ClinicalTrials.gov record NCT07793903
BCT301 in Treating Refractory/Recurrent IgG4-related Disease
Clinical Study to Evaluate the Safety and Efficacy of BCT301 Cell Injection in Patients With Refractory/Recurrent IgG4-Related Disease
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
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 a single-arm, open-label study testing a single infusion of BCT301 cell injection in people with refractory or recurrent IgG4-related disease. Six participants will receive a lymphodepletion pretreatment followed by one infusion (1.8 × 10^8 cells) and be followed for safety and effects on disease activity. The main goal is to assess safety and tolerability; the study also measures disease activity, laboratory markers, pharmacokinetics/pharmacodynamics, and clinical response over time.
What it is studying. The study tests the safety and tolerability of a single infusion of BCT301 cell injection (CAR-iT cells from chemically induced pluripotent stem cells) in people with refractory/recurrent IgG4-related disease. It also measures changes in disease activity (IgG4-RD Responder Index), IgG4 blood levels, response and remission rates, relapse, physician and patient global assessments, and PK/PD characteristics.
What the researchers hope to learn. Scientifically, the study aims to evaluate the safety and tolerability of BCT301 cell injection in refractory/recurrent IgG4-related disease and to collect early data on clinical activity. Secondary goals include measuring changes in IgG4-RD Responder Index scores, response and complete remission rates at set time points, relapse rates, changes in serum IgG4 levels, physician and patient global assessments, and pharmacokinetic/pharmacodynamic characteristics.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is an investigator initiated trial to assess the safety and efficacy of BCT301 cell Injection (CAR-iT cells derived from chemically induced pluripotent stem cells) in patients with refractory/recurrent IgG4-related disease.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Other
- BCT301 Cell Injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Voluntarily participate in this trial and sign the informed consent form;
- Male or female patients aged 18 to 75 years (inclusive), with body weight ≥ 40 kg;
- Women of childbearing potential must have a negative serum pregnancy test before the start of the trial and agree to use effective contraception during the trial and until the last follow-up; male subjects whose partners are of childbearing potential agree to use effective contraception during the trial and until the last follow-up;
- Confirmed diagnosis of IgG4-related disease meeting the classification criteria for IgG4-RD (the 2020 updated version of the comprehensive diagnostic criteria for IgG4-RD established in Japan) or the 2019 ACR/EULAR classification criteria for IgG4-RD; and with at least one of the following organ involvements: pancreas, bile ducts, kidneys, lungs, heart or pericardium, aorta and great vessels, retroperitoneal fibrosis, sclerosing mediastinitis, dura mater, or pituitary gland;
- Clinically meets the criteria for refractory/recurrent IgG4-RD: disease remains active, or relapses after remission, or progresses despite systemic treatment with standard-of-care regimens-glucocorticoids, immunosuppressants (cyclophosphamide, mycophenolate mofetil, methotrexate, azathioprine, etc.)-or biologic agents;
- Currently receiving one or more of the following stable-dose standard therapies:
- If the patient is receiving glucocorticoid therapy, the following conditions must be met: at screening and during the screening period, the maximum glucocorticoid dose is 30 mg/day of prednisone (or equivalent). The glucocorticoid dose must remain stable for ≥ 7 days prior to screening; during the screening period, glucocorticoid dose adjustments must not exceed 5 mg/day of prednisone (or equivalent);
- If the patient is receiving immunomodulators/immunosuppressants: initiation of drug therapy must be ≥ 12 weeks prior to screening. A stable drug dose must be maintained for ≥ 4 weeks prior to screening and during the screening period;
- If biologic agents (e.g., belimumab or telitacicept) were used before the screening period, they must be discontinued for at least 5 half-lives before screening; if anti-CD20 monoclonal antibody therapy was used before the screening period, an interval of 6 months is required before screening;
- IgG4-RD Responder Index (RI) ≥ 2, with disease in an active state;
- Peripheral blood B cells show positive CD19 expression by flow cytometry;
- Major organ function must meet the following requirements (conditions caused by the immune disease itself are exempt):
- Bone marrow hematopoietic function must meet: a. White blood cell count ≥ 3 × 10\^9/L; b. Neutrophil count ≥ 1 × 10\^9/L (no colony-stimulating factor treatment within 2 weeks prior to the examination); c. Hemoglobin ≥ 70 g/L;
- Hepatic function: Alanine aminotransferase (ALT) ≤ 3 × upper limit of normal (ULN), aspartate aminotransferase (AST) ≤ 3 × ULN, total bilirubin (TBIL) ≤ 2 × ULN (excluding Gilbert syndrome, for which total bilirubin ≤ 3.0 × ULN);
- Renal function: Creatinine clearance ≥ 40 mL/min (Cockcroft-Gault formula);
- Coagulation function: International normalized ratio (INR) < 1.5 × ULN, prothrombin time (PT) < 1.5 × ULN;
- Cardiac function: Good hemodynamic stability.
Exclusion criteria
- The patient must not participate in this study if any of the following criteria are met:
- Diseases that, after evaluation by the investigator, are considered inappropriate for participation in this study, for example life-threatening conditions;
- Reduced organ functional reserve not caused by the primary disease:
- Neutrophil count < 1 × 10\^9/L; lymphocyte count < 0.3 × 10\^9/L; hemoglobin < 70 g/L; platelet count < 50 × 10\^9/L;
- ALT > 3 × ULN; AST > 3 × ULN; total bilirubin > 2 × ULN;
- Creatinine clearance < 40 mL/min, estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m², or serum creatinine > 2.5 mg/dL;
- Left ventricular ejection fraction ≤ 45% as diagnosed by echocardiography;
- Blood oxygen saturation ≤ 92%;
- History of alcohol abuse or drug abuse within the past 24 weeks;
- History of malignancy other than B-cell lymphoma within the past 5 years (excluding non-melanoma skin cancer, surgically cured cervical cancer, etc.);
- Presence of infection with human immunodeficiency virus (HIV), agammaglobulinemia, T-cell deficiency virus, syphilis, chronic hepatitis B or C, or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), etc.;
- Known active tuberculosis (TB) infection or bacterial infection;
- History of myocardial infarction, coronary angioplasty or stent placement, unstable angina, active arrhythmia, or other clinically significant cardiac disease requiring clinical intervention within 6 months before the start of screening;
- History of symptomatic deep vein thrombosis or pulmonary embolism within 6 months before the start of screening;
- Known severe allergic reactions to cell-therapy-related components or their excipients, or to other immunotherapeutic agents;
- Prior organ transplantation requiring long-term immunosuppressive medication;
- Known simultaneous participation in other clinical trials affecting the disease or treatment;
- Prior treatment with CD19- and/or BCMA-targeted therapy or any CAR-T cell product targeting any antigen; unless, as assessed by the investigator, the prior treatment has clearly failed (including failure to achieve remission after treatment, remission duration below the required standard, or disease progression), the current disease state is suitable for treatment in this study, and there is no clear evidence that toxicity related to prior treatment may affect the safety of this study;
- Severe psychiatric illness and severe cognitive impairment;
- Pregnant or lactating women, or women planning pregnancy;
- Any condition considered by the investigator to be inappropriate for enrollment in this clinical trial.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is a single-arm, open-label, single-center clinical study in which all patients receive a unified treatment regimen. The primary objective is to evaluate the safety and tolerability of BCT301 Cell Injection in patients with refractory/recurrent IgG4-RD. Secondary objective is to evaluate the improvement in disease activity in patients with refractory/recurrent IgG4-RD treated with BCT301 Cell Injection. Six subjects with refractory/recurrent IgG4-RD meeting the inclusion/exclusion criteria are planned to be enrolled and will receive a single infusion of 1.8 × 10\^8 BCT301 Cell Injection to evaluate the safety, efficacy, and PK/PD characteristics following BCT301 Cell Injection infusion. The study flow consists of a screening period, a lymphodepletion pretreatment period, a treatment period, and a follow-up period. Patients without relapse or deterioration after BCT301 Cell Injection infusion and not requiring protocol-prohibited treatment will undergo a primary follow-up of up to 360 days.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
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.
After a screening period, participants will undergo a short lymphodepletion pretreatment and then receive a single infusion of 1.8 × 10^8 BCT301 Cell Injection. They will be monitored closely for safety, with special attention to cell‑infusion‑related reactions such as cytokine release syndrome (CRS) and immune effector cell‑associated neurotoxicity syndrome (ICANS), especially during the first 28 days. Follow‑up visits continue for up to 360 days and include disease‑activity assessments using the IgG4‑Related Disease Responder Index, blood tests to measure IgG4 and PK/PD (pharmacokinetic/pharmacodynamic) characteristics, and physician and patient global assessments.
How the study is structured. All participants receive the same treatment in a single group (no placebo or comparison group). The sequence is screening, a lymphodepletion pretreatment period, one infusion of 1.8 × 10^8 BCT301 cells, then a follow-up period (primary follow-up up to 360 days if no relapse or prohibited treatment). Safety is closely monitored, with particular attention to infusion-related reactions within 28 days; disease activity and laboratory measures are assessed at prespecified times (for example Day 90, Day 180, Day 360).
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?
- No sites are listed on the registry yet. Where will the study take place?
- 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
- October 2026 (estimated)
- Primary completion
- June 2028 (estimated)
- Study completion
- February 2030 (estimated)
- Target enrollment
- 6 participants (estimated)
- Last updated on registry
- Aug 31, 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
- Peking University Third Hospital
- Principal investigator
- Mu Rong, Professor, Director of Department of Rheumatology & Immunology
Peking University Third Hospital
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.
- Jinxia Zhao, MD 📞 8610 82265699 ✉ zhao-jinxia@163.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- informed consent form
- 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.
- Women of childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- 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.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- 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.
- 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.
- Creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- 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.
- echocardiography
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- Prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- CAR-T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- 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.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- Secondary objective
- An additional result the study measures alongside the primary outcome, for example quality of life or a laboratory value.
- PK
- 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.
- relapse
- A condition that improved with treatment and has since returned or worsened.
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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