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Generalized Myasthenia GravisPhase 3NCT05514873

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

A Phase 3 study testing zilucoplan (RA101495) in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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) Read more about this study

Who is it for?
Ages 18 to 85 years
Conditions the study is for
Generalized Myasthenia Gravis
What is being tested?
zilucoplan (RA101495)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
26 participants
Where
12 sitesUnited States
Study dates
31 Oct 2022to 23 Oct 2024
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participant has been treated with an intravenous (IV) complement component 5 (C5) inhibitor approved for the treatment of generalized myasthenia gravis (gMG) at the recommended dose regimen for at least 3 months (for eculizumab) or 4 months (for ravulizumab) prior to Screening with a clinically stable disease as per the Investigator's judgment.
  • Participant is willing to switch from his/her current IV C5 inhibitor to subcutaneous (SC) zilucoplan (ZLP)
  • Participant has a documented diagnosis of gMG (Myasthenia Gravis Foundation of America; MGFA Class II-IVa) at Screening based on participant history and supported by previous evaluations
  • Participant has a well-documented record of positive serology for acetylcholine receptor binding autoantibodies prior to Screening
  • Participant has no more than a 2-point change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Baseline compared with the Screening Visit
  • Participant has had no change in corticosteroid dose during the Screening Period and no change in corticosteroid dose is anticipated to occur during the 12-week Main Treatment Period
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  • Participant has had no change in immunosuppressive therapy, including dose, during the Screening Period and no change in immunosuppressive therapy is anticipated to occur during the 12-week Main Treatment Period
  • Participant has a record of vaccination with at least 1 dose of a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of ZLP if not vaccinated within 3 years prior to the start of study medication
  • Male and/or female
  • A male participant is recommended to agree to use contraception during the study and for at least 40 days (5 half lives) after the last dose of study medication, and refrain from donating sperm during this period.
  • A female participant is eligible to participate if she is not pregnant; not breastfeeding, and at least one of the following conditions applies:
  • Not a woman of childbearing potential (WOCBP) OR
  • A WOCBP who agrees to follow the contraceptive guidance during the study and for at least 40 days (5 half lives) after the last dose of study medication.
  • Participant is capable of giving signed informed consent

Who cannot join

  • Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study
  • Participant has a known hypersensitivity to any components of the study medication as stated in this protocol
  • Participant has had a thymectomy within 6 months prior to Baseline or has one scheduled to occur during the 12-week Main Treatment Period
  • Participant has a history of meningococcal disease
  • Participant has or has had a current or recent systemic infection within 2 weeks prior to Baseline or an infection requiring IV antibiotics within 4 weeks prior to Baseline
  • Participant has active malignancy (except curatively resected squamous or basal cell carcinoma of the skin) requiring surgery, chemotherapy, or radiation within the prior 12 months (participants with a history of malignancy who have undergone curative resection or otherwise not requiring treatment for at least 12 months prior to Screening with no detectable recurrence are allowed).
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  • Participant has a lifetime history of suicide attempt (including an actual attempt, interrupted attempt, or aborted attempt), or has had suicidal ideation with at least some intent to act in the past 6 months as indicated by a positive response ("Yes") to either question 4 or question 5 of the "Screening/Baseline" version of the C-SSRS at Screening.
  • Participant has alanine transaminase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) >2.5x upper limit of normal (ULN)
  • Participant has bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
  • Participant has current unstable liver or biliary disease per Investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis.
  • QTc interval >450msec for male participants, QTc >470msec for female participants, or QTc >480 msec in participants with bundle branch block
  • Participant has had recent surgery requiring general anesthesia within 2 weeks prior to Screening or is expected to have surgery requiring general anesthesia during the 12-week Main Treatment Period
  • Participant has received a treatment with an experimental drug within 30 days or 5 half lives of the experimental drug (whichever is longer) prior to Baseline
  • Participant has received treatment with rituximab within 6 months prior to Baseline or treatment is planned to occur during the study
  • Participant has received treatment with intravenous immunoglobulin G (IVIG), SC immunoglobulin, or plasma exchange PLEX 4 weeks prior to Baseline or participant is on chronic IVIG, SC immunoglobulin, or PLEX
  • Participant has previously participated in this study or participant has previously been assigned to treatment in a study of the medication under investigation in this study
  • Participant has participated in another study of an investigational study medication (and/or an investigational device) within the previous 30 days or is currently participating in another study of an investigational study medication (and/or an investigational device)
  • Participant has known positive serology for muscle-specific kinase

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

United States12 sites

  • Mg0017 50555

    Austin, Texas, 78759

  • Mg0017 50556

    Chapel Hill, North Carolina, 27599

  • Mg0017 50086

    Charlotte, North Carolina, 28207

  • Mg0017 50076

    Columbus, Ohio, 43221

  • Mg0017 50304

    Dallas, Texas, 75390-8866

  • Mg0017 50569

    Greenfield, Wisconsin, 53228

  • Mg0017 50559

    Los Angeles, California, 90095

  • Mg0017 50557

    O'Fallon, Illinois, 62269

  • Mg0017 50593

    Rancho Mirage, California, 92270

  • Mg0017 50099

    San Francisco, California, 94143

  • Mg0017 50564

    Scottsdale, Arizona, 85251

  • Mg0017 50111

    Seattle, Washington, 98195-0001

Other trials in United States

About this study

Official title: A Phase 3b, Multicenter, Open-Label, Single-Arm Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Participants With Generalized Myasthenia Gravis Switching From Intravenous Complement Component 5 Inhibitors to Subcutaneous Zilucoplan

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)

Being tested
zilucoplan (RA101495)
Keywords
gMG
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • zilucoplan (RA101495)Drug

    Subcutaneous injection

Study groups in NCT05514873
Study groupRoleWhat they receive
0.3 mg/kg zilucoplan (RA101495)ExperimentalStudy participants will be treated with subcutaneous zilucoplan (0.3mg/kg/day)
Receives: Drug: zilucoplan (RA101495)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Main Treatment Period

    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).

    Measured: From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)

  2. Percentage of Participants With TEAEs Leading to Withdrawal of Study Medication Over the Main Treatment Period

    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).

    Measured: From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)

Secondary outcomes (4)

  1. Change From Baseline to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score

    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.

    Measured: From Baseline to Week 12

  2. Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Score

    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.

    Measured: From Baseline to Week 12

  3. Percentage of Participants With Serious TEAEs Over the Main Treatment Period

    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

    Measured: From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)

  4. Percentage of Participants With Study Withdrawal Over the Main Treatment Period

    Percentage of participants with study withdrawal based to pre-defined reasons in the protocol were reported.

    Measured: From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Aug 2022
  2. Study start31 Oct 2022
  3. Primary completion13 Mar 2024
  4. Study completion23 Oct 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05514873
Sponsor's protocol ID
MG0017
Status last verified
Aug 2025
First submitted
22 Aug 2022
Last updated
3 Sep 2025
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Publications

  • Freimer M, Desai U, Govindarajan R, Kang MK, Khan S, Khatri B, Levine T, Macwan S, Shieh PB, Weiss MD, Bloemers J, Boroojerdi B, Delicha EM, Lavrov A, Singh P, Howard JF Jr. Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label study. Ther Adv Neurol Disord. 2025 Jul 5;18:17562864251347283. doi: 10.1177/17562864251347283. eCollection 2025.

    PubMed 40620733

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing zilucoplan (RA101495) in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 12 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Main Treatment Period (time frame: From Baseline (Day 1) to Safety Follow-Up Visit (40 days post last dose) of Main Treatment Period (up to approximately 19 weeks)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05514873, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2025.

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