Clinical Study on the Targeted CD19 Universal CAR-T Cell Injection (RD06-04) for the Treatment of IIM and AAV

NCT06986018 | Not Yet Recruiting Early Phase 1

• 1 other identifier: BHCT-RD06-04-08
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Idiopathic Inflammatory Myopathies; ANCA-Associated Vasculitis

Keywords

CD19-Targeted Universal CAR-T

Outcome Measures

Primary Outcomes (1)

• Adverse Events (TEAE), Serious Adverse Events (SAE), Adverse Events of Special Interest (AESI)

  Up to 2 years

Secondary Outcomes (9)

• The proportion of patients with kidney involvement achieving complete kidney remission (CRR)

  At weeks 12, and months 6, 12, 18, and 24 following CAR-T infusion.

• The change in UPCR from baseline in patients with kidney involvement.

  At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.

• The change in eGFR (estimated glomerular filtration rate) from baseline in patients with kidney involvement.

  At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.

• The change in BVAS score from baseline in AAV patients

  At 6, 12, 18, and 24 months following CAR-T infusion.

• IIM patients were assessed for major clinical remission (Total Improvement Score, TIS) according to the 2016 ACR /EULAR criteria for myocarditis remission.

  At 6, 12, 18, and 24 months following CAR-T infusion.

Study Arms (1)

RD06-04 Cell Injection

EXPERIMENTAL

Drug: RD06-04 Cell Injection Infusion

Interventions

RD06-04 Cell Injection Infusion DRUG

CAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.

RD06-04 Cell Injection

Eligibility Criteria

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

You may qualify if:

• 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).

You may not qualify if:

• 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.
• Clinically significant central nervous system disease or pathological changes not caused by the non-study disease within 12 months prior to screening.
• 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.
• For IIM patients: Presence of severe rhabdomyolysis or CK levels ≥120×ULN at screening.
• History of, or current significant cardiovascular dysfunction.
• History of malignancy within 5 years prior to signing the ICF.
• Pregnant or breastfeeding women.
• 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.
• History of drug or alcohol abuse within 1 year prior to screening.
• 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.

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University People's Hospital

Beijing, 100032, China

Location

MeSH Terms

Conditions

Myositis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

ZhanGuo Li, Doctor

CONTACT

010-88324073; 1i99@bjmu.edu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of the Rheumatology and Immunology Department

Study Record Dates

• First Submitted: April 13, 2025
• First Posted: May 22, 2025
• Study Start: June 14, 2025
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2026
• Last Updated: May 22, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)