Generalized Myasthenia GravisPhase 2/3ziMyGNCT06055959
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
A Phase 2/3 study testing Zilucoplan in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 9 sites in 5 countries.
Trial summary
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG). Read more about this study
- Who is it for?
- Ages 12 to 17 years
- 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
- 9 sitesin 5 countries
- Study dates
- 16 Oct 2024to 22 Jun 2027
- Phase
- Phase 2/3
- 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 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- United States of America (USA) specific inclusion criterion:
- Participant must be 12 to <18 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 to <18 years of age at the time of signing the Informed consent/assent according to local regulation
- Global inclusion criteria:
- Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
Show all 11Show fewer
- Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
- Participants with gMG, including:
- An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to <18 years of age at Screening
- Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to <12 years of age at Screening (does not apply to US)
- Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s)
Who cannot join
- Participant has known positive serology for muscle-specific kinase
- 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 had a thymectomy within 6 months prior to Baseline
- Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
- Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline
Trial locations and contacts
9 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
UCB Cares contact
9 sites in 5 countries
United States2 sites
- Withdrawn
Mg0014 50168
Chicago, Illinois, 60611
- Withdrawn
Mg0014 50574
Denton, Texas, 76208
Poland2 sites, 1 recruiting
- Recruiting
Mg0014 40774
Katowice
- Active, not recruiting
Mg0014 40218
Warsaw
South Korea2 sites, 1 recruiting
- Withdrawn
Mg0014 20104
Seoul
- Recruiting
Mg0014 20220
Seoul
United Kingdom2 sites, 2 recruiting
- Recruiting
Mg0014 40735
Glasgow
- Recruiting
Mg0014 40736
London
About this study
Official title: A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- Zilucoplan
- Keywords
- RA101495, gMG, generalized myasthenia gravis, zilucoplan, pediatric, MG0014
- 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 in 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.
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Measured: Week 4 (Day 29)
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Measured: Week 4 (Day 29)
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
Samples for measurement of C5 will be collected on Day 29 predose.
Measured: Week 4 (Day 29)
Secondary outcomes (9)
Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
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. 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: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence 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 or prolongation of existing hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical events
Measured: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
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. AEs leading to permanent withdrawal of study medication.
Measured: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence 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: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
ADA and anti-PEG antibodies will be evaluated in serum samples.
Measured: Week 4 (Day 29)
Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
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 4 (Day 29)
Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
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 4 (Day 29)
Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
The MGFA-PIS is a physician-determined assessment of clinical symptoms of MG after initiation of MG specific therapy. For the purpose of the current study, Minimal Manifestation will be determined at each scheduled time point after treatment initiation (rather than after 1 year). Change in status (improved, unchanged, worse, exacerbation, or died of MG) will also be determined.
Measured: Week 4 (Day 29)
Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
The PedsQoL generic core scale (Version 4) is a validated instrument that is suitable for use with pediatric populations. PedsQoL generic core scales assess Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. The scale has 23 items with a score range of 0 to 4. Following transformation, the score range of each domain as well as the total score is 0-100 with higher scores indicating higher HRQoL.
Measured: Week 4 (Day 29)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start16 Oct 2024
- Primary completion12 May 2027 (estimated)
- Study completion22 Jun 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 2/3
- 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06055959
- Sponsor's protocol ID
- MG0014
- Other IDs
- U1111-1290-3349, 2022-502072-23
- Acronym
- ziMyG
- Status last verified
- Nov 2026
- First submitted
- 20 Sep 2023
- Last updated
- 5 Nov 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 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT06055959
- Acronym / sponsor ID
- ziMyG / MG0014
- Conditions
- Generalized Myasthenia Gravis
- Keywords
- RA101495, gMG, generalized myasthenia gravis, zilucoplan, pediatric, MG0014
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- UCB Biopharma SRLIndustry (pharma, biotech, devices)
- Study start
- 16 Oct 2024 (actual)
- Primary completion
- 12 May 2027 (estimated)
- Study completion
- 22 Jun 2027 (estimated)
- First posted
- 28 Sep 2023 (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 pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
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 in pre-defined dose based on their weight. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Participant must be 12 to <18 years of age at the time of signing the Informed consent/assent according to local regulation
- Participant must be 2 to <18 years of age at the time of signing the Informed consent/assent according to local regulation
- Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
- Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
- Participants with gMG, including:
Key exclusion criteria (5 listed)
- Participant has known positive serology for muscle-specific kinase
- 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 had a thymectomy within 6 months prior to Baseline
- Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
- Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline
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
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Time frame: Week 4 (Day 29)
Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Time frame: Week 4 (Day 29)
Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
Time frame: Week 4 (Day 29)
Samples for measurement of C5 will be collected on Day 29 predose.
9 secondary outcomes
Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of treatment-emergent serious adverse events (TESAEs)
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of treatment-emergent infections
Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
Time frame: Week 4 (Day 29)
Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
Time frame: Week 4 (Day 29)
Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
Time frame: Week 4 (Day 29)
Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
Time frame: Week 4 (Day 29)
Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
Time frame: Week 4 (Day 29)
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 5 Nov 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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A Clinical Study to Evaluate the Safety and Efficacy of GT802 Injection in the Treatment of Refractory Generalized Myasthenia Gravis
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ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis
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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 contact
UCB Cares study director
001 844 599 2273
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Zilucoplan in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 9 sites in 5 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 11 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Poland, South Korea, United Kingdom, Italy. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact UCB Cares (+18445992273 (USA), ucbcares@ucb.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) (time frame: Week 4 (Day 29)). The study lists 3 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 5 Nov 2026.