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

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

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: rozanolixizumab

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: UCB Cares; Phone Number: (USA) 1-844-599-2273; Email: ucbcares@ucb.com
• Study Contact Backup: Name: UCB Cares; Phone Number: 001 844 599 2273; Email: ucbcares@ucb.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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

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 for 6 weeks.	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 18 weeks)
Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (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 18 weeks)
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)	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 18 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6	Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis throughout the study.	From Baseline to the end of Week 6
Absolute change in total IgG from Baseline at the end of Week 6	Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis throughout the study.	From Baseline to the end of Week 6
Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6	Plasma concentration analyses of MG specific anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) autoantibodies will be done for all study participants on an ongoing basis throughout the study.	From Baseline to the end of Week 6
Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6	Plasma concentration analyses of anti-AChR or anti-MuSK autoantibodies will be done for all study participants on an ongoing basis throughout the study.	From Baseline to the end of Week 6
Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6	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
Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6	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
Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1)	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.	During TP1 and OP1 (up to 14 weeks)
Evaluation of local tolerability at each scheduled assessment during TP1	Local tolerability will be evaluated at each scheduled assessment for all study participants during TP1.	At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks)
Plasma concentration of rozanolixizumab at the 6-week treatment cycle	Plasma concentration analyses of rozanolixizumab will be done for all study participants on an ongoing basis throughout the study.	At the 6-week treatment cycle
Incidence of antidrug antibodies (ADAs) at the end of Week 6	Plasma concentration analyses of ADAs will be done for all study participants on an ongoing basis throughout the study.	At the end of Week 6

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Estimated): May 15, 2024
• Primary Completion (Estimated): June 8, 2026
• Study Completion (Estimated): August 17, 2026

Study Registration Dates

• First Submitted: September 25, 2023
• First Submitted That Met QC Criteria: November 28, 2023
• First Posted (Actual): November 29, 2023

Study Record Updates

• Last Update Posted (Actual): April 12, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: pediatric; Generalized Myasthenia Gravis; gMG; rozanolixizumab
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis; Physiological Effects of Drugs; Immunosuppressive Agents; Immunologic Factors; Rozanolixizumab
• Other Study ID Numbers: MG0006; 2022-502074-16-00 (Registry Identifier: CTIS); U1111-1285-0787 (Other Identifier: Universal Trial Number)

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