Generalized Myasthenia GravisPhase 3NCT06540144
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
A Phase 3 study testing rozanolixizumab in people with Generalized Myasthenia Gravis, run by UCB Biopharma SRL at 11 sites in 5 countries.
Trial summary
The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006. Read more about this study
- Who is it for?
- Ages 2 years and older
- 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
- 13 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 8 Oct 2024to 5 Oct 2027
- Phase
- Phase 3
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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Study participant must meet one of the following:
- Study participant completed MG0006 according to the protocol
- Study participant completed the MG0006 Treatment Period and has a worsening of generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006
Who cannot join
- Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP
- Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (FcRn) drugs
- Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant's ability to participate in this study
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
About this study
Official title: An Open-label Extension Study to Evaluate Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.
- 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. Each treatment cycle in each Treatment Period (TP) consists of 6 subcutaneous (sc) administrations of rozanolixizumab at 1-week intervals. Each Treatment Period will be initiated upon the discretion of the Investigator based on the medical needs of the study-participant. 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 52 weeks)
Occurrence of TEAEs leading to permanent withdrawal of 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 52 weeks)
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit
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 52 weeks)
Secondary outcomes (4)
Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)
Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP.
Measured: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
Absolute change in total IgG from Baseline to the end of Week 6 of each TP
Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP.
Measured: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP
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 of each TP (up to 52 weeks)
Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP
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 of each TP (up to 52 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2024
- Study start8 Oct 2024
- Primary completion5 Oct 2027 (estimated)
- Study completion5 Oct 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
- 13 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
- NCT06540144
- Sponsor's protocol ID
- MG0008
- Other IDs
- 2022-502075-34, U1111-1286-3581
- Status last verified
- Oct 2026
- First submitted
- 22 Jul 2024
- Last updated
- 16 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.