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Generalized Myasthenia GravisPhase 3NCT06471361

A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis

A Phase 3 study testing Zilucoplan in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 11 sites in 3 countries.

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 this study is to evaluate the effectiveness, safety and tolerability of zilucoplan auto-injector (ZLP-AI) self-administration. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Generalized Myasthenia Gravis
What is being tested?
Zilucoplan
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
31 participants
Where
11 sitesin 3 countries
Study dates
27 Aug 2024to 3 Feb 2025
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Study participant is male or female and must be at least 18 years of age at the time of signing the informed consent form (ICF).
  • Study participant must have a documented diagnosis of gMG, based on study participant's history and supported by previous evaluations.
  • Study participant is currently participating in ZLP (zilucoplan) study RA101495-02.302 (NCT04225871) or is administering commercial ZLP on a stable dosing regimen for at least 1 month prior to Screening.
  • Study participants on commercial ZLP need to receive ZLP per the approved local labeling.
  • Study participant is considered reliable and capable of adhering to the study protocol (eg, able to understand and complete questionnaires and able to adhere to the visit schedule) according to the judgement of the Investigator.
  • Study participant is willing and capable of self-administering ZLP using the zilucoplan-auto-injector (ZLP AI) according to the instructions for use (IFU), ie, does not have any visual, physical, or other disability or impairment that interferes with his/her capacity to self-administer; if the participant has a caregiver, he/she may assist the participant with the injection.
Show all 13Show fewer
  • Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine at least 14 days prior to investigational medicinal product (IMP) administration, if not vaccinated within 3 years prior to the start of treatment. Booster vaccination(s) should also be administered as clinically indicated, according to the local standard of care, for participants who have been previously vaccinated against Neisseria meningitidis.
  • Female participants of childbearing potential must have a negative urine pregnancy test prior to the first dose of study drug.
  • Male and/or female study participants
  • A male participant must agree to use contraception during the Treatment Period and for 40 days 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 1 of the following conditions applies:
  • Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance the Treatment Period and for 40 days after the last dose of study medication.
  • Capable of giving signed informed which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.

Who cannot join

  • Study participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
  • Female participants who are breastfeeding, pregnant, or plan to become pregnant during the study.
  • Study participant has a known hypersensitivity to any components of the study medication (and/or an investigational device) as stated in this protocol.
  • Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring intravenous antibiotic treatment) within 6 weeks before Visit 1.
  • Study participant has a history of meningococcal disease.
  • 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 (except studies RA101495-02.201 (NCT03315130), RA101495-02.301 (NCT04115293), or RA101495-02.302 (NCT04225871), which are not excluded, unless the participant was required to withdraw from said studies for a safety reason which could reasonably recur).
  • Participant has participated in another study of an IMP (and/or an investigational device) different from ZLP within the previous 3 months or 5 half-lives, whichever is longer, or is currently participating in another study of an IMP (and/or an investigational device).
  • Current unstable liver or biliary disease at Screening (Visit 1), per Investigator assessment, defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: with exception of stable hepatobiliary conditions (including Gilbert's syndrome, asymptomatic gallstones).

Trial locations and contacts

11 sites in 3 countries.

11 sites in 3 countries

United States7 sites

  • Dv0013 50555

    Austin, Texas, 78759

  • Dv0013 50556

    Chapel Hill, North Carolina, 27599

  • Dv0013 50648

    Columbia, Missouri, 64212

  • Dv0013 50635

    Columbus, Ohio, 43221

  • Dv0013 50636

    Greenfield, Wisconsin, 53228

  • Dv0013 50628

    New Haven, Connecticut, 06511

  • Dv0013 50634

    Tampa, Florida, 33612

Other trials in United States

Poland3 sites

  • Dv0013 40609

    Katowice

  • Dv0013 40759

    Krakow

  • Dv0013 40605

    Poznan

Other trials in Poland

United Kingdom1 site

  • Dv0013 40760

    Oxford

Other trials in United Kingdom

About this study

Official title: A Multicenter, Open-Label, Outpatient Study to Evaluate the Safe And Effective Use of a Zilucoplan Auto-Injector Combination Product for Subcutaneous Self-Administration by Study Participants With Generalized Myasthenia Gravis

The purpose of this study is to evaluate the effectiveness, safety and tolerability of zilucoplan auto-injector (ZLP-AI) self-administration.

Being tested
Zilucoplan
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.

  • ZilucoplanDrug

    Also called RA101495

    Zilucoplan will be self-administered subcutaneously by study participants at pre-specified time points.

Study groups in NCT06471361
Study groupRoleWhat they receive
Zilucoplan-auto-injector (ZLP-AI)ExperimentalStudy participants will self-administer zilucoplan (ZLP) based on their body weight using auto-injector (AI).
Receives: Drug: Zilucoplan

What is being measured

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

  1. Percentage of Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8

    Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of investigational medicinal product (IMP) was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the Safety Set (SS). Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.

    Measured: From Visit 1 (Day 1) to Visit 8 (Day 14)

Secondary outcomes (6)

  1. Percentage of Effective Self-administration of Zilucoplan Using ZLP-AI at Visit 1

    Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of IMP was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the SS. Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.

    Measured: Visit 1 (Day 1)

  2. Percentage of Effective Self-administrations of Zilucoplan Using ZLP-AI at Visit 8

    Effective SA of ZLP was defined as completeness of the delivery as confirmed by the Investigator. The entire dose of IMP was completely delivered (ie, the yellow plunger that was seen through the device window was completely depressed). The percentage of effective SA overall was summarized based on the number of ZLP-AIs used and returned, within participants in the SS. Complete Dose Delivery = Total number (no.) of ZLP-AIs with complete dose delivery/Total no. of ZLP-AIs used and returned.

    Measured: Visit 8 (Day 14)

  3. Percentage of Participants With Serious Adverse Events (SAEs) During the Course of the Study

    An SAE was defined as any untoward medical occurrence that, at any dose, met 1 or more of the criteria listed: Led to death; Led to a life-threatening illness or injury; Led to a permanent impairment of a body structure or a body function; Led to a inpatient or prolonged hospitalization; Led to a medical or surgical intervention to prevent life-threatening illness or injury or permanent impairment to a body structure or a body function; Led to fetal distress, fetal death, or a congenital abnormality or birth defect.

    Measured: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)

  4. Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Course of the Study

    An AE was defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical device. A TEAE was defined as an AE starting on or after the date/time of the first self-injection of ZLP-AI and up to and including 40 days after the final self-injection of ZLP-AI (or last contact depending on which occurs first).

    Measured: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)

  5. Percentage of Participants With Non-serious Adverse Device Effects (ADE) During the Course of the Study

    An ADE was defined as an AE related to the use of an investigational medical device included any AEs resulting from insufficient or inadequate instructions for use, deployment, implantation, installation, or operation, or any malfunction of the investigational medical device as well as any event resulting from use error or from intentional misuse of the investigational medical device.

    Measured: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)

  6. Percentage of Participants With Serious Adverse Device Effects (SADE) During the Course of the Study

    An SAE was defined as any untoward medical occurrence that, at any dose, met 1 or more of the criteria listed: Led to death; Led to a life-threatening illness or injury; Led to a permanent impairment of a body structure or a body function; Led to a inpatient or prolonged hospitalization; Led to a medical or surgical intervention to prevent life-threatening illness or injury or permanent impairment to a body structure or a body function; Led to fetal distress, fetal death, or a congenital abnormality or birth defect. A SADE was defined as an adverse device effect that had resulted in any of the consequences characteristic of a SAE.

    Measured: From Visit 1 (Day 1) up to 40 days after last administration of self-injection of ZLP-AI (up to 54 days after Visit 1)

Timeline

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

  1. First posted24 Jun 2024
  2. Study start27 Aug 2024
  3. Primary completion28 Jan 2025
  4. Study completion3 Feb 2025

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
31 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06471361
Sponsor's protocol ID
DV0013
Other IDs
U1111-1302-4369, 2023-508287-30, 2023
Status last verified
Mar 2026
First submitted
18 Jun 2024
Last updated
27 Mar 2026
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.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Zilucoplan in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 11 sites in 3 countries.

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 13 inclusion and 8 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 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States, Poland, United Kingdom. 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 27 Mar 2026). 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 Effective Self-administrations (SA) of Zilucoplan (ZLP) Using the Zilucoplan-auto-injector (ZLP-AI) From Visit 1 to Visit 8 (time frame: From Visit 1 (Day 1) to Visit 8 (Day 14)). 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 NCT06471361.

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 27 Mar 2026.

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