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efgartigimod PH20

Phase 3

Primary Immune Thrombocytopenia | Monoclonal antibody | Hematology |argenx SE|Last Updated: Jul 21, 2026

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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment380
FDA Designations
No designations recorded
Clinical trial landscape

efgartigimod PH20 · 15 trials · 18 indications

Phase 3 10Phase 2 5
NCT07570316A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' DiseaseGraves' Disease
RECRUITING230 Analytics
NCT07596849A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease.Graves Disease
RECRUITING230 Analytics
NCT06684847A Study of the Efficacy and Safety of Efgartigimod in Patients With Primary Sjögren's SyndromePrimary Sjogrens Disease
ACTIVE NOT_RECRUITING631 Analytics
NCT06558279A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia GravisMyasthenia Gravis, Ocular
ACTIVE NOT_RECRUITING141 Analytics
NCT05979441A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory MyopathyMyositis
ENROLLING BY_INVITATION240 Analytics
NCT04812925A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune ThrombocytopeniaPrimary Immune Thrombocytopenia
ACTIVE NOT_RECRUITING173 Analytics
NCT04818671Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia GravisGeneralized Myasthenia Gravis
COMPLETED184 Analytics
NCT04735432Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia GravisGeneralized Myasthenia Gravis
COMPLETED110 Analytics
NCT04687072A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune ThrombocytopeniaPrimary Immune Thrombocytopenia
COMPLETED207 Analytics
NCT04598451A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)Pemphigus Vulgaris
COMPLETED222 Analytics
PHASE3RECRUITING
A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease
Graves' DiseaseUnlock trial analytics
PHASE3RECRUITING
A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease.
Graves DiseaseUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study of the Efficacy and Safety of Efgartigimod in Patients With Primary Sjögren's Syndrome
Primary Sjogrens DiseaseUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis
Myasthenia Gravis, OcularUnlock trial analytics
PHASE3ENROLLING BY_INVITATION
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
MyositisUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
Primary Immune ThrombocytopeniaUnlock trial analytics
PHASE3COMPLETED
Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis
Generalized Myasthenia GravisUnlock trial analytics
PHASE3COMPLETED
Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis
Generalized Myasthenia GravisUnlock trial analytics
PHASE3COMPLETED
A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
Primary Immune ThrombocytopeniaUnlock trial analytics
PHASE3COMPLETED
A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)
Pemphigus VulgarisUnlock trial analytics
Study Endpoints
Primary Endpoints
Percentage of participants who are euthyroid (fT3, fT4, and TSH within normal ranges) off ATDs at week 24 in part A
Up to 24 weeks (part A)

Free triiodothyronine: \[fT3\]; free thyroxine: \[fT4\]; ATDs: antithyroid drugs

Change from baseline in clinESSDAI score at week 48
Up to 48 weeks

The ESSDAI was designed to measure systemic disease activity in patients with PSjD and consists of 12 domains. The score varies between 0 (no disease activity) and 123 (max disease activity). The clinESSDAI includes all ESSDAI domains except the biological domain.

MGII (PRO) ocular score change from baseline to day 29 in part A
Up to 29 days

The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.

Incidence of AEs, SAEs and AESIs over time
Up to 53 months

AE : adverse event ; SAE : serious adverse event ; AESI : adverse event of special interest.

Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)
216 weeks
Vital sign measurement: blood pressure in the overall population
216 weeks
ECG: PR, QT and QRS interval in the overall population
216 weeks
Laboratory safety evaluations: CRP analysis in the overall population
216 weeks
Number of AEs, SAEs and AESIs
Up to 3.5 years

Adverse events, Serious Adverse event and Adverse events of special interest. Adverse events in the 'Infections and infestations' SOC were defined as AESIs because efgartigimod causes a transient reduction in total IgG levels.

Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set)
From week 0 to week 4

ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration).

Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24
Up to 6 weeks (between Weeks 19 and 24)

A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.

Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy
up to 30 weeks treatment period

Proportion of participants with pemphigus vulgaris who had CRmin within 30 weeks, defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

Change in CASE score in the NMDAR population
up to week 24

CASE= Clinical Assessment Scale in Autoimmune Encephalitis; NMDAR=N-methyl-D-aspartate receptor; Neuropsychological Status. The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness. This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.

Change from baseline in mRSS at week 24
Up to 24 weeks

The Modified Rodnan Skin Score is a scoring tool to assess skin thickness in 17 cutaneous sites across the body. Each site is rated on a semiquantitative score ranging from 0 (normal) to 3 (severe). The total score is the sum of the individual skin scores and can range from 0 to 51.

Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL)
Up to 12 weeks
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd)
Up to 12 weeks
Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis
Up to 12 weeks
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis
Up to 12 weeks
Number of Participants With CRoff at Week 36
at week 36

CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.

Incidence of treatment-emergent adverse events and serious adverse events
Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary Endpoints
Time to becoming euthyroid off ATDs in part A
Up to 24 weeks (part A)
Percentage of participants who are euthyroid and receiving MMI ≤5 mg/day or CBZ ≤7.5 mg/day at week 24 in part A
Up to 24 weeks (part A)
Percentage of participants with fT3 and fT4 levels below the ULN off ATDs at week 24 in part A
Up to 24 weeks (part A)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A - efgartigimod PH20 SC PFSEXPERIMENTALParticipants will receive efgartigimod PH20 SC Via Prefilled Syringe (PFS) during the part A double-blinded treatment period.
Part A - placebo PH20 SC PFSPLACEBO_COMPARATORParticipants will receive placebo PH20 SC Via Prefilled Syringe (PFS) during the part A double-blinded treatment period.
Part B - efgartigimod PH20 SC PFSEXPERIMENTALParticipants will receive efgartigimod PH20 SC Via Prefilled Syringe (PFS) during the part B treatment period.
Part B - placebo PH20 SC PFSEXPERIMENTALParticipants will receive placebo PH20 SC Via Prefilled Syringe (PFS) during the part B treatment period.
Part C - efgartigimod PH20 SC PFSEXPERIMENTALParticipants will receive efgartigimod PH20 SC PFS in the part C open-label treatment period
Double-blinded treatment period: Efgartigimod PH20 SCEXPERIMENTALParticipants receiving efgartigimod PH20 SC during the double-blinded treatment period
Double-blinded treatment period: Placebo PH20 SCPLACEBO_COMPARATORParticipants receiving placebo PH20 SC during the double-blinded treatment period
Open-label treatment periodEXPERIMENTALParticipants receiving efgartigimod PH20 SC during the open-label treatment period
Efgartigimod PH20 SC in part A+BEXPERIMENTALParticipants receiving efgartigimod PH20 SC during part A and part B
Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part BEXPERIMENTALParticipants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B
Efgartigimod PH20 SCEXPERIMENTALParticipants receiving efgartigimod PH20 subcutaneously
efgartigimodEXPERIMENTALPatients receiving efgartigimod intravenous (IV) treatment
Placebo PH20 SCPLACEBO_COMPARATORPatients receiving placebo PH20 SC treatment
placeboEXPERIMENTALpatients receiving placebo on top of prednisone
Part A (Open-Label Lead-in Period): Efgartigimod PH20 SCEXPERIMENTALAll participants will receive efgartigimod PH20 SC open label for 8 weeks
Part B (Double-blinded treatment period): Efgartigimod PH20 SCEXPERIMENTALParticipants will receive efgartigimod PH20 SC for 24 weeks
Part B (Maintenance double-blinded treatment period): Placebo PH20 SCPLACEBO_COMPARATORParticipants will receive placebo for 24 weeks
Part C (Open-Label Extension Period): Efgartigimod PH20 SCEXPERIMENTALParticipants who complete Part B will receive efgartigimod PH20 SC for 24 weeks
Interventions
NameTypeDescription
Efgartigimod PH20 SCBIOLOGICALSubcutaneous injection of Efgartigimod PH20 Via Prefilled Syringe (PFS)
Placebo PH20 SCOTHERSubcutaneous injection of placebo PH20 via Prefilled Syringe (PFS)
efgartigimod IVBIOLOGICALIntravenous infusion of efgartigimod
PlaceboOTHERSubcutaneous injection of placebo
prednisoneDRUGOral prednisone tablets
Efgartigimod PH20 (ARGX-113) SCBIOLOGICALsubcutaneous administrations of efgartigimod PH20 SC given by prefilled syringe (PFS). For participants aged 12 to \<18 years with body weight ≤50 kg, the study drug will be administered by vial and syringe.
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Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites18

Inclusion Criteria: * Is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF. * Has a documented diagnosis of GD with TRAb (anti-thyrotropin receptor antibody) levels \>=ULN (upper limit of normal) at screening. * Has active hyperthyroidism due to G...

Countries:United StatesGeorgiaJapanArgentinaAustraliaAustriaBelgiumBulgariaCanadaChileChinaColombiaEstoniaFranceGermanyGreeceHungaryIrelandIsraelItalyJordanLebanonLithuaniaMexicoNetherlandsPeruPhilippinesPolandPortugalRomaniaSaudi ArabiaSerbiaSlovakiaSouth KoreaSpainSwedenTunisiaTurkey (Türkiye)United KingdomCyprusCzechiaDenmarkFinlandUnited Arab EmiratesTaiwanThailandNew ZealandNorwayRussiaSouth AfricaIndiaUkraineCroatiaPuerto RicoSwitzerlandLatvia
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Recent Changes (Last 90 Days)
LOWJul 21, 2026NCT06655155lastUpdatePostDate: changed
LOWJul 21, 2026NCT05979441lastUpdatePostDate: changed
LOWJul 20, 2026NCT07583641primaryCompletionDate: changed
LOWJul 20, 2026NCT07583641primaryCompletionDate: changed
LOWJul 8, 2026NCT07596849Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 8, 2026NCT07570316Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 8, 2026NCT07596849Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 8, 2026NCT07570316Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 2, 2026NCT06558279primaryCompletionDate: changed
LOWJul 2, 2026NCT06558279primaryCompletionDate: changed
LOWJul 2, 2026NCT06558279primaryCompletionDate: changed
LOWJun 12, 2026NCT05979441lastUpdatePostDate: changed
LOWJun 12, 2026NCT05979441lastUpdatePostDate: changed
LOWJun 4, 2026NCT06392386lastUpdatePostDate: changed
LOWJun 4, 2026NCT06392386lastUpdatePostDate: changed
LOWJun 4, 2026NCT06392386lastUpdatePostDate: changed
LOWJun 4, 2026NCT06392386lastUpdatePostDate: changed
LOWJun 3, 2026NCT06392386lastUpdatePostDate: changed
LOWJun 3, 2026NCT05979441lastUpdatePostDate: changed
LOWJun 3, 2026NCT06392386lastUpdatePostDate: changed