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

Last updated: January 30, 2025
Sponsor: UCB Biopharma SRL
Overall Status: Active - Recruiting

Phase

2/3

Condition

Myasthenia Gravis Generalised

Sarcopenia

Polymyositis (Inflammatory Muscle Disease)

Treatment

rozanolixizumab

Clinical Study ID

NCT06149559
MG0006
U1111-1285-0787
2022-502074-16-00
  • Ages 2-17
  • All Genders

Study Summary

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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Study participant must be ≥2 to <18 years of age inclusive, at the time of signingthe 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 specificautoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK)prior to Screening

  • Study participant has Myasthenia Gravis Foundation of America (MGFA) ClinicalClassification 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 gMGsymptoms and is in need of additional therapy (for example, plasma exchange (PEX) ortreatment with intravenous immunoglobulin (IVIg))

Exclusion

Exclusion Criteria:

  • Study participant with severe weakness affecting oropharyngeal or respiratorymuscles, or who has myasthenic crisis or impending crisis at Screening or Baseline

  • Study participant has a known hypersensitivity to any components of theInvestigational 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 6weeks prior to the first dose of IMP

  • Study participant has received a live vaccination within 4 weeks prior to Baselineor intends to have a live vaccination during the course of the study

Study Design

Total Participants: 12
Treatment Group(s): 1
Primary Treatment: rozanolixizumab
Phase: 2/3
Study Start date:
June 14, 2024
Estimated Completion Date:
August 17, 2026

Connect with a study center

  • Mg0006 40290

    Bologna,
    Italy

    Active - Recruiting

  • Mg0006 40144

    Milano,
    Italy

    Active - Recruiting

  • Mg0006 40734

    Lodz,
    Poland

    Active - Recruiting

  • Mg0006 40155

    Warszawa,
    Poland

    Active - Recruiting

  • Mg0006 20081

    Taipei City,
    Taiwan

    Active - Recruiting

  • Mg0006 20095

    Taipei City,
    Taiwan

    Active - Recruiting

  • Mg0006 50574

    Denton, Texas 76208
    United States

    Site Not Available

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