Generalized Myasthenia GravisPhase 3ziMyG+NCT06435312
An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
A Phase 3 study testing Zilucoplan in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 6 sites in 4 countries.
Trial summary
The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants Read more about this study
- Who is it for?
- Ages 12 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
- 8 participants
- Where
- 6 sitesin 4 countries
- Study dates
- 19 Nov 2024to 19 Nov 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- United States of America (USA) specific inclusion criterion:
- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.
- Rest of World (ROW) specific inclusion criterion:
- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.
- Global specific inclusion criteria:
- Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion
- Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care
Who cannot join
- Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
- Participant has known positive serology for muscle-specific kinase
- Participant has known hypersensitivity to any components of the IMP
- Participant has a prior history of meningococcal disease
Trial locations and contacts
6 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 4 countries
About this study
Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- Zilucoplan
- Keywords
- generalized Myasthenia Gravis, gMG, RA101495, Zilucoplan, pediatric, MG0015
- 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 administered subcutaneously to pediatric study participants
| Study group | Role | What they receive |
|---|---|---|
| Zilucoplan Arm | Experimental | Study participants will receive Zilucoplan at a pre-defined dose based on their weight. Receives: Drug: Zilucoplan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurence of treatment emergent adverse events during the course of the study
An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Measured: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Occurence of treatment-emergent serious adverse events (TESAEs)
A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose:
Results in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.
Measured: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product
An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.
Measured: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Occurence of treatment-emergent infections
Percentage of participants who experienced treatment-emergent infections as adverse events.
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.
Measured: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Secondary outcomes (5)
Plasma concentration of Zilucoplan at Week 52
Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52.
Measured: Week 52
Sheep red blood cell (sRBC) lysis activity at Week 52
Blood samples for measurement of sRBC lysis will be collected at Week 52.
Measured: Week 52
Blood complement component 5 (C5) levels at Week 52
Blood samples for measurement of C5 will be collected at Week 52.
Measured: Week 52
Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52
The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability.
Measured: Week 52
Quantitative Myasthenia Gravis (QMG) score at Week 52
QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2024
- Study start19 Nov 2024
- Primary completion26 Oct 2027 (estimated)
- Study completion19 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by UCB Biopharma SRLThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma SRL
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06435312
- Sponsor's protocol ID
- MG0015
- Other IDs
- 2022-502073-42, U1111-1290-3806
- Acronym
- ziMyG+
- Status last verified
- Sep 2026
- First submitted
- 23 May 2024
- Last updated
- 2 Oct 2026
- Results posted
- No
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT06435312
- Acronym / sponsor ID
- ziMyG+ / MG0015
- Conditions
- Generalized Myasthenia Gravis
- Keywords
- generalized Myasthenia Gravis, gMG, RA101495, Zilucoplan, pediatric, MG0015
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- UCB Biopharma SRLIndustry (pharma, biotech, devices)
- Study start
- 19 Nov 2024 (actual)
- Primary completion
- 26 Oct 2027 (estimated)
- Study completion
- 19 Nov 2027 (estimated)
- First posted
- 30 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Zilucoplan Arm | Experimental | Drug: ZilucoplanStudy participants will receive Zilucoplan at a pre-defined dose based on their weight. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.
- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.
- Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion
- Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care
Key exclusion criteria (4 listed)
- Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
- Participant has known positive serology for muscle-specific kinase
- Participant has known hypersensitivity to any components of the IMP
- Participant has a prior history of meningococcal disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZilucoplanAlso: RA101495Zilucoplan will be administered subcutaneously to pediatric study participants | Drug | Zilucoplan Arm |
Primary outcome
Occurence of treatment emergent adverse events during the course of the study
Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Occurence of treatment-emergent serious adverse events (TESAEs)
Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.
Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product
Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.
Occurence of treatment-emergent infections
Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Percentage of participants who experienced treatment-emergent infections as adverse events. An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.
5 secondary outcomes
Plasma concentration of Zilucoplan at Week 52
Time frame: Week 52
Sheep red blood cell (sRBC) lysis activity at Week 52
Time frame: Week 52
Blood complement component 5 (C5) levels at Week 52
Time frame: Week 52
Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52
Time frame: Week 52
Quantitative Myasthenia Gravis (QMG) score at Week 52
Time frame: Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
UCB Cares study director
001 844 599 2273 (UCB)
6 sites in 4 countries: see every site and its status.
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 6 sites in 4 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 7 inclusion and 4 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: enrolling by invitation, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Poland, United Kingdom, Italy, South Korea. 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?
This study is still under way. Results are not available in the source record yet.
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?
Occurence of treatment emergent adverse events during the course of the study (time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Oct 2026.