Actively Recruiting

Early Phase 1
Age: 18Years - 65Years
All Genders
NCT07339540

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

Led by Tongji Hospital · Updated on 2026-01-14

45

Participants Needed

1

Research Sites

155 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 18Years - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age between 18 and 65 years old at the time of signing the consent form
  • For systemic lupus erythematosus (SLE) cohort: diagnosed by 2012 SLICC or 2019 EULAR/ACR criteria; positive for specific antibodies during screening; treated with glucocorticoids plus immunosuppressants or biologics for at least 3 months with stable dose over 2 weeks; active disease with SLEDAI-2K score of 8 or higher
  • For IgG4-related disease cohort: meet 2019 ACR/EULAR criteria; have recurrent or refractory disease with ineffective conventional treatment; show "Mikulicz-system involvement"
  • For systemic sclerosis cohort: meet 2013 ACR/EULAR criteria; positive for specific antibodies; modified Rodnan skin score of 10 or higher; treated with glucocorticoids plus immunosuppressants or biologics for at least 3 months with stable dose over 2 weeks; active or intolerant disease
  • For idiopathic inflammatory myopathies cohort: meet 2017 EULAR/ACR criteria; positive for at least one myositis-specific or associated antibody; meet at least two clinical or laboratory criteria during screening; treated with glucocorticoids plus immunosuppressants or biologics for at least 3 months with stable dose over 2 weeks; active myositis shown by biopsy or MRI within 6 months
  • For ANCA-associated vasculitis cohort: diagnosed by 2022 ACR/EULAR criteria; treated with glucocorticoids plus immunosuppressants or biologics with stable dose over 2 weeks; positive for related antibodies; active severe disease with BVAS score of 3 or higher
  • Have sufficient organ function
  • Agree to use effective contraception from signing consent until 1 year after study drug administration if of childbearing potential
  • Agree to participate and sign informed consent
Not Eligible

You will not qualify if you...

  • For SLE cohort: uncontrolled lupus crisis within 8 weeks before screening; clinically significant central nervous system diseases unrelated to lupus
  • For systemic sclerosis cohort: high-risk pulmonary arterial hypertension
  • History of major organ or bone marrow transplantation
  • History of grade 2 or higher bleeding within 30 days before screening
  • Use of live vaccines within 8 weeks before infusion
  • Previous treatment using vesicular stomatitis virus G pseudotype virus
  • History or evidence of suicidal thoughts or behavior within 6 to 12 months before consent
  • Pregnant or lactating women
  • Severe or uncontrolled diseases affecting liver, gastrointestinal, kidney, lung, heart, psychiatric, neurological, or musculoskeletal systems; hypertension; or other conditions affecting safety or study integrity
  • Malignant tumor within 3 years before screening except fully treated non-melanoma skin cancer or tumors with no active disease for 3 years
  • Use of B-cell depleting biologic therapy within 3 months before infusion unless B-cell recovery proven
  • Use of immunosuppressants or small molecule drugs within 3 days before infusion
  • Use of other clinical research drugs within 4 weeks before infusion unless prior treatment ineffective or disease progressed and at least 3 half-lives elapsed
  • Use of live vaccines or live therapeutic infectious agents within 2 weeks before infusion
  • Chronic and active infections including hepatitis B, hepatitis C, HIV, or syphilis
  • Active infection requiring intravenous antibiotics or hospitalization
  • Major surgery within 4 weeks before infusion or expected during study except minor local anesthesia procedures
  • Significant cardiovascular disease including high NT-proBNP, advanced heart failure, recent unstable angina or myocardial infarction, recent coronary interventions
  • Known allergies or severe reactions to any component of V001-BCMA
  • Deemed unsuitable for infusion or study participation by researchers

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

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

Wuhan, Hubei, China, 430000

Actively Recruiting

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Research Team

L

lingli Dong, professor

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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