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NCT04650854

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

Completed Phase 3 Results posted Last updated 18 April 2025
What this trial tests

Phase 3 trial testing Rozanolixizumab in Generalized Myasthenia Gravis in 165 participants. Completed in 25 January 2024.

Timeline
3 February 2021
Primary endpoint
25 January 2024
25 January 2024

Quick facts

Lead sponsorUCB Biopharma SRL
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment165
Start date3 February 2021
Primary completion25 January 2024
Estimated completion25 January 2024
Sites69 locations across Georgia, Denmark, France, Italy, Japan, Russia, Serbia, Taiwan

Drugs / interventions tested

Conditions studied

Sponsor

UCB Biopharma SRL — full company profile →

Who can join

18 and older, any sex, with Generalized Myasthenia Gravis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Primary · From Baseline (Day 1) to End of Study (up to 34 months)

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 presen

GroupValue95% CI
Rozanolixizumab ~7 mg/kg78.4
Rozanolixizumab ~10 mg/kg94.1
Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP) Primary · From Baseline (Day 1) to End of Study (up to 34 months)

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.

GroupValue95% CI
Rozanolixizumab ~7 mg/kg9.8
Rozanolixizumab ~10 mg/kg16.7
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) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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 w

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-3.7± 3.5
Rozanolixizumab ~10 mg/kg-3.1± 2.9
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-2.9± 3.1
Rozanolixizumab ~10 mg/kg-3.8± 3.9
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-3.3± 2.7
Rozanolixizumab ~10 mg/kg-3.2± 3.3
Change From Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) Score Within One Treatment Cycle (Cycle 1, 2, and 3) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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 sa

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-4.5± 5.0
Rozanolixizumab ~10 mg/kg-4.1± 4.2
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-3.9± 4.1
Rozanolixizumab ~10 mg/kg-4.9± 5.5
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-5.1± 4.8
Rozanolixizumab ~10 mg/kg-4.2± 4.4
Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) Score Within One Treatment Cycle (Cycle 1, 2, and 3) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-7.6± 7.3
Rozanolixizumab ~10 mg/kg-4.7± 5.7
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-5.7± 5.4
Rozanolixizumab ~10 mg/kg-7.5± 7.0
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-6.8± 5.9
Rozanolixizumab ~10 mg/kg-6.1± 7.2
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) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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 i

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-17.6± 21.0
Rozanolixizumab ~10 mg/kg-14.6± 17.2
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-13.2± 19.2
Rozanolixizumab ~10 mg/kg-19.2± 21.6
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-12.8± 14.4
Rozanolixizumab ~10 mg/kg-15.4± 17.7
Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' Score Within One Treatment Cycle (Cycle 1, 2, and 3) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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.

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-16.5± 18.6
Rozanolixizumab ~10 mg/kg-14.8± 18.5
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-13.6± 21.6
Rozanolixizumab ~10 mg/kg-16.0± 18.2
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-15.3± 16.9
Rozanolixizumab ~10 mg/kg-15.0± 18.4
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) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-13.4± 19.8
Rozanolixizumab ~10 mg/kg-11.6± 16.2
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-10.4± 16.2
Rozanolixizumab ~10 mg/kg-15.5± 18.1
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg-14.0± 16.8
Rozanolixizumab ~10 mg/kg-13.0± 17.9
MG-ADL Responder Rate (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3 [Day 43]) Secondary · Day 43 of Cycle 1, 2, and 3

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 improve

Cycle 1: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg74.3
Rozanolixizumab ~10 mg/kg63.6
Cycle 2: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg62.5
Rozanolixizumab ~10 mg/kg67.7
Cycle 3: Treatment (Day 43)
GroupValue95% CI
Rozanolixizumab ~7 mg/kg73.2
Rozanolixizumab ~10 mg/kg67.2
Time to MG-ADL Response (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3) Secondary · From Baseline (Day 1) to Day 43 of each cycle (Cycles 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.

Cycle 1
GroupValue95% CI
Rozanolixizumab ~7 mg/kg9.008.00 – 15.00
Rozanolixizumab ~10 mg/kg22.0015.00 – 36.00
Cycle 2
GroupValue95% CI
Rozanolixizumab ~7 mg/kg15.0010.00 – 17.00
Rozanolixizumab ~10 mg/kg15.009.00 – 22.00
Cycle 3
GroupValue95% CI
Rozanolixizumab ~7 mg/kg15.009.00 – 15.00
Rozanolixizumab ~10 mg/kg15.009.00 – 17.00
Time Between Consecutive Treatment Cycles Secondary · 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

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

Between Cycle 1 and Cycle 2
GroupValue95% CI
Rozanolixizumab ~7 mg/kg64.09 – 883
Rozanolixizumab ~10 mg/kg51.02 – 856
Between Cycle 2 and Cycle 3
GroupValue95% CI
Rozanolixizumab ~7 mg/kg58.01 – 631
Rozanolixizumab ~10 mg/kg50.015 – 575
Between Cycle 3 and Cycle 4
GroupValue95% CI
Rozanolixizumab ~7 mg/kg42.527 – 399
Rozanolixizumab ~10 mg/kg43.05 – 194
Between Cycle 4 and Cycle 5
GroupValue95% CI
Rozanolixizumab ~7 mg/kg43.09 – 181
Rozanolixizumab ~10 mg/kg43.017 – 242
Between Cycle 5 and Cycle 6
GroupValue95% CI
Rozanolixizumab ~7 mg/kg44.022 – 192
Rozanolixizumab ~10 mg/kg43.015 – 175
Between Cycle 6 and Cycle 7
GroupValue95% CI
Rozanolixizumab ~7 mg/kg36.08 – 348
Rozanolixizumab ~10 mg/kg43.07 – 106
Between Cycle 7 and Cycle 8
GroupValue95% CI
Rozanolixizumab ~7 mg/kg37.022 – 87
Rozanolixizumab ~10 mg/kg37.06 – 84
Between Cycle 8 and Cycle 9
GroupValue95% CI
Rozanolixizumab ~7 mg/kg36.08 – 120
Rozanolixizumab ~10 mg/kg36.07.0 – 134

Adverse events — posted to ClinicalTrials.gov

Time frame: Enrolled, but not treated: From Screening until End of Study (up to 34 months); Randomized participants: From Baseline (Day 1) until End of Study (up to 34 months). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Enrolled, But Not Treated
Serious: 3/8 (38%)
Deaths: 1/8
Rozanolixizumab ~7 mg/kg
Serious: 16/102 (16%)
Deaths: 1/102
Rozanolixizumab ~10 mg/kg
Serious: 31/102 (30%)
Deaths: 3/102

Serious adverse events (48 terms)

ReactionSystemEnrolled, But Not TreatedRozanolixizumab ~7 mg/kgRozanolixizumab ~10 mg/kg
Myasthenia gravisNervous system disorders
Myasthenia gravis crisisNervous system disorders
COVID-19Infections and infestations
Iron deficiency anaemiaBlood and lymphatic system disorders
Cardiac failureCardiac disorders
Angina unstableCardiac disorders
Myocardial infarctionCardiac disorders
Atrial fibrillationCardiac disorders
Adrenal disorderEndocrine disorders
Macular holeEye disorders
Large intestine polypGastrointestinal disorders
Duodenal ulcer haemorrhageGastrointestinal disorders
Oesophageal food impactionGastrointestinal disorders
AstheniaGeneral disorders
PyrexiaGeneral disorders
General physical health deteriorationGeneral disorders
AppendicitisInfections and infestations
Complicated appendicitisInfections and infestations
DiverticulitisInfections and infestations
PeritonitisInfections and infestations
Sinusitis aspergillusInfections and infestations
Pneumonia bacterialInfections and infestations
EndocarditisInfections and infestations
Meningitis asepticInfections and infestations
COVID-19 pneumoniaInfections and infestations
Other adverse events (34 terms — click to expand)

ReactionSystemEnrolled, But Not TreatedRozanolixizumab ~7 mg/kgRozanolixizumab ~10 mg/kg
HeadacheNervous system disorders
DiarrhoeaGastrointestinal disorders
COVID-19Infections and infestations
PyrexiaGeneral disorders
NauseaGastrointestinal disorders
Blood immunoglobulin G decreasedInvestigations
ArthralgiaMusculoskeletal and connective tissue disorders
Upper respiratory tract infectionInfections and infestations
NasopharyngitisInfections and infestations
Abdominal painGastrointestinal disorders
DizzinessNervous system disorders
Oropharyngeal painRespiratory, thoracic and mediastinal disorders
VomitingGastrointestinal disorders
Low density lipoprotein increasedInvestigations
Back painMusculoskeletal and connective tissue disorders
HypertensionVascular disorders
Oedema peripheralGeneral disorders
Blood cholesterol increasedInvestigations
Lymphocyte count decreasedInvestigations
Influenza like illnessGeneral disorders
Abdominal pain upperGastrointestinal disorders
Urinary tract infectionInfections and infestations
Blood triglycerides increasedInvestigations
MyalgiaMusculoskeletal and connective tissue disorders
Myasthenia gravisNervous system disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
CoughRespiratory, thoracic and mediastinal disorders
FallInjury, poisoning and procedural complications
Interferon gamma release assay positiveInvestigations
Hepatic painGeneral disorders
Urinary tract infection bacterialInfections and infestations
Streptococcal urinary tract infectionInfections and infestations
HypomagnesaemiaMetabolism and nutrition disorders
Fluid overloadMetabolism and nutrition disorders

Most-reported serious reactions: Myasthenia gravis, Myasthenia gravis crisis, COVID-19, Iron deficiency anaemia, Cardiac failure, Angina unstable, Myocardial infarction, Atrial fibrillation.

Data from ClinicalTrials.gov NCT04650854 adverse events section.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
    Bril V, Drużdż A, Grosskreutz J, Habib AA, et al · · 2023 · cited 176× · PMID 37059507 · DOI 10.1016/s1474-4422(23)00077-7
  2. Immunotherapies in MuSK-positive Myasthenia Gravis; an IgG4 antibody-mediated disease.
    Vakrakou AG, Karachaliou E, Chroni E, Zouvelou V, et al · · 2023 · cited 33× · PMID 37564637 · DOI 10.3389/fimmu.2023.1212757
  3. Antibody Therapies in Autoimmune Neuromuscular Junction Disorders: Approach to Myasthenic Crisis and Chronic Management.
    Vanoli F, Mantegazza R. · · 2022 · cited 26× · PMID 35165857 · DOI 10.1007/s13311-022-01181-3
  4. New and emerging treatments for myasthenia gravis.
    DeHart-McCoyle M, Patel S, Du X. · · 2023 · cited 25× · PMID 37560511 · DOI 10.1136/bmjmed-2022-000241
  5. Rozanolixizumab: First Approval.
    Hoy SM. · · 2023 · cited 24× · PMID 37656420 · DOI 10.1007/s40265-023-01933-1
  6. New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making.
    Gerischer L, Doksani P, Hoffmann S, Meisel A. · · 2025 · cited 18× · PMID 39869260 · DOI 10.1007/s40259-024-00701-1
  7. Targeting the Neonatal Fc Receptor in Autoimmune Diseases: Pipeline and Progress.
    Gjølberg TT, Mester S, Calamera G, Telstad JS, et al · · 2025 · cited 10× · PMID 40156757 · DOI 10.1007/s40259-025-00708-2
  8. Exploring the depths of IgG4: insights into autoimmunity and novel treatments.
    Ünlü S, Sánchez Navarro BG, Cakan E, Berchtold D, et al · · 2024 · cited 9× · PMID 38698867 · DOI 10.3389/fimmu.2024.1346671

Verify or expand the search:

Other trials of Rozanolixizumab

Trials testing the same drug.

Other recruiting trials for Generalized Myasthenia Gravis

Currently open trials in the same condition.

Other UCB Biopharma SRL trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04650854.

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