CD19 CAR-T cells for hard-to-treat inflammatory myopathy and ANCA-associated vasculitis

Trial details

Who runs it

Peking University People's Hospital

Trial code

NCT06986018

Record updated

22 May 2025

Original description

This is an open-label, investigator-initiated clinical trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with refractory IIM and AAV. The study plans to enroll a total of 12 participants, with 6 cases each for IIM and AAV. Enrollment for both diseases will proceed in parallel. The dose will be 6×10\^6 CAR+T cells/kg (±30%), and patients will receive a single infusion of RD06-04.Read full original descriptionShow less

Clinical Study on the Safety, Efficacy, and Pharmacokinetics of a Universal CD19-Targeted CAR-T Cell Injection (RD06-04) in the Treatment of Patients With Refractory Inflammatory Myopathy and ANCA-Associated Vasculitis

This is an open-label, investigator-initiated clinical trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with refractory IIM and AAV. The study plans to enroll a total of 12 participants, with 6 cases each for IIM and AAV. Enrollment for both diseases will proceed in parallel. The dose will be 6×10\^6 CAR+T cells/kg (±30%), and patients will receive a single infusion of RD06-04.

Exclusion: Clinically significant central nervous system disease or pathological changes not caused by the non-study disease within 12 months prior to screening; History of drug or alcohol abuse within 1 year prior to screening; As determined by the investigator, the primary diagnosis is a rheumatic autoimmune disease other than the disease under study, which the investigator believes may confound the efficacy evaluation of the study disease; History of allogeneic bone marrow or stem cell transplantation or solid organ transplantation (such as kidney, lung, heart, liver) or plans for such transplantation in the future; History of, or current significant cardiovascular dysfunction; History of malignancy within 5 years prior to signing the ICF; History of recurrent infections requiring hospitalization and intravenous antibiotics (e.g., three or more episodes of the same type of infection within the past year); Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA in peripheral blood; positive for hepatitis C virus (HCV) antibody with detectable HCV RNA in peripheral blood; positive for human immunodeficiency virus (HIV) antibody; positive for syphilis antibody; Any condition that, in the investigator's opinion, may affect study participation, pose a safety risk to the patient, or potentially confound the interpretation of study results; For IIM patients: Presence of severe rhabdomyolysis or CK levels ≥120×ULN at screening; Pregnant or breastfeeding women.

General Inclusion Criteria:; The subject voluntarily participates in this trial and has signed the informed consent form; Age ≥18 years and ≤70 years, regardless of gender; Organ Function and Laboratory Tests:; Liver Function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3× upper limit of normal (ULN), total bilirubin (TBIL) ≤2×ULN (except for Gilbert syndrome); Renal Function: Creatinine ≤1.5×ULN or creatinine clearance ≥40 ml/min; Blood Routine: Neutrophil count ≥1×10\^9/L, hemoglobin ≥60 g/L, platelet count ≥50×10\^9/L, lymphocyte count >0.3×10\^9/L; Coagulation Function: International normalized ratio (INR) ≤1.5×ULN, or prothrombin time (PT) ≤1.5×ULN; Oxygen saturation (SpO2) ≥92% at rest while breathing room air; Echocardiography shows left ventricular ejection fraction (LVEF) ≥50%; Female subjects of childbearing potential must have a negative serum or urine pregnancy test result during screening; Females of childbearing potential must agree to use highly effective contraception from at least 28 days before the start of lymphodepletion until 12 months after the infusion of RD06-04. Males of reproductive potential must agree to use an effective barrier method of contraception from the start of lymphodepletion until 12 months after the infusion of RD06-04 and must not donate semen or sperm during the entire trial period; For IIM participants:; Diagnosed with IIM (including probable or definite diagnosis, i.e., a probability of ≥55%) according to the 2017 ACR/EULAR classification criteria. Currently, the ENMC considers that the subtypes of IIM mainly include dermatomyositis (DM), antisynthetase syndrome (ASS), and immune-mediated necrotizing myopathy (IMNM); For AAV participants:; Meets the diagnostic criteria for ANCA-associated vasculitis as established by the 2022 ACR/EULAR, including microscopic polyangiitis (MPA), granulomatosis with polyangiitis (GPA), and eosinophilic granulomatosis with polyangiitis (EGPA).

ClinicalTrials.gov NCT06986018