the Safety and Efficacy of Targeted BCMA In Vivo LV Injection for Recurrent or Refractory Autoimmune Diseases

NCT07339540 | Recruiting Early Phase 1

• 1 other identifier: PRG2503E2
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

This study is designed as a single arm, open label, single center clinical trial to evaluate the safety, tolerability, efficacy, pharmacokinetic or pharmacodynamic characteristics of the investigational drug V001-BCMA in autoimmune disease.

Conditions

Recurrent or Refractory IgG4 Related Diseases; Recurrent or Refractory Systemic Sclerosis; Recurrent or Refractory ANCA Associated Vasculitis; Recurrent or Refractory Systemic Lupus Erythematosus; Recurrent or Refractory Idiopathic Inflammatory Myopathy

Outcome Measures

Primary Outcomes (1)

• The incidence of adverse events of V001-BCMA single infusion.

  For all participants

  At Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

Secondary Outcomes (53)

• BILAG-2004 Scale Score

  At baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

• PGA

  At baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

• SF-36

  At baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

• DORIS score

  At Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

• LLDAS

  At Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

Other Outcomes (3)

• Virus Leak Detection

  baseline、Day2、Day4、Day6、Day8、Day10、Day14、Day21、Day28

• immunogenicity

  baseline、Day28、Month3，Month6，Month9，Month12，Month18，Month24 post infusion

• Single-cell sequencing

  During the screening period, at either 3 months or 6 months, the specific timing of the test will be determined by the researcher

Study Arms (1)

V001-BCMA injection

EXPERIMENTAL

Drug: V001-BCMA

Interventions

V001-BCMA DRUG

Targeted BCMA In-vivo LV Injection (Code: V001-BCMA) is a third-generation non-replicating self-inactivating lentiviral vector. Its envelope protein has been engineered to express targeting molecules on the lentiviral surface for specific recognition of T cells, while its nucleic acid contains a T cell-specific promoter and a CAR gene. After specifically targeting and binding to T cells, V001-BCMA enables the expression of CAR on the surface of T cells, forming CAR-T cells. These CAR-T cells can then specifically kill target cells.

V001-BCMA injection

Eligibility Criteria

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

You may qualify if:

• \. The age at the time of signing the informed consent form is ≥18 years old and ≤65 years old;
• \. For cohort 1: recurrent or refractory systemic lupus erythematosus (all of the following four items must be met simultaneously)
• Diagnosed with SLE according to the 2012 SLICC or 2019 EULAR/ACR revised criteria.
• During screening, the patient exhibits positive anti-nuclear antibodies, and/or positive anti-ds-DNA antibodies, and/or positive anti-Smith antibodies.
• Before screening, patients must have received treatment with glucocorticoids combined with immunosuppressants and/or biologics for at least 3 months, with a stable dose for more than 2 weeks, and the disease remains active or the patient is intolerant to the medication.
• During the screening period, the SLEDAI-2K score is ≥8 points
• \. For cohort 2: recurrent or refractory IgG4-related disease (all three of the following criteria must be met simultaneously)
• Meet the American College of Rheumatology (ACR)/EULAR 2019 classification criteria for IgG4-RD.
• Patients with clinical manifestations of recurrent or refractory IgG4-RD and ineffective conventional treatment
• Meet the clinical phenotype of "Mikulicz-system involvement"
• \. For cohort 3: relapsed or refractory systemic sclerosis (all of the following 5 items must be met simultaneously)
• Meet the 2013 American College of Rheumatology (ACR) and European League Against Rheumatism (EULAR) classification criteria for SSc.
• Anti-Scl 70 antibody positive, or anti-centromere protein antibody positive, or anti-RNA polymerase III antibody positive
• Patients with a modified Rodnan skin score (mRSS) of ≥10 at screening
• Before screening, patients must have received treatment with glucocorticoids combined with immunosuppressants and/or biologics for at least 3 months, with a stable dose for more than 2 weeks, and the disease remains active or intolerable.

You may not qualify if:

• \. For cohort 1: relapsed or refractory systemic lupus erythematosus
• Subjects with uncontrolled lupus crisis within 8 weeks before screening were assessed by the investigator as unsuitable for participation in this study.
• Before screening, patients with clinically significant central nervous system diseases or pathological changes not caused by lupus, including but not limited to cerebrovascular accident, aneurysm, epilepsy, convulsion/convulsion, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disease, brain organic syndrome, or mental illness, should be excluded.
• \. For cohort 3: relapsed or refractory systemic sclerosis. High-risk pulmonary arterial hypertension, according to the "Risk Stratification of Arterial Pulmonary Arterial Hypertension (PAH)" in the "Guidelines for the Diagnosis and Treatment of Pulmonary Arterial Hypertension in China (2021 Edition)".
• \. History of major organ transplantation (such as heart, lung, kidney, liver) or hematopoietic stem cell/bone marrow transplantation.
• \. Subjects with a history of ≥ Grade 2 bleeding within 30 days prior to screening, as assessed by the investigator, were deemed unsuitable for enrollment.
• \. Use of any live vaccines against infectious diseases within 8 weeks before infusion.
• \. Received any treatment using vesicular stomatitis virus G (VSVG) pseudotype virus.
• \. The subject has a history or evidence of suicidal thoughts within 6 months before signing the ICF, or any suicidal behavior within 12 months before signing the ICF, and the researcher believes that there is a significant risk of suicide.
• \. Pregnant or lactating women;
• \. The patient has a history of severe and/or uncontrolled liver, gastrointestinal, kidney, lung, cardiovascular, psychiatric, neurological, or musculoskeletal diseases, hypertension, or any other medical condition that, in the opinion of the investigator, may affect the integrity of the patient's participation in the study, or may endanger the safety of the subject or affect the validity of the study results.
• \. Suffered from malignant tumor within 3 years before screening, except for the following conditions: received radical treatment for malignant tumor and had no known active disease within ≥3 years before enrollment; or had fully treated non-melanoma skin cancer and currently had no evidence of disease;
• \. Received any B-cell depleting biologic therapy (e.g., rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab, etc.) within 3 months prior to infusion, unless B-cell recovery is proven.
• \. Received immunosuppressants and other small molecule drugs within 3 days before infusion.
• \. Use of any other clinical research drugs within 4 weeks before infusion. However, if the study treatment is ineffective or the disease progresses during the study period, and at least 3 half-lives have elapsed before screening, enrollment is allowed.

Sponsors & Collaborators

• Tongji Hospital: lead

Study Sites (1)

Tongji Hospital, Affiliated to Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, 430000, China

RECRUITING

MeSH Terms

Conditions

Recurrence; Lupus Erythematosus, Systemic; Immunoglobulin G4-Related Disease; Scleroderma, Systemic; Myositis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular

Central Study Contacts

lingli Dong, professor

CONTACT

83665519; tjhdongll@163.com

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: professor

Study Record Dates

• First Submitted: November 26, 2025
• First Posted: January 14, 2026
• Study Start: November 4, 2025
• Primary Completion (Estimated): October 30, 2028
• Study Completion (Estimated): October 30, 2028
• Last Updated: January 14, 2026

Record last verified: 2026-01

Locations

CN (1)