Clinical trial NCT07025330: A Study of Efgartigimod in Patients With IgG4-Related Disease
Official title: A Phase IIa, Single-Site, Open-Label Study of Efgartigimod in Patients With IgG4-Related Disease
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: IgG4-related Disease
Keywords: efgartigimod
IgG4-RD
IgG4-related disease
FcRn
FcRn inhibitor
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2025-06-17
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this clinical trial is to learn if efgartigimod can treat IgG4-related disease in adults. The main questions it aims to answer are:

In patients with IgG4-related disease, does treatment with efgartigimod reduce the volume of the:

* lacrimal gland(s) and/or
* salivary gland(s) and/or
* pancreas

Participants will:

* Receive efgartigimod once weekly for up to 12 weeks
* Visit the clinic every one to six weeks for checkups and tests
* Be asked to complete questionnaires to see how they feel on efgartigimod


Eligibility criteria: Key Inclusion Criteria:

* Have a clinical diagnosis of IgG4-related disease that requires treatment in the opinion of the investigator
* Meet the 2019 ACR/EULAR Classification Criteria for IgG4-Related Disease
* Have a serum IgG4 concentration greater than or equal to 2 times the upper limit of normal at Screening
* Have involvement of the lacrimal gland(s), salivary gland(s), and/or pancreas

  * If lacrimal and/or salivary glands are involved, it must be symptomatic, including but not limited to discomfort, pain, dryness, headache, or vision changes
  * If the pancreas is involved, it must be asymptomatic, diffuse enlargement without signs or symptoms of obstruction or evidence of major organ dysfunction in the opinion of the investigator
* Have a prior inadequate response to, or intolerance of, glucocorticoids, or who have experienced recurrent symptoms after previous treatment with glucocorticoids
* Are not receiving current treatment with immunosuppressive medications
* All women must test negative for pregnancy and agree to use a reliable method of birth control

Key Exclusion Criteria:

* Any exclusion criteria listed in the 2019 ACR/EULAR Classification Criteria for IgG4-Related Disease
* Prior treatment with an FcRn inhibitor
* Have conventional synthetic disease-modifying antirheumatic drug (csDMARD) or immunosuppressive use as follows:

  * Treatment with glucocorticoids within 28 days prior to Baseline or planned treatment during the study
  * Treatment with csDMARDs including but not limited to hydroxychloroquine, methotrexate, leflunomide, or sulfasalazine within 28 days prior to Baseline or planned treatment during the study
  * Treatment with cytotoxic or immunosuppressive drugs including but not limited to cyclophosphamide, mycophenolic acid, azathioprine, cyclosporine, sirolimus, or tacrolimus within 28 days prior to Baseline or planned treatment during the study
  * Treatment with a janus kinase (JAK) inhibitor including but not limited to tofacitinib, baricitinib, upadacitinib, or filgotinib within 28 days prior to Baseline or planned treatment during the study
  * Treatment with a Bruton's tyrosine kinase (BTK) inhibitor including but not limited to ibrutinib, zanubrutinib, acalabrutinib, pirtobrutinib, or rilzabrutinib within 28 days prior to Baseline or planned treatment during the study
* Have biologic disease-modifying antirheumatic drug (bDMARD) use as follows:

  * Treatment with etanercept, adalimumab, or anakinra within 28 days before Baseline or planned treatment during the study
  * Treatment with infliximab, certolizumab pegol, golimumab, abatacept, or tocilizumab within 56 days before Baseline or planned treatment during the study
  * Treatment with a B cell depleting agent including but not limited to rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab, ianalumab, or obexelimab ≤ 6 months prior to Baseline
  * Patients who received B-cell targeted therapy \> 6 and ≤ 12 months prior to Baseline must have a B-cell count that is within the laboratory reference range at Screening
  * Treatment with a BAFF antagonist including but not limited to belimumab or tabalumab within 6 months before Baseline or planned treatment during the study
  * Treatment with an IL-17 antagonist including but not limited to secukinumab, ixekizumab, or brodalumab within 6 months before Baseline or planned treatment during the study
  * Prior treatment with other bDMARDs may be allowed at the discretion of the investigator
* A history of, or current, inflammatory or autoimmune disease (that could affect the interpretation of safety or efficacy outcomes) other than IgG4-related disease
* Evidence of active tuberculosis, HIV, or hepatitis B or C infection
* History of cancer except for skin basal or squamous cell carcinoma, cervical dysplasia or carcinoma in situ that has been treated and is considered cured \> 1 year prior to Baseline, prostate cancer considered cured for \> 5 years with a normal prostate specific antigen, or colon cancer considered cured \> 5 years

Primary outcomes:
measure: Change in volume on FDG-PET/MRI of lacrimal gland(s) and/or
timeFrame: From Baseline to Week 12
measure: Change in volume on FDG-PET/MRI of salivary gland(s) and/or
timeFrame: From Baseline to Week 12
measure: Change in volume of pancreas on FDG-PET/MRI
timeFrame: From Baseline to Week 12
Secondary outcomes:
measure: Change in FDG avidity (SUVmax) of lacrimal glands on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (SUVmean) of lacrimal glands on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (total gland glycolysis) of lacrimal glands on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (SUVmax) of salivary glands on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (SUVmean) of salivary glands on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (total gland glycolysis) of salivary glands on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (SUVmax) of pancreas on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (SUVmean) of pancreas on PET
timeFrame: Baseline to Week 12
measure: Change in FDG avidity (total pancreatic glycolysis) of pancreas on PET
timeFrame: Baseline to Week 12
measure: Change in exchange transfer (K^trans) of lacrimal glands on MRI
timeFrame: From Baseline to Week 12
measure: Change in apparent diffusion coefficient (ADC) of lacrimal glands on MRI
timeFrame: From Baseline to Week 12
measure: Change in microvascular volume fraction (f) of lacrimal glands on MRI
timeFrame: From Baseline to Week 12
measure: Change in exchange transfer (K^trans) of salivary glands on MRI
timeFrame: From Baseline to Week 12
measure: Change in apparent diffusion coefficient (ADC) of salivary glands on MRI
timeFrame: From Baseline to Week 12
measure: Change in microvascular volume fraction (f) of salivary glands on MRI
timeFrame: From Baseline to Week 12
measure: Change in apparent diffusion coefficient (ADC) of pancreas on MRI
timeFrame: From Baseline to Week 12
measure: Change in T1 mapping of pancreas on MRI
timeFrame: From Baseline to Week 12
measure: Change in T2 mapping of pancreas on MRI
timeFrame: From Baseline to Week 12
measure: Change in extracellular volume (ECV) of pancreas on MRI
timeFrame: From Baseline to Week 12
measure: Change in microvascular perfusion fraction of pancreas on MRI
timeFrame: From Baseline to Week 12
measure: Change in serum IgG4 level
timeFrame: From Baseline to Week 12
measure: Change in serum IgG level
timeFrame: From Baseline to Week 12
measure: Change in serum IgE level
timeFrame: From Baseline to Week 12
measure: Change in plasmablast count
timeFrame: From Baseline to Week 12
measure: Change in absolute regulatory B cell count
timeFrame: From Baseline to Week 12
measure: Change in IgG4-RD Responder Index
timeFrame: From Baseline to Week 12
measure: Change in physician global assessment of disease
timeFrame: From Baseline to Week 12
measure: Change in patient global assessment of disease
timeFrame: From Baseline to Week 12
measure: Change in patient global assessment of ocular symptoms
timeFrame: From Baseline to Week 12
measure: Change in patient global assessment of salivary symptoms
timeFrame: From Baseline to Week 12
measure: Change in FACIT-F fatigue score
timeFrame: From Baseline to Week 12
measure: Change in C3 laboratory assessment
timeFrame: From Baseline to Week 12
measure: Change in C4 laboratory assessment
timeFrame: From Baseline to Week 12
measure: Change in total IgG laboratory assessment
timeFrame: From Baseline to Week 12
measure: Change in ESR laboratory assessment
timeFrame: From Baseline to Week 12
measure: Change in CRP laboratory assessment
timeFrame: From Baseline to Week 12
measure: Number of participants with safety endpoints of interest
timeFrame: From Screening to end of follow-up at Week 18
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
Study officials:
affiliation: Stanford University
name: Matthew C Baker, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: