INEBILIZUMAB for Immunoglobulin G4-related disease

Inclusion criteria

• {"criterion_text":"- 101\tParticipants must weigh ≥ 17 kg to be eligible for enrollment.\n- 102\tParticipant has provided informed consent/assent before initiation of any study specific activities/procedures. Participant’s legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent, and the participant has provided written assent based on local regulations and/or guidelines before any study-specific activities/procedures being initiated.\n- 103\tAge 2 to < 18 years at the time of screening. For participants who reach the age of legal consent during the clinical study, notification will be required, and a new consent form must be signed by the participant for continuation in the study.\n- 104\tClinical diagnosis of IgG4-RD.\n- 105\tFulfillment of the 2019 American College of Rheumatology (ACR) and the European League Against Rheumatism (EULAR) classification criteria as determined by the principal investigator (PI) at screening. Specifically, participants must meet the classification criteria entry requirements (including involvement of one of the following organs: pancreas, bile ducts/biliary tree, orbits, lungs, kidneys, lacrimal glands, major salivary glands, retroperitoneum, aorta, pachymeninges, or thyroid gland [Riedel’s thyroiditis]), must not meet any of the classification criteria exclusions, and must achieve at least 20 classification criteria inclusion points.\n- 106\tReceipt of all age-appropriate and locally-required vaccinations before screening.\n- 107\tParticipants requiring treatment in addition to or other than GCs for IgG4 RD according to PI’s assessment at screening.\n- 108\tParticipants who are on GCs for the treatment of IgG4-RD should remain on a stable dose for at least 2 weeks before enrollment (day 1). Tapering post enrollment will be at PI’s discretion."}

Exclusion criteria

• {"criterion_text":"- 201\tParticipants with any of the following abnormal liver function tests in the presence of hepatobiliary IgG4-RD activity: •\taspartate aminotransferase (AST) > 10 × upper limit of normal (ULN) •\talanine aminotransferase (ALT) > 10 × ULN •\ttotal bilirubin (TBL) > 5 × ULN Screening liver function tests may be repeated before day 1 to permit abnormal values due to hepatobiliary IgG4-RD activity to respond to glucocorticoid treatment.\n- 218\tReceipt of any of the following before day 1: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy.\n- 202\tMalignancy (except any of the following nonmelanoma skin cancers, or cervical or breast ductal carcinoma in situ) within the last 5 years.\n- 219\tReceipt of any of the following within 2 months before day 1: azathioprine, mycophenolate mofetil, cyclosporine, methotrexate, cyclophosphamide, tocilizumab, satralizumab, eculizumab, and mitoxantrone.\n- 220\tReceipt of rituximab or any experimental B-cell depleting agent (eg, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab), or any non-depleting B-cell-directed therapy (eg, belimumab), abatacept, within 6 months before screening unless B cell counts have returned to ≥ one-half the LLN.\n- 221\tReceipt of intravenous immunoglobulin (IVIG) within 1 month before day 1.\n- 222\tReceipt of natalizumab within 6 months before day 1.\n- 223\tSevere drug allergic history or anaphylaxis to 2 or more food products or medicines (including known sensitivity to acetaminophen/paracetamol, diphenhydramine or equivalent antihistamine, and methylprednisolone or equivalent glucocorticoid).\n- 224\tReceipt of any live or attenuated vaccine (administration of inactivated [killed] vaccine is acceptable) within 4 weeks before day 1, Bacillus Calmette-Guérin vaccine within 1 year of screening, or blood transfusion within 4 weeks before screening or during screening.\n- 225\tCurrently receiving treatment in another investigational device or drug study, or less than 5 half-lives since ending treatment in another investigational device or drug study. This does not apply to other investigational procedures or participation in observational research studies.\n- 226\tParticipants of childbearing potential unwilling to use protocol-specified method of contraception (see Section 11.5 [Appendix 5]) during treatment and for an additional 6 months after the last dose of inebilizumab.\n- 210\tKnown history of congenital or acquired immunodeficiency (eg, due to human immunodeficiency virus [HIV] infection, splenectomy, immunosuppression-related or idiopathic T-cell deficiencies) that predisposes the participant to infection.\n- 227\tParticipants who are breastfeeding or who plan to breastfeed while on study through 6 months after the last dose of investigational product.\n- 228\tParticipants planning to become pregnant while on study through 6 months after the last dose of investigational product.\n- 203\tMajor surgery within 8 weeks before screening.\n- 229\tParticipants of childbearing potential with a positive pregnancy test assessed at screening by a highly sensitive urine or serum pregnancy test.\n- 230\tParticipants with a partner of childbearing potential who are unwilling to practice sexual abstinence (refrain from heterosexual intercourse) or use contraception during treatment and for an additional 6 months after the last dose of investigational product. Refer to Section 11.5 (Appendix 5) for additional contraceptive information.\n- 231\tParticipants assigned male at birth with a pregnant partner who are unwilling to practice abstinence or use a condom during treatment and for an additional 6 months after the last dose of investigational product.\n- 232\tParticipants unwilling to abstain from donating sperm during treatment and for an additional 6 months after the last dose of investigational product.\n- 233\tParticipant has known sensitivity to any of the products to be administered during dosing.\n- 234\tParticipant likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures (eg, Clinical Outcome Assessments) to the best of the participant’s and investigator’s knowledge.\n- 235\tHistory or evidence of any other clinically significant disorder, condition, or disease (except for those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to participant’s safety, or interfere with the study evaluation, procedures, or completion.\n- 211\tPositive test for chronic hepatitis B infection at screening, defined as either: (1) positive hepatitis B surface antigen (HBsAg); or (2) a positive hepatitis B core antibody (anti-HBc) PLUS negative hepatitis B surface antibody (anti-HBs). Note: Participants with a positive anti-HBs only, or a positive anti-HBc plus positive anti-HBs and negative HBsAg, are eligible to enroll.\n- 204\tSpontaneous or induced abortion, still or live birth, or pregnancy ≤ 4 weeks before screening.\n- 205\tEvidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality, including any of the following at screening (one repeat test may be conducted to confirm results within the same screening period): •\tplatelet count < 75000/μL (or < 75 × 109/L) •\tabsolute neutrophil count < 1200 cells/μL •\ttotal Ig < 600 mg/dL •\tCD4 T lymphocyte count < 300 cells/µL •\themoglobin < 8 g/dL (or < 80 g/L)\n- 206\tEstimated glomerular filtration rate < 45 mL/min/1.73 m2.\n- 207\tB-cell counts < one-half of the lower limit of normal (LLN) for age according to the central laboratory.\n- 208\tDiagnosed with a concurrent autoimmune disease that is uncontrolled or requires any prohibited medication (unless approved by the medical monitor).\n- 209\tClinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, hospitalization, or, in the investigator’s opinion, represents an additional risk to the participant, within 2 months before day 1 of study.\n- 212\tHistory of untreated hepatitis C infection, or positive antibody test for hepatitis C virus (HCV) unless the participant is considered to be cured following antiviral therapy and has an HCV viral load below the limit of detection at least 24 weeks after completion of treatment at site or central lab.\n- 213\tHistory of active or latent tuberculosis (TB), or a positive QuantiFERON-TB Gold test at screening, unless treatment for TB was completed per local guidelines.\n- 214\tNegative test for varicella zoster virus (VZV)-IgG.\n- 215\tParticipants with any of the following abnormal liver function tests in the absence of hepatobiliary IgG4-RD activity: •\tAST > 2 × ULN •\tALT > 2 × ULN •\tTBL > 2 × ULN unless AST, ALT, and hemoglobin are within central laboratory normal range and the participant has a known history of Gilbert syndrome\n- 216\tHistory of alcohol or drug abuse that, in the opinion of the Investigator, might affect participant safety or compliance with visits or interfere with safety or other study assessments\n- 217\tKnown positive anti-neutrophil cytoplasmic antibodies targeted against proteinase 3 or myeloperoxidase if done based on participant records."}

Primary endpoints

• {"endpoint_text":"- •\tPK parameters, including maximum plasma concentration (Cmax), area under the plasma concentration-time curve (AUC), terminal half-life (t½), clearance (CL), and volume of distribution at steady-state (Vss)","definition_or_measurement_approach":"Plasma pharmacokinetic measurements including Cmax, AUC, terminal half-life (t½), clearance (CL), and volume of distribution at steady-state (Vss) derived from plasma concentration-time data following IV administration."}
• {"endpoint_text":"- •\tChange from baseline in CD20+ B cell counts","definition_or_measurement_approach":"Peripheral blood CD20+ B cell counts measured at baseline and subsequent time points; endpoint is change from baseline."}
• {"endpoint_text":"- •\tIncidence of adverse events, serious adverse events, events of interest (EOIs), and change from baseline in laboratory parameters and vital signs","definition_or_measurement_approach":"Safety assessed by reporting of adverse events/serious adverse events/EOIs, and serial laboratory tests and vital signs compared to baseline."}

Secondary endpoints

• {"endpoint_text":"- •\tDisease activity endpoints include: - Time-to-first treated flare across 52 weeks - Proportion of flare-free participants across 52 weeks - Annualized flare rate across 52 weeks","definition_or_measurement_approach":"Disease activity measured by time-to-first treated flare within 52 weeks, proportion of participants without flares at 52 weeks, and annualized flare rate over 52 weeks."}
• {"endpoint_text":"- •\tPresence of antidrug antibodies (ADA) before and after initiation of treatment","definition_or_measurement_approach":"Immunogenicity assessed by detection of ADA in blood samples collected before and after treatment initiation."}
• {"endpoint_text":"- •\tPercent reduction from baseline in daily glucocorticoid dose at week 52 taken for the purpose of IgG4 RD control","definition_or_measurement_approach":"Change in daily glucocorticoid dose from baseline to week 52 expressed as percent reduction; measured by recorded medication dosing for IgG4-RD control."}

Greece

Earliest CTIS Part Ii Submission Date

20-11-2025

Latest Decision Or Authorization Date

25-02-2026

Processing Time Days

97

Number Of Sites

2

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

06-02-2026

Latest Decision Or Authorization Date

05-03-2026

Processing Time Days

27

Number Of Sites

1

Number Of Participants

1

Spain

Earliest CTIS Part Ii Submission Date

09-02-2026

Latest Decision Or Authorization Date

04-03-2026

Processing Time Days

23

Number Of Sites

1

Number Of Participants

1

Poland

Earliest CTIS Part Ii Submission Date

30-01-2026

Latest Decision Or Authorization Date

07-03-2026

Processing Time Days

36

Number Of Sites

3

Number Of Participants

3

Primary sponsor

Full Name

Amgen Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Excelya Greece CRO Single Member S.A.

Responsibilities

Submission activities in Greece; sponsorDuties codes: 1, 15; contact: info-greece@excelya.com

Name

Almac Clinical Technologies LLC

Responsibilities

sponsorDuties code: 3; contact: info@almacgroup.com

Name

PPD Development LP

Responsibilities

sponsorDuties code: 4; contact: CRGBioALogfile.sm@thermofisher.com

Third parties

• {"country":"United States","full_name":"Labcorp","duties_or_roles":"sponsorDuties codes: 4; contact: cliftob@labcorp.com","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Greece","full_name":"Excelya Greece CRO Single Member S.A.","duties_or_roles":"sponsorDuties codes: 1;15 - Submission activities in Greece; contact: info-greece@excelya.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"sponsorDuties codes: 3; contact: info@almacgroup.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"sponsorDuties codes: 4; contact: ctasubmissions@labcorp.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 4; contact: CRGBioALogfile.sm@thermofisher.com","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"sponsorDuties codes: 4; contact: ctasubmissions@labcorp.com","organisation_type":"Pharmaceutical company"}