A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

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

The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).

Study Overview

• Status: Completed
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: Rozanolixizumab

• Study Type: Interventional
• Enrollment (Actual): 165
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Edmonton, Canada
    • Mg0007 50071
  • Montreal, Canada
    • Mg0007 50066
  • Montreal, Canada
    • Mg0007 50124
  • Quebec, Canada
    • Mg0007 50070
  • Toronto, Canada
    • Mg0007 50069
• Czechia
  • Ostrava - Poruba, Czechia
    • Mg0007 40125
  • Praha 2, Czechia
    • Mg0007 40124
• Denmark
  • Aalborg, Denmark
    • Mg0007 40128
  • Aarhus, Denmark
    • Mg0007 40127
  • Copenhagen, Denmark
    • Mg0007 40126
• France
  • Bordeaux, France
    • Mg0007 40129
  • Limoges, France
    • Mg0007 40360
  • Nice Cedex 1, France
    • Mg0007 40132
  • Paris, France
    • Mg0007 40133
  • Strasbourg, France
    • Mg0007 40131
• Georgia
  • Tbilisi, Georgia
    • Mg0007 20160
  • Tbilisi, Georgia
    • Mg0007 20161
  • Tbilisi, Georgia
    • Mg0007 20163
  • Tbilisi, Georgia
    • Mg0007 20164
  • Tbilisi, Georgia
    • Mg0007 20165
• Germany
  • Essen, Germany
    • Mg0007 40134
  • Göttingen, Germany
    • Mg0007 40140
  • Jena, Germany
    • Mg0007 40139
  • Leipzig, Germany
    • Mg0007 40078
  • Münster, Germany
    • Mg0007 40177
• Italy
  • Bologna, Italy
    • Mg0007 40283
  • Milano, Italy
    • Mg0007 40144
  • Napoli, Italy
    • Mg0007 40307
  • Pavia, Italy
    • Mg0007 40146
  • Roma, Italy
    • Mg0007 40148
  • Roma, Italy
    • Mg0007 40150
• Japan
  • Bunkyo-ku, Japan
    • Mg0007 20035
  • Chiba-shi, Japan
    • Mg0007 20068
  • Hanamaki-shi, Japan
    • Mg0007 20078
  • Hiroshima, Japan
    • Mg0007 20079
  • Kobe, Japan
    • Mg0007 20075
  • Nagasaki-shi, Japan
    • Mg0007 20071
  • Sendai, Japan
    • Mg0007 20077
  • Shinjuku-ku, Japan
    • Mg0007 20070
  • Shinjuku-ku, Japan
    • Mg0007 20076
  • Suita, Japan
    • Mg0007 20032
• Poland
  • Gdansk, Poland
    • Mg0007 40155
  • Lodz, Poland
    • Mg0007 40154
  • Lublin, Poland
    • Mg0007 40151
  • Poznan, Poland
    • Mg0007 40153
• Russian Federation
  • Novosibirsk, Russian Federation
    • Mg0007 20169
  • Saint-petersburg, Russian Federation
    • Mg0007 20001
  • Saint-petersburg, Russian Federation
    • Mg0007 20028
  • Saint-petersburg, Russian Federation
    • Mg0007 20055
• Serbia
  • NIS, Serbia
    • Mg0007 40467
• Spain
  • Barcelona, Spain
    • Mg0007 40160
  • Hospitalet de Llobregat, Spain
    • Mg0007 40157
  • Murcia, Spain
    • Mg0007 40350
  • San Sebastián de Los Reyes, Spain
    • Mg0007 40308
• Taiwan
  • Taipei City, Taiwan
    • Mg0007 20086
  • Taipei City, Taiwan
    • Mg0007 20081
• United States
  • California
    • Orange, California, United States, 92868
      • Mg0007 50092
    • San Francisco, California, United States, 94117
      • Mg0007 50099
  • Florida
    • Miami, Florida, United States, 33136
      • Mg0007 50122
    • Miami, Florida, United States, 33144
      • Mg0007 50120
    • Tampa, Florida, United States, 33612
      • Mg0007 50073
  • Georgia
    • Augusta, Georgia, United States, 30912-0004
      • Mg0007 50075
  • Hawaii
    • Honolulu, Hawaii, United States, 96817
      • Mg0007 50323
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Mg0007 50114
  • Kentucky
    • Lexington, Kentucky, United States, 40536-0284
      • Mg0007 50121
  • New York
    • New York, New York, United States, 10021
      • Mg0007 50077
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157
      • Mg0007 50090
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Mg0007 50096
  • Texas
    • Houston, Texas, United States, 77030
      • Mg0007 50113

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Study participant must meet one of the following:

  1. completed MG0003 [NCT03971422]
  2. required rescue therapy during the Observation Period in MG0003 or
  3. completed at least 6 visits in MG0004 [NCT04124965]
• Body weight ≥35 kg at Baseline (Day 1)
• Study participants may be male or female

Exclusion Criteria:

• Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications
• Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
• Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, or MG0004, or permanently discontinued study drug in either study
• Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab
• Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Rozanolixizumab dosage regimen 1; Study participants randomized/assigned to dosage regimen 1 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.	Drug: Rozanolixizumab; Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.; Other Names: UCB7665
Experimental: Rozanolixizumab dosage regimen 2; Study participants randomized/assigned to dosage regimen 2 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.	Drug: Rozanolixizumab; Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.; Other Names: UCB7665

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which did not necessarily had 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. TEAE was defined as any event that was not present prior to first dose of investigational medicinal product (IMP), or any unresolved event already present that worsened in intensity following treatment, up to 8 weeks after end of Treatment Period or after last dose of IMP in participants who discontinued study or IMP.	From Baseline (Day 1) to End of Study (up to 34 months)
Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP)	An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.	From Baseline (Day 1) to End of Study (up to 34 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3)	The MG-ADL is an 8-item PRO instrument developed on basis of QMG. The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0= no symptoms or impaired performance and 3= most severe symptoms or impaired performance. The total score ranges from 0 to 24, with higher score indicating more disability. A positive change in score indicates worsening and negative change indicates improvement. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline [Day 1]) value for that cycle.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
Change From Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) Score Within One Treatment Cycle (Cycle 1, 2, and 3)	The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change in the score indicates worsening and a negative change indicates improvement. For the analyses done by study cycle, Baseline values were the last available values prior to or on the same date (and same time if time was collected for the individual assessment) of first administration of IMP at each cycle (ie, Baseline [Day 1]) value for that cycle.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) Score Within One Treatment Cycle (Cycle 1, 2, and 3)	MG-C scale consists of 10 items which included ptosis (score range=0 to 3), double vision on lateral gaze left/right/both (score range=0 to 4), eye closure (score range=0 to 2), talking (score range=0 to 6), chewing (score range=0 to 6), swallowing (score range=0 to 6), breathing (score range=0 to 9), neck flexion or extension (score range=0 to 4), shoulder abduction (score range=0 to 5) and hip flexion (score range= 0 to 5), lower scores= lower disease activity. Total MG-C score obtained by summing responses to each individual item and score ranges from 0 - 50, (lower scores indicating lower disease activity). Positive change = worsening, and negative change = improvement. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline [Day 1]) value for that cycle.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score Within One Treatment Cycle (Cycle 1, 2, and 3)	The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular symptoms (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option that best described severity of ocular, bulbar, and respiratory symptoms over past 7 days using a 4-point Likert scale ("none" to "severe") and frequency of experiencing physical fatigue and muscle weakness fatigability over past 7 days using a 5-point Likert scale (1="none of the time" - 5= "all of the time"), for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicates more severe symptoms. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline [Day 1]) value for that cycle.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' Score Within One Treatment Cycle (Cycle 1, 2, and 3)	The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option based on frequency of experiencing physical fatigue (19-33) over past 7 days using a 5-point Likert scale (1="none of time" - 5="all of time") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicated severe symptoms.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' Score Within One Treatment Cycle (Cycle 1, 2, and 3)	The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Bulbar symptoms are now recognised as bulbar muscle weakness. Study participants were asked to choose response option that best described severity of bulbar muscle weakness (6-15) symptoms over past 7 days using a 4-point Likert scale (1="none" to 4="severe") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicated severe symptoms.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
MG-ADL Responder Rate (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3 [Day 43])	The MG-ADL is an 8-item PRO instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A MG-ADL responder was defined as achieving at least 2.0-point improvement (decrease) in the MG-ADL score from Baseline.	Day 43 of Cycle 1, 2, and 3
Time to MG-ADL Response (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3)	Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline. Time to first MG-ADL response (in days) by study cycle was defined as date of first MG-ADL Response within study cycle - date of MG-ADL Baseline within study cycle + 1.	From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
Time Between Consecutive Treatment Cycles	Time between consecutive treatment cycles: Study participants were assessed for MG worsening prior to repeated cycles. In case of symptom worsening (eg, an increase of 2.0 points on MG-ADL or 3.0 points on QMG scale) between assessments, resulted additional treatment, study participants undergone another 6-week treatment cycle followed by an Observation Period, based on Investigator's discretion. Time between treatment cycles was calculated as: date of first sc infusion in consecutive cycle - date of last sc infusion before new cycle + 1 (or date of censoring - date of last sc infusion before potential new cycle + 1).	From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years

Collaborators and Investigators

• Sponsor: UCB Biopharma SRL
• Investigators: Study Director: UCB Cares, 001 844 599 22733 (UCB)

Study record dates

Study Major Dates

• Study Start (Actual): February 3, 2021
• Primary Completion (Actual): January 25, 2024
• Study Completion (Actual): January 25, 2024

Study Registration Dates

• First Submitted: November 5, 2020
• First Submitted That Met QC Criteria: November 24, 2020
• First Posted (Actual): December 3, 2020

Study Record Updates

• Last Update Posted (Actual): April 18, 2025
• Last Update Submitted That Met QC Criteria: April 17, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: gMG; rozanolixizumab; UCB7665; generalized myasthenia gravis
• Additional Relevant MeSH Terms: Neurologic Manifestations; Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Manifestations; Pathologic Processes; 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; Muscle Weakness; Myasthenia Gravis; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Rozanolixizumab
• Other Study ID Numbers: MG0007; 2020-003230-20 (EudraCT 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 study 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.: Yes