as AEs. An AESI is an event of medical interest specific to the understanding of the IP and which may require close monitoring and collection of additional information. A SAE is considered "serious" if it results in any of the following outcomes: * Death; * A life-threatening AE. An event is considered "life-threatening" if its occurrence places the patient or subject at immediate risk of death; * Inpatient hospitalization or prolongation of existing hospitalization; * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; * A congenital anomaly/birth defect; * May require medical or surgical intervention to prevent one of the outcomes listed in this definition.

Treatment emergent ADA were defined as ADA positive post-baseline only or boosted pre-existing ADA titer. Outcome measure is reported for the overall population.

Treatment emergent ADA were defined as ADA positive post-baseline only or boosted pre-existing ADA titer. Outcome measure is reported in Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

Treatment emergent ADA were defined as ADA positive post-baseline only or boosted pre-existing ADA titer. Outcome measure is reported for the Anti-AChR-Ab+ population.

On inebilizumab dosing days, inebilizumab pharmacokinetic (PK) serum samples were collected pre-dose and approximately 15 minutes (± 5 minutes) after completion of the IP infusion. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

On inebilizumab dosing days, inebilizumab PK serum samples were collected pre-dose and approximately 15 minutes (± 5 minutes) after completion of the IP infusion. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.

On inebilizumab dosing days, inebilizumab PK serum samples were collected pre-dose and approximately 15 minutes (± 5 minutes) after completion of the IP infusion. Outcome measure is reported for the Anti-AChR-Ab+ and Anti-MuSK-Ab+ populations.