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Enrolling by Invitation

NCT Number: NCT06540144

An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis

The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.

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Key information

Conditions

Generalized Myasthenia Gravis Autoimmune Diseases Autoimmune Diseases of the Nervous System Immune System Diseases Myasthenia Gravis Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodegenerative Diseases Neuromuscular Diseases Neuromuscular Junction Diseases Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Mg0008 40290, Bologna, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Study participant must meet one of the following:

  • Study participant completed MG0006 according to the protocol
  • Study participant completed the MG0006 Treatment Period and has a worsening of generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006

Exclusion criteria

  • Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP
  • Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (FcRn) drugs
  • Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant's ability to participate in this study

Treatment and study plan

rozanolixizumab

Drug

rozanolixizumab solution for injection

Other names: UCB7665

Primary outcomes

  1. Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit

    Time frame: From Baseline up to the EOS Visit (up to 52 weeks)

    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

  2. Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit

    Time frame: From Baseline up to the EOS Visit (up to 52 weeks)

    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.

  3. Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit

    Time frame: From Baseline up to the EOS Visit (up to 52 weeks)

    AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.

Secondary outcomes

  1. Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)

    Time frame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

    Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP.

  2. Absolute change in total IgG from Baseline to the end of Week 6 of each TP

    Time frame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

    Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP.

  3. Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP

    Time frame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

    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.

  4. Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP

    Time frame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

    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.

Sponsors and collaborators

Lead sponsor

UCB Biopharma SRL

Industry

Registry information

Official study title

An Open-label Extension Study to Evaluate Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Aug 6, 2024
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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