Adults 18 to 85, any sex, with Myasthenia Gravis, Generalized. 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.
Mean Change From Baseline in the Quantitative Myastenia Gravis (QMG) Score at Week 25. Posterior Median Was Used as Measure Type.Primary· week 25
QMG score is an established validated measure of disease severity used in MG trials (Jaretzki et al 2000). The scoring system is based on quantitative testing of sentinel muscle groups by means of a 4 point scale ranging from 0 (no symptoms) to 3 (severe symptoms). The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and the total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) (Sharshar et al 2000, Bedlack et al 2005).
Group
Value
95% CI
CFZ533
-4.07
-5.67 – -2.47
Placebo
-2.93
-4.53 – -1.33
Mean Changes From Baseline in the Myasthenia Gravis Composite (MGC) Score. Posterior Median Was Used as Measure Type.Secondary· From baseline to week 49
The Myasthenia Gravis Composite (MGC) score is another key efficacy outcome measure, ranging from 0 to 50. It is reliable and demonstrates concurrent and longitudinal construct validity in the MG practice care setting (Burns et al 2010). The MGC scale covers 10 important functional domains most frequently involved in patients with MG. The proportion of bulbar and respiratory items reflect the clinical importance of these domains in the disease, and are appropriately weighted.
The assessment of each of the 10 test items provides immediate insight into the status of that particular functional d
Group
Value
95% CI
CFZ533
-8.00
-9.83 – -6.16
Placebo
-5.62
-7.45 – -3.78
Proportion of Patients With Improvement or Worsening by ≥ 3 Points in the QMG ScoreSecondary· at week 49
QMG score is an established validated measure of disease severity used in MG trials (Jaretzki et al 2000). The scoring system is based on quantitative testing of sentinel muscle groups by means of a 4 point scale ranging from 0 (no symptoms) to 3 (severe symptoms). The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and the total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) (Sharshar et al 2000, Bedlack et al 2005).
improvement by ≥ 3 points in the QMG score
Group
Value
95% CI
CFZ533
10
Placebo
9
worsening by ≥ 3 points in the QMG score
Group
Value
95% CI
CFZ533
2
Placebo
2
Proportion of Patients Intolerant to Steroid TaperSecondary· week 49
Group
Value
95% CI
CFZ533
NA
Placebo
NA
Number of Patients Who Discontinued Due to Inefficacy or WorseningSecondary· week 49
Group
Value
95% CI
CFZ533
0
Placebo
0
Mean Change From Baseline in the Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL)Secondary· week 25
The MG-ADL is an 8-item survey to assess functional performance of daily activities that are sometimes impaired by MG e.g. talking, breathing, swallowing etc. (Muppidi et al 2011). The higher score on MG-ADL scale (0-24 points) indicates worse functional performance of daily activities.
Group
Value
95% CI
CFZ533
-2.6
± 2.97
Placebo
-1.1
± 3.23
Mean Changes From Baseline in the QMG Score at Week 49Secondary· week 49
QMG (quantitative myasthenia gravis) score is an established validated measure of disease severity used in MG trials (Jaretzki et al 2000). The scoring system is based on quantitative testing of sentinel muscle groups by means of a 4 point scale ranging from 0 (no symptoms) to 3 (severe symptoms). The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and the total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) (Sharshar et al 2000, Bedlack et al 2005). A decrease in the QMG score indicated an improvement. Results given as a c
Group
Value
95% CI
CFZ533
-2.9
± 5.16
Placebo
-2.6
± 4.30
Mean Change From Baseline in the Myasthenia Gravis Quality of Life (MG QOL-15)Secondary· week 25
The MG-QOL15 is a 15-item survey, completed by MG patients and it is designed to assess some aspects of quality of life (QoL) related to MG (Burns et al 2011) e.g. assesment of mood, eating, speaking, driving a car etc.. The higher score on MG-QOL15 scale (0-60 points) indicates worse QoL.
Group
Value
95% CI
CFZ533
-9.7
± 11.0
Placebo
-6.7
± 10.86
Free CD40 on B CellsSecondary· week 1, week 25
CD40 receptor occupancy by CFZ533 in peripheral blood was assessed by flow cytometry analysis, measuring free or total CD40 receptors on whole blood B cells. Free CD40 on CD19-positive B cells, using PE-conjugated CFZ533 whose binding was prevented by bound, unconjugated CFZ533 (drug bound to CD40 on peripheral blood B cells). The more CD40 was occupied by unlabeled CFZ533, the less binding of labeled CFZ533, manifest as a lower mean fluorescence intensity (MFI) of CD40 on B cells. MFI from free CD40 on B cells was converted into Molecules of Equivalent Soluble Fluorochrome (MESF) using PE-MES
Free CD40 on B cells week 1 predose
Group
Value
95% CI
CFZ533
34242.9
± 18455.80
Placebo
31025.9
± 16138.97
Free CD40 on B cells week 25
Group
Value
95% CI
CFZ533
5259.1
± 11341.57
Placebo
24908.3
± 5022.03
Total Soluble CD40 (sCD40) in PlasmaSecondary· week1, week 25
PD
week 1
Group
Value
95% CI
CFZ533
0.1778
± 0.13077
Placebo
0.1577
± 0.17243
week 25
Group
Value
95% CI
CFZ533
191.1278
± 69.67597
Placebo
0.1163
± 0.18298
Plasma CFZ533 Concentration at Steady State Conditions (Week 17)Secondary· week 17
Group
Value
95% CI
CFZ533
120
± 40.5
Adverse events — posted to ClinicalTrials.gov
Time frame: up to week 49.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
CFZ533 10 mg/kg IV Infusion
Serious: 7/22 (32%)
Deaths: 0/22
Placebo IV Infusion
Serious: 4/22 (18%)
Deaths: 2/22
Serious adverse events (13 terms)
Reaction
System
CFZ533 10 mg/kg IV Infusion
Placebo IV Infusion
Influenza
Infections and infestations
—
—
Myasthenia gravis
Nervous system disorders
—
—
Febrile neutropenia
Blood and lymphatic system disorders
—
—
Myocardial ischaemia
Cardiac disorders
—
—
Glaucoma
Eye disorders
—
—
Abdominal pain upper
Gastrointestinal disorders
—
—
Constipation
Gastrointestinal disorders
—
—
Pyrexia
General disorders
—
—
Hepatitis toxic
Hepatobiliary disorders
—
—
Pneumonia
Infections and infestations
—
—
Brachial plexopathy
Nervous system disorders
—
—
Myasthenia gravis crisis
Nervous system disorders
—
—
Radial nerve palsy
Nervous system disorders
—
—
Other adverse events (124 terms — click to expand)
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics/pharmacodynamics and efficacy of CFZ533 as an add-on therapy to standard of care in patients with moderate to severe myasthenia gravis (MG).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04129528 — Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects
· Phase 2
· completed
NCT03781414 — Study of Efficacy, Safety, Tolerability, Pharmacokinetic (PK) and Pharmacodynamic (PD) of an Anti-CD40 Monoclonal Antibo
· Phase 2
· terminated
NCT03905525 — Study of Safety and Efficacy of Multiple Doses of CFZ533 in Two Distinct Populations of Patients With Sjogren's Syndrome
· Phase 2
· completed
NCT03827798 — Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa
· Phase 2
· active not recruiting
NCT03656562 — Study the Efficacy and Safety of VAY736 and CFZ533 in SLE Patients
· Phase 2
· completed
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Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 5 January 2021
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/NCT02565576.