MG0006
MG0006
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
Brief summary
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).
Medical Condition
Generalized Myasthenia Gravis
Min. Age
12
Years
Max. Age
17
Years
Who Can Join?
All
Status
Not yet recruiting
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
Study Medication Description
Study Medication:
RYSTIGGO
Other Descriptive Name:
rozanolixizumab
Placebo
No
Comparator:
No
Refer to a friend
Study Dates
March 2024
Planned Start Date of Enrollment
August 2026
Planned Study Completion Date