Clinical Study of BCT301 Cell Injection Therapy for Refractory Autoimmune Diseases

NCT07301164 | Not Yet Recruiting Early Phase 1

• 1 other identifier: BCT301
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.

Conditions

Systemic Sclerosis (SSc); Inflammatory Myositis; Antiphospholipid Syndrome; ANCA Associated Vasculitis; Sjogren Syndrome; System Lupus Erythematosus

Outcome Measures

Primary Outcomes (1)

• Dose-limiting toxicities of BCT301

  Adverse events occurring within 28 days post-infusion that were possibly, probably, or definitely related to BCT301 cell injection (per NCI-CTCAE v5.0).

  28 days

Secondary Outcomes (25)

• Efficacy of refractory systemic lupus erythematosus（SLE）- The change of SLEDAI-2K

  90, 180, 360 days

• Efficacy of refractory SLE-The change of PGA

  90, 180, 360 days

• Efficacy of refractory SLE-The change of SF36

  90, 180, 360 days

• Efficacy of refractory SLE-The change of antibodies

  90, 180, 360 days

• Efficacy of refractory systemic sclerosis (SSc)-The change of mRSS

  90, 180, 360 days

Study Arms (1)

BCT301

EXPERIMENTAL

Biological: BCT301

Interventions

BCT301 BIOLOGICAL

BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells.Participants will receive a single infusion of BCT301 cell injection at escalating dose levels.Prior to infusion, patients undergo a lymphodepleting conditioning regimen with fludarabine and cyclophosphamide given intravenously for three consecutive days.

BCT301

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Voluntarily sign the informed consent form.
• Male or female, aged 18-80 years (inclusive), with a body weight ≥40 kg.
• Female participants of childbearing potential and male participants with female partners of childbearing potential must use medically approved contraceptive methods or practice abstinence during the treatment period and for at least 6 months after the end of the treatment. Female participants of childbearing potential must have a negative serum human chorionic gonadotropin (HCG) test within 7 days prior to enrollment and must not be breastfeeding.
• Participants currently receiving one or more of the following treatments at stable doses: glucocorticoids, antimalarials, immunosuppressants:
• If the participant is receiving glucocorticoid therapy, the following conditions must be met: the maximum dose at screening and during the screening period is 30 mg/day of prednisone (or equivalent). The dose must have been stable for ≥7 days prior to screening, and adjustments during the screening period must not exceed 5 mg/day of prednisone (or equivalent);
• If the participant is receiving antimalarials and/or conventional immunosuppressants: the treatment must have been initiated ≥12 weeks prior to screening. The dose must have been stable for ≥8 weeks prior to screening and remain stable during the screening period;
• If biological agents (belimumab, telitacicept, rituximab, etc.) were used prior to the screening period, a washout period of at least 5 half-lives must be completed before screening.
• Peripheral blood B cells must show positive CD19 expression as detected by flow cytometry.
• \. Systemic Lupus Erythematosus (SLE)
• Meet the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria for SLE.
• Have moderate to severe disease activity at screening, with a Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2000) score \>6.
• Have inadequate response to conventional therapy or experience disease relapse after remission. Conventional therapy includes glucocorticoids (at full or pulse doses), two or more immunosuppressants, or biologic agents for at least 6 months, including cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, azathioprine, leflunomide, methotrexate, belimumab, telitacicept, and rituximab.
• Have positive serological autoantibody tests: positive antinuclear antibodies (ANAs) and/or anti-ds-DNA antibodies and/or anti-Sm antibodies.
• \. Systemic Sclerosis (SSc)
• Meet the 2013 EULAR/ACR classification criteria for SSc.

You may not qualify if:

• Study participants who meet any of the following criteria will be excluded from the study:
• Any medical condition that, in the opinion of the investigator, would contraindicate participation in the study, such as a life-threatening illness.
• Decreased organ function reserve not attributable to the primary disease:
• a) Neutrophil count \<1×10⁹/L; lymphocyte count \<0.3×10⁹/L; hemoglobin \<70 g/L; platelet count \<50×10⁹/L; b) Alanine aminotransferase (ALT) \>3 × upper limit of normal (ULN); aspartate aminotransferase (AST) \>3 × ULN; total bilirubin \>2 × ULN; c) Creatinine clearance \<40 mL/min; estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m²; or serum creatinine \>2.5 mg/dL; d) Left ventricular ejection fraction (LVEF) \<45% as measured by echocardiography; e) Oxygen saturation \<92% on room air.
• History of alcohol or substance abuse within the past 24 weeks.
• History of malignancy other than B-cell lymphoma.
• Presence of infections including human immunodeficiency virus (HIV), hypogammaglobulinemia, T-cell deficiency, syphilis, chronic hepatitis B or C, or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
• Known active tuberculosis (TB) infection or active bacterial infection.
• History of myocardial infarction, coronary angioplasty or stenting, unstable angina, clinically significant arrhythmia, or other clinically significant cardiac disease within 6 months prior to screening.
• Symptomatic deep vein thrombosis or pulmonary embolism within 6 months prior to screening, except in cases of antiphospholipid syndrome (APS).
• History of severe allergic reaction to any component of cellular therapy or other immunotherapeutic agents.
• Prior organ transplant requiring ongoing immunosuppressive therapy.
• Concurrent participation in another clinical trial that may interfere with disease assessment or study treatment.
• Prior treatment with CD19- and/or BCMA-targeted therapy or any CAR-T cell product; except in cases where prior therapy is deemed to have clearly failed (e.g., no response, short duration of response, or disease progression) as assessed by the investigator, the current disease state warrants the study treatment, and there is no clear evidence that toxicity from prior therapy would compromise the safety of the current study.
• Severe psychiatric disorder or significant cognitive impairment.

Sponsors & Collaborators

• Peking University Third Hospital: lead

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Scleroderma, Systemic; Antiphospholipid Syndrome; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Sjogren's Syndrome

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Xerostomia; Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Dry Eye Syndromes; Lacrimal Apparatus Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: December 24, 2025
• Study Start: December 11, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: December 24, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share