Generalized Myasthenia GravisPhase 2/3roMyGNCT06149559
A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
A Phase 2/3 study testing rozanolixizumab in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 9 sites in 4 countries.
Trial summary
The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG). Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- rozanolixizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 14 Jun 2024to 5 Oct 2026
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Study participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation
- Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening
- Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening
- Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening
- Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin (IVIg))
Who cannot join
- Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline
- Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-neonatal-Fc receptor (FcRn) medications
- Study participant with any active or untreated thymoma
- Study participant has a history of thymectomy within 6 months prior to Screening
- Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP
- Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
About this study
Official title: An Open-label, Single-arm Study Evaluating the Activity, Safety, and Pharmacokinetics of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG).
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- rozanolixizumab
- Keywords
- Generalized Myasthenia Gravis, gMG, rozanolixizumab, pediatric
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rozanolixizumabDrug
Also called UCB7665
rozanolixizumab solution for injection
| Study group | Role | What they receive |
|---|---|---|
| rozanolixizumab | Experimental | Study participants will receive pre-defined doses of rozanolixizumab for 6 weeks. Receives: Drug: rozanolixizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose:
- Results in death
- Is life-threatening
- Requires in patient hospitalisation or prolongation of existing hospitalisation
- Results in persistent disability/incapacity
- Is a congenital anomaly or birth defect
- Important medical events
Measured: From Baseline up to the EOS Visit (up to 18 weeks)
Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit
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 up to the EOS Visit (up to 18 weeks)
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)
AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.
Measured: From Baseline up to the EOS Visit (up to 18 weeks)
Secondary outcomes (10)
Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6
Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis throughout the study.
Measured: From Baseline to the end of Week 6
Absolute change in total IgG from Baseline at the end of Week 6
Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis throughout the study.
Measured: From Baseline to the end of Week 6
Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6
Plasma concentration analyses of MG specific anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) autoantibodies will be done for all study participants on an ongoing basis throughout the study.
Measured: From Baseline to the end of Week 6
Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6
Plasma concentration analyses of anti-AChR or anti-MuSK autoantibodies will be done for all study participants on an ongoing basis throughout the study.
Measured: From Baseline to the end of Week 6
Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6
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: From Baseline to the end of Week 6
Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6
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: From Baseline to the end of Week 6
Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1)
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: During TP1 and OP1 (up to 14 weeks)
Evaluation of local tolerability at each scheduled assessment during TP1
Local tolerability will be evaluated at each scheduled assessment for all study participants during TP1.
Measured: At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks)
Plasma concentration of rozanolixizumab at the 6-week treatment cycle
Plasma concentration analyses of rozanolixizumab will be done for all study participants on an ongoing basis throughout the study.
Measured: At the 6-week treatment cycle
Incidence of antidrug antibodies (ADAs) at the end of Week 6
Plasma concentration analyses of ADAs will be done for all study participants on an ongoing basis throughout the study.
Measured: At the end of Week 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2023
- Study start14 Jun 2024
- Primary completion5 Oct 2026
- Study completion5 Oct 2026
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
- 14 participants (actual)
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
- NCT06149559
- Sponsor's protocol ID
- MG0006
- Other IDs
- 2022-502074-16-00, U1111-1285-0787
- Acronym
- roMyG
- Status last verified
- Oct 2026
- First submitted
- 25 Sep 2023
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm Study Evaluating the Activity, Safety, and Pharmacokinetics of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT06149559
- Acronym / sponsor ID
- roMyG / MG0006
- Conditions
- Generalized Myasthenia Gravis
- Keywords
- Generalized Myasthenia Gravis, gMG, rozanolixizumab, pediatric
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- UCB Biopharma SRLIndustry (pharma, biotech, devices)
- Study start
- 14 Jun 2024 (actual)
- Primary completion
- 5 Oct 2026 (actual)
- Study completion
- 5 Oct 2026 (actual)
- First posted
- 29 Nov 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 the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years 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 |
|---|---|---|
| rozanolixizumab | Experimental | Drug: rozanolixizumabStudy participants will receive pre-defined doses of rozanolixizumab for 6 weeks. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Study participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation
- Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening
- Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening
- Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening
- Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin (IVIg))
Key exclusion criteria (6 listed)
- Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline
- Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-neonatal-Fc receptor (FcRn) medications
- Study participant with any active or untreated thymoma
- Study participant has a history of thymectomy within 6 months prior to Screening
- Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP
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 |
|---|---|---|
| rozanolixizumabAlso: UCB7665rozanolixizumab solution for injection | Drug | rozanolixizumab |
Primary outcome
Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
Time frame: From Baseline up to the EOS Visit (up to 18 weeks)
Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in patient hospitalisation or prolongation of existing hospitalisation * Results in persistent disability/incapacity * Is a congenital anomaly or birth defect * Important medical events
Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit
Time frame: From Baseline up to the EOS Visit (up to 18 weeks)
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.
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)
Time frame: From Baseline up to the EOS Visit (up to 18 weeks)
AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.
10 secondary outcomes
Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6
Time frame: From Baseline to the end of Week 6
Absolute change in total IgG from Baseline at the end of Week 6
Time frame: From Baseline to the end of Week 6
Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6
Time frame: From Baseline to the end of Week 6
Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6
Time frame: From Baseline to the end of Week 6
Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6
Time frame: From Baseline to the end of Week 6
Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6
Time frame: From Baseline to the end of Week 6
Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1)
Time frame: During TP1 and OP1 (up to 14 weeks)
Evaluation of local tolerability at each scheduled assessment during TP1
Time frame: At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks)
Plasma concentration of rozanolixizumab at the 6-week treatment cycle
Time frame: At the 6-week treatment cycle
Incidence of antidrug antibodies (ADAs) at the end of Week 6
Time frame: At the end of Week 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 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.
Related trials for Generalized Myasthenia Gravis
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A Study of Nipocalimab Administered to Adults With 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. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing rozanolixizumab in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 9 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 5 inclusion and 6 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 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 9 sites in Italy, Japan, Poland, Turkey (Türkiye). 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (time frame: From Baseline up to the EOS Visit (up to 18 weeks)). 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 27 Oct 2026.