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

An Open-label Extension Study to Evaluate 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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: rozanolixizumab

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 3

Contacts and Locations

Study Locations

• Italy
  • Bologna, Italy
    • Mg0008 40290
  • Milan, Italy
    • Mg0008 40144
  • Naples, Italy
    • Mg0008 40733
• Japan
  • Fuchu-shi, Japan
    • Mg0008 20340
  • Sagamihara, Japan
    • Mg0008 20343
  • Ōbu, Japan
    • Mg0008 20339
• Poland
  • Warsaw, Poland
    • Mg0008 40155
  • Warsaw, Poland
    • Mg0008 40734
• Taiwan
  • Taipei, Taiwan
    • Mg0008 20081
  • Taipei, Taiwan
    • Mg0008 20095

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: rozanolixizumab; Study participants will receive pre-defined doses of rozanolixizumab. Each treatment cycle in each Treatment Period (TP) consists of 6 subcutaneous (sc) administrations of rozanolixizumab at 1-week intervals. Each Treatment Period will be initiated upon the discretion of the Investigator based on the medical needs of the study-participant.	Drug: rozanolixizumab; rozanolixizumab solution for injection; Other Names: UCB7665

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit	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	From Baseline up to the EOS Visit (up to 52 weeks)
Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit	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.	From Baseline up to the EOS Visit (up to 52 weeks)
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit	AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.	From Baseline up to the EOS Visit (up to 52 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)	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.	From Baseline to the end of Week 6 of each TP (up to 52 weeks)
Absolute change in total IgG from Baseline to the end of Week 6 of each TP	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.	From Baseline to the end of Week 6 of each TP (up to 52 weeks)
Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP	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.	From Baseline to the end of Week 6 of each TP (up to 52 weeks)
Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP	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.	From Baseline to the end of Week 6 of each TP (up to 52 weeks)

Collaborators and Investigators

• Sponsor: UCB Biopharma SRL
• Investigators: Study Director: UCB Cares, 001 844 599 2273

Study record dates

Study Major Dates

• Study Start (Actual): October 8, 2024
• Primary Completion (Estimated): August 17, 2027
• Study Completion (Estimated): August 17, 2027

Study Registration Dates

• First Submitted: July 22, 2024
• First Submitted That Met QC Criteria: August 1, 2024
• First Posted (Actual): August 6, 2024

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 7, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: pediatric; Generalized Myasthenia Gravis; gMG; rozanolixizumab
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; rozanolixizumab
• Other Study ID Numbers: MG0008; U1111-1286-3581 (Other Identifier: WHO universal trial number (UTN)); 2022-502075-34 (Registry Identifier: EU CTIS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• IPD Sharing Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• IPD Sharing Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No