A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis

Study Overview
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Brief Summary
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The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥12 to <18 years with generalized Myasthenia Gravis (gMG).

Official Title
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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

Conditions
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Generalized Myasthenia Gravis

Intervention / Treatment
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* Drug: rozanolixizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: US specific: - Study participant must be ≥12 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation Rest of world: - Study participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation Global: 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)) Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rozanolixizumab<br>Study participants will receive pre-defined doses of rozanolixizumab for 6 weeks. | Drug: rozanolixizumab<br>* rozanolixizumab solution for injection<br>* Other names: UCB7665;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 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. | 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. | From Baseline up to the EOS Visit (up to 18 weeks) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | At the end of Week 6 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Generalized Myasthenia Gravis, gMG, rozanolixizumab, pediatric