An Exploratory Study of CD19/CD22/BCMA CAR-T Cells (BZE2204) in Subjects With Relapsed or Refractory Autoimmune Diseases

NCT07174843 | Recruiting Early Phase 1

• 1 other identifier: BZE2204-C-01
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single arm, open-label, dose escalation and expansion study to evaluate the safety, tolerability and preliminary efficacy of autologous chimeric antigen receptor T (CAR-T) cells targeting CD19/CD22/BCMA(BZE2204) in patients with relapsed or refractory active autoimmune diseases, including idiopathic inflammatory myopathies(IIM), immune thrombocytopenia(ITP), systemic lupus erythematosus(SLE).

Conditions

Idiopathic Inflammatory Myopathies(IIM); Immune Thrombocytopenia(ITP); Systemic Lupus Erythematosus(SLE)

Keywords

Autoimmune diseases; SLE; IIM; ITP; CAR-T; tri-targets

Outcome Measures

Primary Outcomes (2)

• The frequency and severity of adverse events(AE) and serious adverse events(SAE)

  From leukapheresis to 6 months post CAR-T infusion

• The frequency of dose-limiting toxicity(DLT)

  Day0 to Day28

Secondary Outcomes (12)

• Chimeric antigen receptor T cell (CAR-T) levels in peripheral blood at each time point.

  Day0-Month6 post infusion

• Chimeric antigen receptor transgene levels in peripheral blood at each time point

  Day0-Month6 post infusion

• Cytokines levels of IL-2(interleukin), IL-4, IL-6,IL-8, IL-10, IL-12p70, IL-13, IL-1β, TNF-a, IFN-r in peripheral blood at each time point.

  Day-3 to Month6 post infusion

• B cell levels in peripheral blood at each time point

  Day-3 to Month6 post infusion

• Immunoglobulins in peripheral blood at each time point

  Day-3 to Month6 post infusion

Study Arms (1)

BZE2204 CAR-T cell therapy

EXPERIMENTAL

Biological: CD19/CD22/BCMA CAR-T cells(BZE2204)

Interventions

CD19/CD22/BCMA CAR-T cells(BZE2204) BIOLOGICAL

Subjects will undergo leukapheresis to isolate peripheral blood mononuclear cells (PBMCs) for the production of BZE2204 CAR T cells. On day0 subjects will receive a single dose BZE2204 CAR T cells by intravenous (IV) injection.

BZE2204 CAR-T cell therapy

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females, aged 18-70 years old
• Adequate bone marrow, hepatic, renal, coagulation and pulmonary function defined as:
• Bone marrow reservation: absolute neutrophil count (ANC) ≥1 ×10\^9/L; absolute lymphocyte count (ALC)≥ 0.5 ×10\^9/L; hemoglobulin ≥80 g/L; platelets ≥50 ×10\^9/L(except for ITP);
• Hepatic function: i: Serum alanine aminotransferase (ALT)/aspartate aminotransferase (AST) ≤ 5 upper limit of normal(ULN) (except for elevations are evaluated to be related to autoimmune disease by investigators)and ii: total bilirubin ≤ 2 ULN, (except for Gilbert's syndrome patients, those with total bilirubin ≤ 3 ULN and direct bilirubin ≤ 1.5 ULN can be enrolled).
• Renal function: serum creatinine ≤ 1.5 ULN , or estimated glomerular filtration rate(eGFR) ≥ 60 mL/min/1.73m2 \[eGFR=186×age\^-0.203×SCr\^-1.154（mg/dl）,female×0.742\]
• Coagulation function: International normalized ratio (INR) or prothrombin time (PT) ≤1.5 ULN
• Pulmonary function: Have the minimum level of pulmonary reserve, defined as ≤ CTCAE (Common Terminology Criteria for Adverse Events) grade 1 dyspnea and the SaO2(oxygen saturation)≥ 91% on room air
• Life expectancy \> 6 months
• Subjects with relapsed or refractory active IIM also need meet following criteria:
• Subjects with suspected or confirmed dermatomyositis(DM), polymyositis(PM), anti-synthetase syndrome(ASS) and immune-mediated necrotizing myopathy(IMNM, need to be assessed by the investigator that the patient has no safety instability) based on the 2017 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria
• Positive (+ or above) for at least one myositis-specific antibody (MSA) or myositis-associated antibody (MAA), including anti-TIF-1γ, NXP-2, Mi-2α, Mi-2β, MDA-5, SAE-1/2, SRP, HMGCR, Jo-1, PL-7, PL-12, HA, EJ, OJ, KS, Zo, Tyr, PM-Scl100, PM-Scl75, SSA/Ro-52, SSB/LA, Ku, RNA-PIII, cN1A, etc
• At screening, the subject must have moderate to severe IIM, defined as manual muscle testing (MMT) ≤ 141 and 2 of the following criteria are met; or CT suggests active interstitial lung disease(ILD)
• Physician global activity assessment (PGA) ≥ 2 cm (Visual analogue scale VAS 10 cm);
• Patient global activity assessment (PtGA) ≥ 2 cm ( VAS 10 cm scale);
• Extramuscular global assessment (Myositis Disease Activity Assessment Tool \[MDAAT\]) ≥ 2.0 cm (VAS 10 cm scale);

You may not qualify if:

• A history of severe hypersensitivity or allergic reactions, or contraindications or hypersensitivity to any component of the investigational drug
• Presence of any serious heart diseases defined in the protocol
• A medical history of severe central nervous system or symptoms within 6 months
• Any concurrent malignancy or a history of malignancy with exceptions indicated in the protocol
• Clinically significant hemorrhage symptoms or definite bleeding tendencies (except for events caused by ITP) within 6 months prior to screening; arteriovenous thrombosis events within 6 months prior to screening
• Any positive results of contagious diseases as following:
• Human immunodeficiency virus (HIV) antibody positive;
• HBsAg positive; or HBcAb/HBeAb positive (subjects with HBV DNA copy numbers below the lower limit of detection can be enrolled);
• hepatitis C antibody (HCV-Ab) positive (the subjects with HCV RNA below the lower limit of detection can be enrolled) or a known medical history of hepatitis C;
• Treponema pallidum antibody (TP-Ab) positive
• Epstein-Barr virus(EBV), cytomegalovirus(CMV) antibody positive and copy number is above the limit
• Active tuberculosis or latent tuberculosis that has not been adequately treated
• Evidenced viral, bacterial or fungal infection that is uncontrolled or requires systemic antimicrobial therapy
• Requirements of wash-out period for specific treatment are not met(detailed in protocol)
• Subjects with relapsed or refractory active IIM will be excluded in the following situations:

Sponsors & Collaborators

• Shanghai Cell Therapy Group Co.,Ltd: lead

Study Sites (1)

Shanghai Mengchao Cancer Hospital

Shanghai, Shanghai Municipality, 200240, China

RECRUITING

MeSH Terms

Conditions

Myositis; Purpura, Thrombocytopenic, Idiopathic; Lupus Erythematosus, Systemic; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Central Study Contacts

Jinxing Lou

CONTACT

021-67091399; loujx@shcell.com

Study Design

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

Study Record Dates

• First Submitted: August 19, 2025
• First Posted: September 16, 2025
• Study Start: September 1, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: September 16, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)