A Phase 3 Open-label Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
NCT Number: NCT06540144
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE3
Conditions: Generalized Myasthenia Gravis
Interventions: DRUG: rozanolixizumab
Sponsor: UCB Biopharma SRL
Sex: ALL
Age: CHILD, ADULT, OLDER_ADULT
Enrollment: 12
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-10-01
Completion Date: 2027-12-31

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 ≥12 at the time of the Screening Visit of MG0006.

Primary Outcome Measures: 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, 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., 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., From Baseline up to the EOS Visit (up to 52 weeks)
Secondary Outcome Measures: 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., 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., 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., 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., From Baseline to the end of Week 6 of each TP (up to 52 weeks)