A Phase 3 interventional study of Ravulizumab and Placebo in Generalized Myasthenia Gravis, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 106 sites in 15 countries. Per ClinicalTrials.gov, last updated 2024-05-28.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 3, Interventional, and Treatment
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
327 studies on the registry are indexed under Myasthenia Gravis; 144 are open to participants now.
This study's enrollment of 175 is above the median of 44 across 213 interventional studies indexed under Myasthenia Gravis.
Browse Myasthenia Gravis studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 24 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 73 (72%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Medical Conditions
Use of the following within the time period specified below:
Participants will receive ravulizumab for the duration of the study.
Biological: Ravulizumab
Participants will receive placebo during the 26-week randomized-controlled period of the study, after which they will enter the open-label extension period of the study and receive ravulizumab.
Drug: Placebo
Concentrated sterile, preservative-free aqueous solution (10 milligrams \[mg\]/milliliter \[mL\]) in single-use, 30-mL vial for intravenous (IV) infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
Also known as: ALXN1210, Ultomiris
Matching, sterile, preservative-free aqueous solution in single-use, 30-mL vial for IV infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26
The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess 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. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Estimates were based on Mixed Effect Repeated Measures (MMRM) that included treatment group, stratification factor region, and MG-ADL total score at baseline, study visit, and study visit by treatment group interaction.
Time frame: Baseline, Week 26
Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26
The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. The QMG scoring system was considered to be an objective evaluation of therapy for MG and was based on quantitative testing of sentinel muscle groups. A decrease in score indicated improvement. Estimates were based on MMRM that included treatment group, stratification factor region, and QMG total score at baseline, study visit, and study visit by treatment group interaction.
Time frame: Baseline, Week 26
Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26
The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. A decrease in score indicated improvement. Percentage of participants with a ≥5-point reduction in the QMG total score are reported. Estimates were based on a generalized linear mixed model (GLMM) that included treatment group, stratification factor region and QMG total score at baseline, study visit and study visit by treatment group interaction.
Time frame: Week 26
Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26
The revised Myasthenia Gravis Qualify of Life 15-item scale (MG-QOL15r) is a health-related QoL evaluative instrument specific to participants with MG. The MG-QOL15r was designed to provide information about participants' perception of impairment and disability, determine the degree to which disease manifestations are tolerated, and to be administered and interpreted easily. Each item was graded on a scale of 0 to 2, with 2 being the most severe. The range of MG-QOL15r score is 0 to 30. Higher scores indicated greater extent of and dissatisfaction with MG-related dysfunction. Estimates are based on MMRM that included treatment group, stratification factor region and MG-QOL15r score at baseline, study visit and study visit by treatment group interaction.
Time frame: Baseline, Week 26
Change From Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26
The Neuro-QOL Fatigue is a reliable and validated brief 19-item survey of fatigue, completed by the participant. Each items was rated on a scale of 1 to 5, with 5 being the most severe. The range of total score is 19 to 95. Higher scores indicated greater fatigue and greater impact of MG on activities. Estimates were based on MMRM that included treatment group, stratification factor region and Neuro-QoL Fatigue score at baseline, study visit, and study visit by treatment group interaction.
Time frame: Baseline, Week 26
Percentage of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26
The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess 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. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Percentage of participants with a ≥3-point reduction in the MG-ADL total score are reported. Estimates were based on a GLMM that included treatment group, stratification factor region and MG-ADL total score at baseline, study visit and study visit by treatment group interaction.
Time frame: Week 26
| Milestone | Ravulizumab to Ravulizumab | Placebo to Ravulizumab |
|---|---|---|
| Started | 86 | 89 |
| Received at least 1 dose of study drug | 86 | 89 |
| Completed | 79 | 83 |
| Not completed | 7 | 6 |
| Withdrew: Withdrawal by subject | 2 | 1 |
| Withdrew: Physician decision | 1 | 2 |
| Withdrew: Adverse event | 0 | 2 |
| Withdrew: Sponsor decision | 0 | 1 |
| Withdrew: Death | 2 | 0 |
| Withdrew: Protocol violation | 1 | 0 |
| Withdrew: Noncompliance | 1 | 0 |
| Milestone | Ravulizumab to Ravulizumab | Placebo to Ravulizumab |
|---|---|---|
| Started | 78 | 83 |
| Completed | 60 | 63 |
| Not completed | 18 | 20 |
| Withdrew: Participants who transitioned to alternative treatment by the investigator when the study closed | 3 | 5 |
| Withdrew: Death | 3 | 3 |
| Withdrew: Physician decision | 5 | 3 |
| Withdrew: Withdrawal by subject | 7 | 9 |
The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess 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. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Estimates were based on Mixed Effect Repeated Measures (MMRM) that included treatment group, stratification factor region, and MG-ADL total score at baseline, study visit, and study visit by treatment group interaction.
| units on a scale | Ravulizumab | Placebo |
|---|---|---|
| Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26 | -3.1 ± 0.38 | -1.4 ± 0.37 |
The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. The QMG scoring system was considered to be an objective evaluation of therapy for MG and was based on quantitative testing of sentinel muscle groups. A decrease in score indicated improvement. Estimates were based on MMRM that included treatment group, stratification factor region, and QMG total score at baseline, study visit, and study visit by treatment group interaction.
| units on a scale | Ravulizumab | Placebo |
|---|---|---|
| Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26 | -2.8 ± 0.46 | -0.8 ± 0.45 |
The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. A decrease in score indicated improvement. Percentage of participants with a ≥5-point reduction in the QMG total score are reported. Estimates were based on a generalized linear mixed model (GLMM) that included treatment group, stratification factor region and QMG total score at baseline, study visit and study visit by treatment group interaction.
| percentage of participants | Ravulizumab | Placebo |
|---|---|---|
| Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26 | 30.0 (19.2 to 43.5) | 11.3 (5.6 to 21.5) |
The revised Myasthenia Gravis Qualify of Life 15-item scale (MG-QOL15r) is a health-related QoL evaluative instrument specific to participants with MG. The MG-QOL15r was designed to provide information about participants' perception of impairment and disability, determine the degree to which disease manifestations are tolerated, and to be administered and interpreted easily. Each item was graded on a scale of 0 to 2, with 2 being the most severe. The range of MG-QOL15r score is 0 to 30. Higher scores indicated greater extent of and dissatisfaction with MG-related dysfunction. Estimates are based on MMRM that included treatment group, stratification factor region and MG-QOL15r score at baseline, study visit and study visit by treatment group interaction.
| score on a scale | Ravulizumab | Placebo |
|---|---|---|
| Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26 | -3.3 ± 0.71 | -1.6 ± 0.70 |
The Neuro-QOL Fatigue is a reliable and validated brief 19-item survey of fatigue, completed by the participant. Each items was rated on a scale of 1 to 5, with 5 being the most severe. The range of total score is 19 to 95. Higher scores indicated greater fatigue and greater impact of MG on activities. Estimates were based on MMRM that included treatment group, stratification factor region and Neuro-QoL Fatigue score at baseline, study visit, and study visit by treatment group interaction.
| units on a scale | Ravulizumab | Placebo |
|---|---|---|
| Change From Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26 | -7.0 ± 1.92 | -4.8 ± 1.87 |
The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess 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. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Percentage of participants with a ≥3-point reduction in the MG-ADL total score are reported. Estimates were based on a GLMM that included treatment group, stratification factor region and MG-ADL total score at baseline, study visit and study visit by treatment group interaction.
| percentage of participants | Ravulizumab | Placebo |
|---|---|---|
| Percentage of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26 | 56.7 (44.3 to 68.3) | 34.1 (23.8 to 46.1) |
Collected over Day 1 (after dosing) up to 2 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Randomized-Controlled Period: Ravulizumab | 2/86 (2.3%) | 20/86 (23.3%) | 77/86 (89.5%) |
| Randomized-Controlled Period: Placebo | 0/89 (0%) | 14/89 (15.7%) | 75/89 (84.3%) |
| Open-Label Extension Period: Placebo to Ravulizumab | 3/83 (3.6%) | 32/83 (38.6%) | 79/83 (95.2%) |
| Open-Label Extension Period: Ravulizumab to Ravulizumab | 3/78 (3.8%) | 24/78 (30.8%) | 77/78 (98.7%) |
| Event | Randomized-Controlled Period: Ravulizumab | Randomized-Controlled Period: Placebo | Open-Label Extension Period: Placebo to Ravulizumab | Open-Label Extension Period: Ravulizumab to Ravulizumab |
|---|---|---|---|---|
| COVID-19Infections and infestations | 0/86 | 1/89 | 2/83 | 4/78 |
| Myasthenia gravisNervous system disorders | 0/86 | 3/89 | 1/83 | 4/78 |
| COVID-19 pneumoniaInfections and infestations | 2/86 | 0/89 | 2/83 | 0/78 |
| CellulitisInfections and infestations | 0/86 | 2/89 | 2/83 | 1/78 |
| PneumoniaInfections and infestations | 0/86 | 0/89 | 2/83 | 1/78 |
| Spinal compression fractureInjury, poisoning and procedural complications | 0/86 | 0/89 | 2/83 | 0/78 |
| PyrexiaGeneral disorders | 0/86 | 0/89 | 2/83 | 0/78 |
| Transient ischaemic attackNervous system disorders | 2/86 | 0/89 | 0/83 | 0/78 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 1/86 | 0/89 | 0/83 | 1/78 |
| ErysipelasInfections and infestations | 0/86 | 0/89 | 1/83 | 1/78 |
| Event | Randomized-Controlled Period: Ravulizumab | Randomized-Controlled Period: Placebo | Open-Label Extension Period: Placebo to Ravulizumab | Open-Label Extension Period: Ravulizumab to Ravulizumab |
|---|---|---|---|---|
| COVID-19Infections and infestations | 5/86 | 3/89 | 26/83 | 30/78 |
| HeadacheNervous system disorders | 16/86 | 23/89 | 17/83 | 12/78 |
| DiarrhoeaGastrointestinal disorders | 13/86 | 11/89 | 10/83 | 6/78 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 6/86 | 7/89 | 9/83 | 11/78 |
| NasopharyngitisInfections and infestations | 3/86 | 5/89 | 11/83 | 8/78 |
| Back painMusculoskeletal and connective tissue disorders | 7/86 | 5/89 | 10/83 | 6/78 |
| FallInjury, poisoning and procedural complications | 0/86 | 0/89 | 10/83 | 2/78 |
| NauseaGastrointestinal disorders | 9/86 | 9/89 | 9/83 | 7/78 |
| Upper respiratory tract infectionInfections and infestations | 0/86 | 0/89 | 9/83 | 2/78 |
| Urinary tract infectionInfections and infestations | 5/86 | 4/89 | 8/83 | 7/78 |
Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
| Age, Continuous(years) | Ravulizumab | Placebo | Total |
|---|---|---|---|
| Mean | 58.0 ± 13.82 | 53.3 ± 16.05 | 55.6 ± 15.14 |
| Sex: Female, Male(Participants) | Ravulizumab | Placebo | Total |
|---|---|---|---|
| Female | 44 | 45 | 89 |
| Male | 42 | 44 | 86 |
| Ethnicity (NIH/OMB)(Participants) | Ravulizumab | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 2 | 5 | 7 |
| Not Hispanic or Latino | 79 | 78 | 157 |
| Unknown or Not Reported | 5 | 6 | 11 |
| Race/Ethnicity, Customized(Participants) | Ravulizumab | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 1 |
| Asian | 15 | 16 | 31 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 2 | 4 | 6 |
| White | 67 | 61 | 128 |
| Other | 0 | 1 | 1 |
| Unknown or Not Reported | 2 | 6 | 8 |
Showing the first 100 of 106 sites across 15 countries.
Documents are hosted by the registry — open the source record to download them.
Ran from Mar 2019 to primary completion in May 2021, 25 months.
Results were first posted in May 2022, 1 year after primary completion. A results delay was requested in Mar 2022.
Of 43 industry-sponsored phase 3 interventional studies under Myasthenia Gravis, half reached primary completion within 27 months of starting (the median), counting those still running; a quarter within 19 and three quarters within 39. This study's 25 months is close to that.
Counting every sponsor, half of 52 studies reached it within 30 months; a quarter within 20 and three quarters within 43.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ravulizumab
Alexion Pharmaceuticals, Inc. →