IgG4-related Disease
Conditions
Keywords
efgartigimod, IgG4-RD, IgG4-related disease, FcRn, FcRn inhibitor
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
Interventions
DRUGEfgartigimod
efgartigimod 1000 mg subcutaneous injection given once weekly
Sponsors
Stanford University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in volume on FDG-PET/MRI of lacrimal gland(s) and/or | From Baseline to Week 12 | — |
| Change in volume on FDG-PET/MRI of salivary gland(s) and/or | From Baseline to Week 12 | Salivary glands include parotid glands, submandibular glands, sublingual glands |
| Change in volume of pancreas on FDG-PET/MRI | From Baseline to Week 12 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in FDG avidity (SUVmax) of lacrimal glands on PET | Baseline to Week 12 | — |
| Change in FDG avidity (SUVmean) of lacrimal glands on PET | Baseline to Week 12 | — |
| Change in FDG avidity (total gland glycolysis) of lacrimal glands on PET | Baseline to Week 12 | Total gland glycolysis is SUVmean x gland volume |
| Change in FDG avidity (SUVmax) of salivary glands on PET | Baseline to Week 12 | — |
| Change in FDG avidity (SUVmean) of salivary glands on PET | Baseline to Week 12 | — |
| Change in FDG avidity (total gland glycolysis) of salivary glands on PET | Baseline to Week 12 | Total gland glycolysis is SUVmean x gland volume |
| Change in FDG avidity (SUVmax) of pancreas on PET | Baseline to Week 12 | — |
| Change in FDG avidity (SUVmean) of pancreas on PET | Baseline to Week 12 | — |
| Change in FDG avidity (total pancreatic glycolysis) of pancreas on PET | Baseline to Week 12 | Total pancreatic glycolysis is SUVmean x pancreatic volume |
| Change in exchange transfer (K^trans) of lacrimal glands on MRI | From Baseline to Week 12 | — |
| Change in apparent diffusion coefficient (ADC) of lacrimal glands on MRI | From Baseline to Week 12 | — |
| Change in microvascular volume fraction (f) of lacrimal glands on MRI | From Baseline to Week 12 | — |
| Change in exchange transfer (K^trans) of salivary glands on MRI | From Baseline to Week 12 | — |
| Change in apparent diffusion coefficient (ADC) of salivary glands on MRI | From Baseline to Week 12 | — |
| Change in microvascular volume fraction (f) of salivary glands on MRI | From Baseline to Week 12 | — |
| Change in apparent diffusion coefficient (ADC) of pancreas on MRI | From Baseline to Week 12 | — |
| Change in T1 mapping of pancreas on MRI | From Baseline to Week 12 | — |
| Change in T2 mapping of pancreas on MRI | From Baseline to Week 12 | — |
| Change in extracellular volume (ECV) of pancreas on MRI | From Baseline to Week 12 | — |
| Change in microvascular perfusion fraction of pancreas on MRI | From Baseline to Week 12 | — |
| Change in serum IgG4 level | From Baseline to Week 12 | — |
| Change in serum IgG level | From Baseline to Week 12 | — |
| Change in serum IgE level | From Baseline to Week 12 | — |
| Change in plasmablast count | From Baseline to Week 12 | — |
| Change in absolute regulatory B cell count | From Baseline to Week 12 | — |
| Change in IgG4-RD Responder Index | From Baseline to Week 12 | — |
| Change in physician global assessment of disease | From Baseline to Week 12 | Symptoms rated on a 100 mm VAS. Score range: 0 to 100, higher scores correspond to worse disease state. |
| Change in patient global assessment of disease | From Baseline to Week 12 | Symptoms rated on a 100 mm VAS. Score range: 0 to 100, higher scores correspond to worse disease state. |
| Change in patient global assessment of ocular symptoms | From Baseline to Week 12 | Symptoms rated on a 100 mm VAS. Score range: 0 to 100, higher scores correspond to worse disease state. |
| Change in patient global assessment of salivary symptoms | From Baseline to Week 12 | Symptoms rated on a 100 mm VAS. Score range: 0 to 100, higher scores correspond to worse disease state. |
| Change in FACIT-F fatigue score | From Baseline to Week 12 | Total score range: 0-52, lower scores correspond with more fatigue. |
| Change in C3 laboratory assessment | From Baseline to Week 12 | — |
| Change in C4 laboratory assessment | From Baseline to Week 12 | — |
| Change in total IgG laboratory assessment | From Baseline to Week 12 | — |
| Change in ESR laboratory assessment | From Baseline to Week 12 | — |
| Change in CRP laboratory assessment | From Baseline to Week 12 | — |
| Number of participants with safety endpoints of interest | From Screening to end of follow-up at Week 18 | Safety endpoints of interest include hypogammaglobulinemia, severe infection requiring hospitalization or IV antibiotics, and mortality |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORMatthew C Baker, MD, MS
Stanford University
Outcome results
None listed