Trial · NCT04115293
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
NCT04115293, Phase 3, Completed, Ra Pharmaceuticals, Inc.; results first posted 2023-01-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis. Trials, record NCT04115293. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04115293
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
- NCT number
- NCT04115293
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 174 participants, actual
- Lead sponsor
- Ra Pharmaceuticals, Inc.
- Conditions
- Myasthenia Gravis, Generalized
- Interventions
- zilucoplan (RA101495), Placebo
- First posted
- 2019-10-04
- Last updated
- 2025-07-16
- Results first posted
- 2023-01-17
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score | From Baseline to End of Treatment (Week 12) | The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement. |
| Secondary | Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score | From Baseline to End of Treatment (Week 12) | The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement. |
| Secondary | Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score | From Baseline to End of Treatment (Week 12) | The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing \[0 to 6\], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement. |
| Secondary | Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score | From Baseline to End of Treatment (Week 12) | The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement. |
| Secondary | Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period | From Baseline to End of Treatment (Week 12) | Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1. |
| Secondary | Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy | End of Treatment (Week 12) | Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption. |
| Secondary | Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy | End of Treatment (Week 12) | Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. |
| Secondary | Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12 | End of Treatment (Week 12) | Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. |
| Secondary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up]) | A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score | From Baseline to End of Treatment (Week 12) | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score | From Baseline to End of Treatment (Week 12) | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score | From Baseline to End of Treatment (Week 12) | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score | From Baseline to End of Treatment (Week 12) | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period | From Baseline to End of Treatment (Week 12) | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Days | MEDIAN |
| SECONDARY | Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy | End of Treatment (Week 12) | The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy | End of Treatment (Week 12) | The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12 | End of Treatment (Week 12) | The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up]) | The Safety Set (SS) included all participants who received at least 1 dose of study drug based on the actual study treatment received. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score | Placebo | -2.30 | 95% Confidence Interval -3.17 to -1.43 | score on a scale | |
| Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score | Zilucoplan 0.3 mg/kg | -4.39 | 95% Confidence Interval -5.28 to -3.50 | score on a scale | |
| Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score | Placebo | -3.25 | 95% Confidence Interval -4.32 to -2.17 | score on a scale | |
| Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score | Zilucoplan 0.3 mg/kg | -6.19 | 95% Confidence Interval -7.29 to -5.08 | score on a scale | |
| Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score | Placebo | -5.42 | 95% Confidence Interval -6.98 to -3.86 | score on a scale | |
| Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score | Zilucoplan 0.3 mg/kg | -8.62 | 95% Confidence Interval -10.22 to -7.01 | score on a scale | |
| Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score | Placebo | -3.16 | 95% Confidence Interval -4.65 to -1.67 | score on a scale | |
| Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score | Zilucoplan 0.3 mg/kg | -5.65 | 95% Confidence Interval -7.17 to -4.12 | score on a scale | |
| Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period | Placebo | NA | Full Range NA to NA | Days | |
| Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period | Zilucoplan 0.3 mg/kg | NA | Full Range NA to NA | Days |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 88 | |
| Overall Study | STARTED | Zilucoplan 0.3 mg/kg | 86 | |
| Overall Study | COMPLETED | Placebo | 84 | |
| Overall Study | COMPLETED | Zilucoplan 0.3 mg/kg | 82 | |
| Overall Study | NOT COMPLETED | Placebo | 4 | |
| Overall Study | NOT COMPLETED | Zilucoplan 0.3 mg/kg | 4 |
Source ClinicalTrials.gov
Adverse events64
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 2 | 88 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | Zilucoplan 0.3 mg/kg | 9 | 86 | 9 |
| other | Vomiting | Gastrointestinal disorders | Placebo | 5 | 88 | 5 |
| other | Vomiting | Gastrointestinal disorders | Zilucoplan 0.3 mg/kg | 3 | 86 | 3 |
| other | Injection site bruising | General disorders | Placebo | 8 | 88 | 11 |
| other | Injection site bruising | General disorders | Zilucoplan 0.3 mg/kg | 14 | 86 | 18 |
| other | Injection site pain | General disorders | Placebo | 3 | 88 | 3 |
| other | Injection site pain | General disorders | Zilucoplan 0.3 mg/kg | 8 | 86 | 9 |
| other | Urinary tract infection | Infections and infestations | Placebo | 4 | 88 | 4 |
| other | Urinary tract infection | Infections and infestations | Zilucoplan 0.3 mg/kg | 7 | 86 | 7 |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 1 | 88 |
| Total, all-cause mortality | Zilucoplan 0.3 mg/kg | 1 | 86 |
| Total, other adverse events | Placebo | 34 | 88 |
| Total, other adverse events | Zilucoplan 0.3 mg/kg | 41 | 86 |
| Total, serious adverse events | Placebo | 13 | 88 |
| Total, serious adverse events | Zilucoplan 0.3 mg/kg | 11 | 86 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Zilucoplan 0.3 mg/kg vs Placebo: deaths from any cause | Trial comparisons |
| Zilucoplan 0.3 mg/kg vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04115293
GET https://conductscience.org/api/trials/v1/NCT04115293/events?page=2