ClinicalTrials.gov record NCT07222553
Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Inebilizumab in Pediatric Participants With IgG4-RD
Open-label, Uncontrolled, Multicenter Trial to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Inebilizumab in Children From 2 Years to Less Than 18 Years of Age With Immunoglobulin G4-related Disease (IgG4-RD)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is an open-label, single-group Phase 2 trial of intravenous inebilizumab in children with immunoglobulin G4–related disease (IgG4-RD). The study will collect drug level and immune-response data and monitor safety and tolerability, with measurements reported up to Day 561 and clinical flare outcomes up to Week 52. It enrolls pediatric participants (registered record lists ages 2 to <18 years).
What it is studying. The study aims to characterize how inebilizumab behaves in children’s bodies (pharmacokinetics), how it affects immune cells (pharmacodynamics), and to assess safety and tolerability when given by IV to pediatric participants with IgG4-RD. It will also record disease flares, antidrug antibodies, and changes in glucocorticoid use over the study period.
What the researchers hope to learn. To provide data on inebilizumab pharmacokinetics and pharmacodynamics in children and to assess its safety and tolerability; secondary goals include measuring flares, antidrug antibodies, and steroid-dose changes.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD), as well as to assess the safety and tolerability, of inebilizumab in pediatric participants with IgG4-RD.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Inebilizumab
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Participants must weigh ≥ 17 kg to be eligible for enrollment.
- Participant 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.
- Age 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.
- Clinical diagnosis of IgG4-RD.
- Fulfillment 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.
- Receipt of all age-appropriate and locally-required vaccinations before screening.
- Participants requiring treatment in addition to or other than glucocorticoids (GCs) for IgG4-RD according to PI's assessment at screening.
- Participants 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.
- Key
Exclusion criteria
- Participants with any of the following abnormal liver function tests in the presence of hepatobiliary IgG4-RD activity:
- aspartate aminotransferase (AST) > 10 × upper limit of normal (ULN)
- alanine aminotransferase (ALT) > 10 × ULN
- total 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 GC treatment.
- Evidence 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):
- platelet count < 75000/μL (or < 75 × 109/L)
- absolute neutrophil count < 1200 cells/μL
- total Ig < 600 mg/dL
- CD4 T lymphocyte count < 300 cells/µL
- hemoglobin < 8 g/dL (or < 80 g/L).
- Estimated glomerular filtration rate < 45 mL/min/1.73 m\^2.
- B-cell counts < one-half of the lower limit of normal (LLN) for age according to the central laboratory.
- Diagnosed with a concurrent autoimmune disease that is uncontrolled or requires any prohibited medication (unless approved by the medical monitor).
- Clinically 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.
- Known 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.
- Positive test for chronic hepatitis B infection at screening, defined as either: (1) Positive hepatitis B surface antigen (HBsAg); or (2) 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.
- Receipt of any of the following before Day 1: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy.
- Receipt of any of the following within 2 months before Day 1: azathioprine, mycophenolate mofetil, cyclosporine, methotrexate, cyclophosphamide, tocilizumab, satralizumab, eculizumab, and mitoxantrone.
- Receipt 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.
- Receipt 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.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will receive inebilizumab by intravenous (IV) infusion. They will attend regular clinic visits for blood tests to measure drug levels, B‑cell counts, antibodies to the drug, and safety labs, and for monitoring of disease flares and adverse events, with follow-up through Day 561. Glucocorticoid dosing may be adjusted by the investigator during the study. Age-appropriate vaccinations must be up to date before screening, and recent live vaccines or recent blood transfusions can disqualify participation.
How the study is structured. Interventional, open-label, single-group Phase 2 study: all participants receive IV inebilizumab, there is no placebo or randomization, and participants are followed for drug levels, immune measures, and safety (primary measures reported through Day 561).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in Brazil, China, Greece, Poland, Spain, Turkey, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on IgG4-related disease
Important dates
- Study start
- Oct 31, 2026 (estimated)
- Primary completion
- Jun 10, 2031 (estimated)
- Study completion
- Jun 10, 2031 (estimated)
- Target enrollment
- 15 participants (estimated)
- Last updated on registry
- Aug 27, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Amgen
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Amgen Call Center 📞 866-572-6436 ✉ medinfo@amgen.com
Participating sites (14)
- University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
Tampa, Florida, 33612 · United States
- University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514 · United States
- University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224 · United States
- Centro Oncologia Leonardo Da Vinci
Fortaleza, Ceará, 60140-025 · Brazil
- Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730 · China
- The Second Xiangya Hospital of Central South University
Changsha, Hunan, 410000 · China
- Euroclinic Children Hospital
Athens, 11521 · Greece
- General Children Hospital Panagioti and Aglaias Kyriakou
Athens, 11527 · Greece
- Narodowy Instytut Geriatrii Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher
Warsaw, 02-637 · Poland
- Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, 04-730 · Poland
Terms used on this page · explanations by MedicalUpdates.ai
- pharmacokinetics
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- pharmacodynamics
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- legally authorized representative
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- principal investigator
- The doctor or scientist responsible for running the study at a site or overall.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- stable dose
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- Estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- prohibited medication
- A medicine that participants may not use during the study, usually because it could interfere with the study treatment or its measurement.
- bone marrow transplant
- A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
- Key Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Key Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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