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

Launched by UCB BIOPHARMA SRL · Nov 28, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called rozanolixizumab for children and teenagers aged 2 to 17 who have moderate to severe generalized Myasthenia Gravis (gMG). Myasthenia Gravis is a condition that causes weakness in the muscles, and the trial is looking to see how safe and tolerable this treatment is when given as an injection under the skin. To be eligible for the study, participants must have a confirmed diagnosis of gMG and have already tried other treatments without satisfactory results.

Participants in the trial will receive the study treatment and will be closely monitored for any side effects or reactions. It's important to note that the trial is currently recruiting participants, but there are some health conditions that would prevent someone from joining, such as severe muscle weakness or certain infections. Overall, this study aims to find a new therapy that could help improve the lives of young people living with Myasthenia Gravis.

Gender

ALL

Eligibility criteria

• 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