An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors
CompletedPhase 3Results postedLast updated 3 September 2025
What this trial tests
Phase 3 trial testing zilucoplan (RA101495) in Generalized Myasthenia Gravis in 26 participants. Completed in 23 October 2024.
Adults 18 to 85, any sex, with Generalized Myasthenia Gravis. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Main Treatment PeriodPrimary· From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)
An AE was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) and up to and including 40 days after the final dose (or last contact depending on which occurs first).
Group
Value
95% CI
Zilucoplan 0.3 mg/kg
73.1
Percentage of Participants With TEAEs Leading to Withdrawal of Study Medication Over the Main Treatment PeriodPrimary· From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)
An AE was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was 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).
Group
Value
95% CI
Zilucoplan 0.3 mg/kg
7.7
Change From Baseline to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) ScoreSecondary· From Baseline to Week 12
The MG-ADL is a brief 8-item interviewer-administered patient-reported outcome (PRO) designed to evaluate MG symptom severity. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. Each item is assessed on a 4-point scale, where a score of 0 represents normal function and a score of 3 represents severely decreased ability to perform that function. The total MG-ADL score ranges from 0 to 24, with a higher score indicating more severe impairment. A positive change in score indicates worsening and negative change indicates improvement.
Group
Value
95% CI
Zilucoplan 0.3 mg/kg
-1.15
-2.11 – -0.19
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) ScoreSecondary· From Baseline to Week 12
The QMG is a standardized and validated quantitative strength scoring system that included 13 items in the following categories: ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. Scoring for each item ranges from no weakness (0) to severe weakness (3), with an overall score range from 0 to 39. Higher scores represent more severe impairment. A positive change in score indicates worsening and negative change indicates improvement.
Group
Value
95% CI
Zilucoplan 0.3 mg/kg
-1.24
-2.64 – 0.16
Percentage of Participants With Serious TEAEs Over the Main Treatment PeriodSecondary· From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)
Treatment-emergent serious adverse events (serious TEAEs) were any untoward medical incidence in a participant during administered study treatment, whether or not these events were related to study treatment and additionally were emergent untoward medical occurrence that at any dose:
* Results in death
* Is life-threatening
* Required in patient hospitalisation or prolongation of existing hospitalisation
* Results in persistent disability/incapacity
* Was a congenital anomaly or birth defect
* Important medical events
Group
Value
95% CI
Zilucoplan 0.3 mg/kg
3.8
Percentage of Participants With Study Withdrawal Over the Main Treatment PeriodSecondary· From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)
Percentage of participants with study withdrawal based to pre-defined reasons in the protocol were reported.
Group
Value
95% CI
Zilucoplan 0.3 mg/kg
11.5
Adverse events — posted to ClinicalTrials.gov
Time frame: From Baseline up to 84 weeks (Overall Treatment Period).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The purpose of the study is to evaluate the safety and tolerability of switching from intravenous (IV) complement component 5 (C5) inhibitors to subcutaneous (SC) Zilucoplan in study participants with generalized myasthenia gravis (gMG)
Publications & conference data
4 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04225871 — Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis
· Phase 3
· active not recruiting
NCT04115293 — Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
· Phase 3
· completed
NCT03315130 — Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
· Phase 2
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by UCB Biopharma SRL
Last refreshed: 3 September 2025
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