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efgartigimod

Phase 3

Primary Immune Thrombocytopenia | Monoclonal antibody | Hematology |argenx SE|Last Updated: Jul 9, 2026

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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment232
FDA Designations
No designations recorded
Clinical trial landscape

efgartigimod · 16 trials · 26 indications

Phase 3 5Phase 2 7Phase 1 4
NCT06544499A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune ThrombocytopeniaPrimary Immune Thrombocytopenia (ITP)
RECRUITING69 Analytics
NCT06298552A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia GravisGeneralized Myasthenia Gravis
ACTIVE NOT_RECRUITING119 Analytics
NCT04980495An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia GravisGeneralized Myasthenia Gravis
COMPLETED69 Analytics
NCT04225156A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).Primary Immune Thrombocytopenia
COMPLETED101 Analytics
NCT04188379A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).Primary Immune Thrombocytopenia
COMPLETED131 Analytics
PHASE3RECRUITING
A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Primary Immune Thrombocytopenia (ITP)Unlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
Generalized Myasthenia GravisUnlock trial analytics
PHASE3COMPLETED
An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia Gravis
Generalized Myasthenia GravisUnlock trial analytics
PHASE3COMPLETED
A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
Primary Immune ThrombocytopeniaUnlock trial analytics
PHASE3COMPLETED
A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
Primary Immune ThrombocytopeniaUnlock trial analytics
Study Endpoints
Primary Endpoints
Extent of disease control, defined as the number of cumulative weeks during the 24-week Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L
Up to 24 weeks
MG-ADL total score change from baseline
Up to 29 days (part A)

The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)

Mean of the Average MG-ADL Total Score Change From Baseline During the Visit of Week 1 Through Week 21 by Regimen Arm
Up to 21 weeks

The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment.

Frequency and severity of Adverse Events
Up to 60 weeks
Frequency and severity of vital signs
Up to 60 weeks
Frequency and severity of laboratory assessments
Up to 60 weeks
Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial.
From Week 19 up to Week 24

Percentage of participants with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between Week 19 and 24 of the study.

Incidence of adverse events and serious adverse events in parts A and B
Up to 21 weeks
Efgartigimod serum concentrations in the DBTP
Up to 24 weeks
Total IgG levels in the DBTP
Up to 24 weeks
Number of Participants With TEAEs, TESAEs and TEAESIs
From the first dose of study drug (Day 1) up to 60 days post last study drug, approximately 56 weeks

An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was any untoward medical occurrence that, at any dose: resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or any other medically important event. Treatment-emergent adverse events (TEAEs) were defined as AEs with onset on or after the first administration of study drug up to and including 60 days after the last study drug administration. Adverse events in the 'Infections and infestations' SOC were defined as AE of Special Interest (AESIs) because efgartigimod causes a transient reduction in total IgG levels.

Percentage of Participants Meeting Overall CRESS Response of at Least 3 of 5 Items at Week 24
Week 24

A Composite of Relevant Endpoints for Sjögren's Syndrome (CRESS) responder is defined as improvements in at least 3 of the 5 items of CRESS (systemic disease activity, patient-reported symptoms, tear gland function, salivary gland function and serology. The score ranges from 0 to 9 (higher score = worse symptoms).

Change from baseline to week 24 in urine protein creatinine ratio (UPCR)
up to 24 weeks
Change From Baseline to Week 24 in the COMPASS 31 (2-week Recall Version)
Baseline (Day 1) and Week 24

Composite Autonomic Symptom Score (COMPASS) 31 modified version (2-week recall) is a self-rated questionnaire to evaluate the severity and distribution of autonomic symptoms in various autonomic nerve disorders. It consists of 31 questions in 6 weighted domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal {GI}-mixed upper and diarrhea, bladder, and pupillomotor). A weighted total score of 0 (mild) to 100 (severe) was determined by adding a maximum raw score for each domain. Higher scores indicated a more severe degree of autonomic symptoms.

Change From Baseline to Week 24 in the MaPS
Baseline (Day 1) and Week 24

The Malmö POTS Symptom Score (MaPS) score is a dedicated POTS symptom scoring questionnaire. The score consists of 12 questions that assess symptom burden related (tachycardia, palpitations, dizziness, presyncope) and unrelated to orthostatic intolerance (GI symptoms, insomnia, concentration difficulties). Participants graded their symptoms for the past 7 days using a visual analog scale ranging from 0 (no symptoms) to 10 (worst possible). The total score was calculated by summing up the items/individual items and range was 0 to 120 points, with higher scores indicating more severe symptoms.

Number of Participants With TEAEs and TESAEs
From the first dose of study drug (Day 1) up to 60 days post last dose of study drug, up to 236 days

An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was any untoward medical occurrence that at any dose resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or any other medically important event. Treatment-emergent adverse events (TEAEs) were defined as AEs with onset on or after the first administration of study drug up to and including 60 days after the last study drug administration.

Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Clearance (CL)
up to 26 weeks

Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod

Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Volume of Distribution (Vd)
up to 26 weeks

Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod

Total Immunoglobulin G (IgG) levels as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
up to 26 weeks

Total Immunoglobulin G levels will be measured from blood samples

Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
up to 26 weeks

Total Immunoglobulin G (IgG) levels will be measured from blood samples

Reduction in serum C7 antibody levels
26 weeks

The proportion of patients who exhibit reduction in serum C7 antibody levels at week 26 as compared to week 1.

Adverse Events and Effects
38 weeks

Occurrence of adverse events and effects.

The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) improvement
26 weeks

The overall improvement of EB symptoms at week 26 as compared to week 1, measured by percentage change of a participant's EBDASI score (overall total score, total activity score, and total damage score). The EBDASI is scored in the range of 0 - 506, with a lower score corresponding to mild disease and higher score corresponding to more severe disease.

Efgartigimod PK parameters (Cmax)
up to 64 days
Variation in the immune response assessed by comparing pneumococcal capsular polysaccharide (PCP) titers pre- and postadministration of the PNEUMOVAX 23 vaccine between study arms
throughout the study (up to 12 weeks)
Percentage reduction in total IgG levels, compared to baseline, at day 29 (week 4), 7 days after the fourth IV or SC administration of efgartigimod
After four weeks (day 29)
Secondary Endpoints
Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP
Up to 6 weeks
Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP
Up to 8 weeks
Proportion of participants achieving a platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP
Up to 12 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Efgartigimod IVEXPERIMENTALParticipants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)
Placebo IVEXPERIMENTALParticipants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)
PlaceboPLACEBO_COMPARATORPatients receiving placebo during part A and receiving efgartigimod IV during part B
Efgartigimod IV - continuous regimenEXPERIMENTALParticipants receiving efgartigimod IV on a continuous regimen
Efgartigimod IV - cyclic regimenEXPERIMENTALParticipants receiving efgartigimod IV on a cyclic regimen
efgartigimodEXPERIMENTALpatients receiving efgartigimod
Efgartigimod IV + Empasiprubart IVEXPERIMENTALParticipants receive efgartigimod IV in part A, B and C and empasiprubart IV in part B
Efgartigimod IV (part A + C)EXPERIMENTALParticipants not eligible for part B, receiving efgartigimod IV in part A and C
Efgartigimod IV armEXPERIMENTALpatients receiving infusions of Efgartigimod IV
Placebo armPLACEBO_COMPARATORpatients receiving infusions of placebo IV
Mild renal impairmentEXPERIMENTALPatients with 60 \<= eGFR \<90
Moderate renal impairmentEXPERIMENTALPatients with 30 \<= eGFR \<60
Severe renal impairmentEXPERIMENTALPatients with eGFR \<30
Normal renal functionEXPERIMENTALPatients with eGFR \>=90
Efgartigimod-1EXPERIMENTALWeekly efgartigimod infusions and the pneumovax 23 vaccine on day 22
Efgartigimod-2EXPERIMENTALWeekly efgartigimod infusions and the pneumovax 23 vaccine on day 36
efgartigimod PH20 SCEXPERIMENTALsubcutaneous injections of efgartigimod PH20 SC
Interventions
NameTypeDescription
Efgartigimod IVBIOLOGICALIntravenous infusion of efgartigimod
Placebo IVOTHERIntravenous infusion of placebo
efgartigimodBIOLOGICALIntravenous infusion of efgartigimod
PlaceboOTHERIntravenous infusion of placebo
Empasiprubart IVBIOLOGICALIntravenous infusion of empasiprubart
PNEUMOVAX 23BIOLOGICALPNEUMOVAX 23 vaccine
efgartigimod PH20 SCBIOLOGICALsubcutaneous injections of efgartigimod PH20 SC
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Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites94

Inclusion Criteria: * Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF). * Has documented baseline mean platelet count of \<30 x 10\^9/L before randomization * Has a documented duration of primary immune thrombocytopenia...

Countries:United StatesAustriaBulgariaChinaCroatiaCzechiaFranceGermanyHungaryIrelandItalyPolandPortugalRomaniaSerbiaSpainUnited KingdomBelgiumCanadaCyprusDenmarkFinlandGeorgiaGreeceNetherlandsNorwaySaudi ArabiaJapanRussiaTurkey (Türkiye)UkraineLithuania
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Recent Changes (Last 90 Days)
LOWJul 9, 2026NCT07194850lastUpdatePostDate: changed
LOWJul 9, 2026NCT07194850lastUpdatePostDate: changed
MEDIUMJun 27, 2026NCT04980495TRIAL_REMOVED: changed
MEDIUMJun 27, 2026NCT04980495TRIAL_REMOVED: changed
LOWJun 12, 2026NCT07284420lastUpdatePostDate: changed
LOWJun 12, 2026NCT07194850lastUpdatePostDate: changed
LOWJun 12, 2026NCT07284420lastUpdatePostDate: changed
LOWJun 12, 2026NCT07194850lastUpdatePostDate: changed
LOWJun 3, 2026NCT07284420lastUpdatePostDate: changed
LOWJun 3, 2026NCT07194850lastUpdatePostDate: changed
LOWJun 3, 2026NCT07284420lastUpdatePostDate: changed
LOWJun 3, 2026NCT07194850lastUpdatePostDate: changed
LOWJun 2, 2026NCT07284420lastUpdatePostDate: changed
LOWJun 2, 2026NCT07284420lastUpdatePostDate: changed
LOWJun 2, 2026NCT07284420lastUpdatePostDate: changed
LOWMay 29, 2026NCT07194850lastUpdatePostDate: changed
LOWMay 29, 2026NCT07284420lastUpdatePostDate: changed
LOWMay 29, 2026NCT07194850lastUpdatePostDate: changed
LOWMay 29, 2026NCT07284420lastUpdatePostDate: changed
LOWMay 29, 2026NCT07194850lastUpdatePostDate: changed