RECRUITINGPhase 2 / Phase 3INTERVENTIONAL
A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
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
About This Trial
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).
Who May Be Eligible (Plain English)
Who May Qualify:
- Study participant must be ≥2 to \<18 years of age inclusive, at the time of signing the willing to sign a consent form/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 Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
* 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))
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
Treatments Being Tested
DRUG
rozanolixizumab
rozanolixizumab solution for injection
Locations (13)
Mg0006 50574
Flower Mound, Texas, United States
Mg0006 40290
Bologna, Italy
Mg0006 40144
Milan, Italy
Mg0006 40733
Naples, Italy
Mg0006 20340
Fuchu-shi, Japan
Mg0006 20339
Ōbu, Japan
Mg0006 20343
Sagamihara, Japan
Mg0006 40155
Warsaw, Poland
Mg0006 40734
Warsaw, Poland
Mg0006 20081
Taipei, Taiwan
Mg0006 20095
Taipei, Taiwan
Mg0006 40841
Altindağ/ankara, Turkey (Türkiye)
Mg0006 40836
Kocaeli, Turkey (Türkiye)