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Active, not recruitingNCT07339540Updated Sep 17, 2026

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

An Early Phase 1 interventional study of V001-BCMA in Recurrent or Refractory Systemic Lupus Erythematosus, Recurrent or Refractory IgG4 Related Diseases and Recurrent or Refractory Systemic Sclerosis, sponsored by Tongji Hospital. Active, not recruiting at 1 site in China. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-17.

Sponsored by Tongji Hospital · Early Phase 1, Interventional, and Treatment

Phase
Early Phase 1
Study type
Interventional
Enrollment
45
Allocation
Not applicable
Ages
18 Years to 65 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Recurrent or Refractory Systemic Lupus Erythematosus
  • Recurrent or Refractory IgG4 Related Diseases
  • Recurrent or Refractory Systemic Sclerosis
  • Recurrent or Refractory Idiopathic Inflammatory Myopathy
  • Recurrent or Refractory ANCA Associated Vasculitis
03

In context

Recurrence

4,278 studies on the registry are indexed under Recurrence; 987 are open to participants now.

This study's planned enrollment of 45 is close to the median of 50 across 3,373 interventional studies indexed under Recurrence.

Browse Recurrence studies →

Lead sponsor

Tongji Hospital is the lead sponsor of 373 studies on the registry; 204 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 1. The age at the time of signing the informed consent form is ≥18 years old and ≤65 years old;

  • 2. For cohort 1: recurrent or refractory systemic lupus erythematosus (all of the following four items must be met simultaneously)

    1. Diagnosed with SLE according to the 2012 SLICC or 2019 EULAR/ACR revised criteria.
    2. During screening, the patient exhibits positive anti-nuclear antibodies, and/or positive anti-ds-DNA antibodies, and/or positive anti-Smith antibodies.
    3. 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.
    4. During the screening period, the SLEDAI-2K score is ≥8 points

  • 3. For cohort 2: recurrent or refractory IgG4-related disease (all three of the following criteria must be met simultaneously)

    1. Meet the American College of Rheumatology (ACR)/EULAR 2019 classification criteria for IgG4-RD.
    2. Patients with clinical manifestations of recurrent or refractory IgG4-RD and ineffective conventional treatment
    3. Meet the clinical phenotype of "Mikulicz-system involvement"

  • 4. For cohort 3: relapsed or refractory systemic sclerosis (all of the following 5 items must be met simultaneously)

    1. Meet the 2013 American College of Rheumatology (ACR) and European League Against Rheumatism (EULAR) classification criteria for SSc.
    2. Anti-Scl 70 antibody positive, or anti-centromere protein antibody positive, or anti-RNA polymerase III antibody positive
    3. Patients with a modified Rodnan skin score (mRSS) of ≥10 at screening
    4. 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.

  • 5. For cohort 4: relapsed or refractory idiopathic inflammatory myopathies (all of the following 5 criteria must be met)

    1. Meet the 2017 EULAR/ACR classification criteria for inflammatory myopathies (including dermatomyositis DM, polymyositis PM, anti-synthetase syndrome ASS, and necrotizing myositis NM).
    2. At least one myositis-specific antibody (MSA) or myositis-associated antibody (MAA) positive (+ or above)
    3. During the screening period, the patient meets 2 of the following criteria: PGA(VAS)≥2cm (VAS-10cm scale); PtGA(VAS)≥2cm (VAS-10cm scale); HAQ>0.25; one or more muscle enzymes are elevated (CK, LDH, AST, ALT) ≥1.5×ULN; overall muscle extrinsic assessment (MDAAT) ≥2.0cm (VAS-10cm scale)
    4. After at least 3 months of treatment with glucocorticoids and immunosuppressants and/or biologics, with stable doses for more than 2 weeks, the disease remains active or the patient is intolerant to the medication.
    5. Active myositis is present in muscle biopsy or muscle MRI during the screening period or within the first 6 months before the screening period.

  • 6. For cohort 5: relapsed or refractory AAV (all three of the following conditions must be met simultaneously)

    1. According to the 2022 ACR/EULAR criteria, the patient is diagnosed with AAV (GPA or MPA subtype). According to the KDIGO guidelines, after 3 months of treatment with glucocorticoids combined with immunosuppressants such as cyclophosphamide or biologics such as rituximab, and the dose is stable for more than 2 weeks, the disease is still active or the patient is intolerant to the drug;
    2. The patient is currently or has been in the course of AAV related antibodies positive;
    3. Severe disease requiring treatment (BVAS score ≥3.0).
  • 7. Possess sufficient organ function
  • 8. Men with fertility and women of childbearing age must agree to use effective contraception from the time they sign the informed consent form until 1 year after the study drug is administered. Blood pregnancy tests for women of childbearing age must be negative at screening and before infusion;
  • 9. The subject or his/her guardian agrees to participate in this clinical study and signs the informed consent form (ICF), indicating that he/she understands the purpose and procedures of this clinical study and is willing to participate in the study.

Exclusion criteria

Exclusion Criteria:

  • 1. For cohort 1: relapsed or refractory systemic lupus erythematosus

    1. Subjects with uncontrolled lupus crisis within 8 weeks before screening were assessed by the investigator as unsuitable for participation in this study.
    2. 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.
  • 2. 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)".
  • 3. History of major organ transplantation (such as heart, lung, kidney, liver) or hematopoietic stem cell/bone marrow transplantation.
  • 4. Subjects with a history of ≥ Grade 2 bleeding within 30 days prior to screening, as assessed by the investigator, were deemed unsuitable for enrollment.
  • 5. Use of any live vaccines against infectious diseases within 8 weeks before infusion.
  • 6. Received any treatment using vesicular stomatitis virus G (VSVG) pseudotype virus.
  • 7. 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.
  • 8. Pregnant or lactating women;
  • 9. 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.
  • 10. 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;
  • 11. 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.
  • 12. Received immunosuppressants and other small molecule drugs within 3 days before infusion.
  • 13. 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.
  • 14. The patient has received live vaccines or live therapeutic infectious pathogens within 2 weeks before infusion.
  • 15. The presence of chronic and active hepatitis B (excluding HBV-DNA levels below 500IU/ml), hepatitis C (HCV), human immunodeficiency virus (HIV) infection, or syphilis infection;
  • 16. Active infection exists, requiring intravenous antibiotic therapy or hospitalization;
  • 17. Patients who have undergone major surgery other than diagnosis or biopsy within 4 weeks before infusion, or are expected to undergo major surgery during the study period; note: patients who plan to undergo surgical procedures under local anesthesia can participate in the study.

Kyphoplasty or vertebroplasty are not considered major surgery;

  • 18. Obvious evidence of cardiovascular disease as follows: a. N-terminal pro-B-type natriuretic peptide (NT-proBNP) > 8500ng/L; b. New York Heart Association (NYHA) classification of heart failure as III or IV; c. Patients who have received inpatient treatment for unstable angina or myocardial infarction within 6 months before the first dose, or who have received percutaneous coronary intervention and have received the latest stent placement within 6 months, or who have received coronary artery bypass grafting within 6 months;
  • 19. Individuals who have known allergies, hypersensitivity reactions, intolerances, or contraindications to any component of V001-BCMA, or who have previously experienced severe allergic reactions.
  • 20. Those who were deemed unsuitable for infusion or otherwise unsuitable for participation in the study by the researchers.
05

Study design

Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
45 participants (estimated)

Study arms

  • Experimental
    V001-BCMA injection

    Drug: V001-BCMA

Interventions

  • DrugV001-BCMA

    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.

06

What researchers measure

Primary outcomes

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

    For all participants

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

Secondary outcomes

  1. BILAG-2004 Scale Score

    The BILAG-2004 index assesses disease activity in systemic lupus erythematosus (SLE) to guide treatment. It evaluates nine organ systems separately, grading each from A to E based on clinical features: A (severe, requiring aggressive therapy), B (moderate), C (mild), D (previous involvement), and E (no involvement). Its focus is on tracking changes per system for targeted management.

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

  2. PGA

    Physician Global Assessment. PGA score is used to assess the disease activity status of patient by physician. PGA min-max(0-3, the higher score represents the worse result)

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

  3. SF-36

    The 36-Item Short Form Health Survey. For SLE and AAV. SF-36 score min-max(0-100,the higher score represents the better result)

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

  4. DORIS score

    The DORIS score defines remission criteria in systemic lupus erythematosus (SLE), guiding treatment goals and trial endpoints. Its core assessment includes: 1) clinical SLEDAI=0; 2) Physician Global Assessment (PGA)\<0.5; 3) prednisone ≤5mg/day; 4) stable immunosuppressive/biologic therapy. It also requires stable/improved serology and sustained duration of remission.

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

  5. LLDAS

    The LLDAS defines a "low disease activity state" in SLE, a key treatment target linked to better long-term outcomes. Core criteria include: 1) SLEDAI-2K ≤4; 2) no new disease activity; 3) Physician Global Assessment (PGA) ≤1.0; 4) prednisone ≤7.5mg/day; 5) stable immunosuppressive/biologic doses.

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

  6. Renal function response

    Only for LN patients

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

  7. Cmax

    For all subjects. Parameters of CAR copy number

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  8. Tmax

    For all subjects

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  9. AUC0-28d

    For all subjects

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  10. AUC0-90d

    For all subjects

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  11. Tlast

    For all subjects

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  12. 24-hour urine protein

    Only for LN

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  13. UPCR

    UPCR (Urine Protein-to-Creatinine Ratio) is a key index to measure assessing treatment response in lupus nephritis.

    Time frame: Baseline, Day 2, Day 6, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24

  14. Proportion of CAR-T cells in T cells

    For all subjects

    Time frame: baseline、Day 2、Day 6、Day 10、Day 14、Day 21、Day 28 after infusion

  15. serum sBCMA level

    BCMA expression levels of memory B cells and plasma blast cells in peripheral blood

    Time frame: Screening period, baseline, Day 14, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 post-infusion.

  16. Time to disease recurrence

    For IgG4-RD. Defined as the number of days between the day of infusion and the date of first treatment for IgG4-RD recurrence, as determined by a clinical professional, during the follow-up period

    Time frame: through study completion (at most 52 weeks)

  17. Changes in lymphocyte subpopulations

    For all subjects

    Time frame: Screening period, Day 14, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion, and at recurrence

  18. Proportion of patients with improved disease activity (IgG4-RD RI)

    For IgG4-RD. Improvement is defined as a decrease of ≥2 from the baseline disease activity score

    Time frame: Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  19. annualized recurrence rate

    For IgG4-RD

    Time frame: through study completion, an average of 1 year

  20. The proportion of subjects who achieved complete remission without relapse at week 52

    For IgG4-RD

    Time frame: at week 52

  21. Change in CRISS-25 score from baseline

    For systemic sclerosis. The score of CRISS-25 min-max(0-1.0, the higher score represents the better result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  22. Change in forced vital capacity (FVC) (mL) from baseline

    For systemic sclerosis

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  23. Change in mRSS from baseline

    For systemic sclerosis. The score of mRSS min-max(0-51, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  24. Change from baseline in Quantitative Interstitial Lung Disease (QILD) imaging score

    For systemic sclerosis. The min-max score (0-100%, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  25. Change from baseline in Quantitative Lung Fibrosis (QLF) imaging score

    For systemic sclerosis. The min-max score (0-100%, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  26. Changes in Scleroderma Patient Skin Report (SSPRO) compared to baseline

    For systemic sclerosis.The min-max score (0-108, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  27. Changes in Scleroderma Clinical Trial Consortium Damage Index (SCTC-DI) compared to baseline

    For systemic sclerosis. The min-max score (0-55, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  28. Changes in EUSTAR activity index scores compared to baseline

    For systemic sclerosis The min-max score (0-10, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  29. Changes in Capillaroscopic skin ulcer risk index (CSURI) compared to baseline

    For systemic sclerosis. CSURI is a continuous variable; the higher the score, the greater the ulcer risk.

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  30. Changes in Gastrointestinal questionnaire scores of the University of California, Los Angeles Scleroderma Clinical Trial Alliance compared to baseline

    For systemic sclerosis.The min-max score (0-3, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  31. Changes in serum SSc-related antibodies compared to baseline

    For systemic sclerosis

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  32. Changes in Muscle Strength Score (MRC) compared to baseline

    For idiopathic inflammatory myopathies. The min-max score (0-60, the higher score represents the better result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  33. The changes in total improvement score (TIS) compared to baseline

    For idiopathic inflammatory myopathies. The min-max score (0-100, the higher score represents the better result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  34. Changes in lesion size and severity index (CDASI) compared to baseline

    For idiopathic inflammatory myopathies. The CDASI is a core instrument for assessing the severity of cutaneous dermatomyositis. It consists of two independent subscales: Activity (scored 0-100, with higher scores indicating worse status) and Damage (scored 0-32, with higher scores indicating worse status).

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  35. Physician's overall disease activity score

    For idiopathic inflammatory myopathies. The min-max score (0-10, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  36. Changes in Manual Muscle Testing 8 (MMT8) compared to baseline

    For idiopathic inflammatory myopathies. The min-max score (0-150, the higher score represents the better result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  37. Changes in Expanded Myositis Disease Activity Index (EMDA) compared to baseline

    For idiopathic inflammatory myopathies. The min-max score (0-10, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  38. Changes in MDAAT score compared to baseline

    For idiopathic inflammatory myopathies. the overall score of extramuscular disease activity was assessed using the MDAAT scoring tool. The min-max score (0-60, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  39. Myositis Core Set Measures

    For idiopathic inflammatory myopathies

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  40. the remission rate of myositis (defined as an increase in TIS of ≥20, ≥40, and ≥60 points) was assessed according to the 2016 ACR/EULAR criteria for myositis remission

    For idiopathic inflammatory myopathies

    Time frame: Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  41. Time to improvement (TIS≥20, ≥40 and ≥60)

    For idiopathic inflammatory myopathies

    Time frame: up to 52 weeks

  42. Duration of remission (time from clinical remission to deterioration/time from remission to first recurrence)

    For idiopathic inflammatory myopathies

    Time frame: up to 52 weeks

  43. Changes in myositis specific antibodies and myositis associated antibodies in serum compared to baseline after injection

    For idiopathic inflammatory myopathies

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  44. Changes in ILD on CT imaging compared to baseline (only for patients with ILD at baseline)

    For idiopathic inflammatory myopathy patients with ILD in the baseline period

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  45. Pulmonary function examination evaluates changes in ventilation and diffusion function compared to baseline

    For idiopathic inflammatory myopathies. Pulmonary function examination can comprehensively evaluate the ventilation function, air exchange function, and airway responsiveness of the lungs, which is of great significance for the diagnosis, condition evaluation, and efficacy judgment of respiratory diseases

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  46. Myositis specific antibody levels

    For idiopathic inflammatory myopathies

    Time frame: Screening period, baseline, Day 14, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 post-infusion.

  47. Myositis related antibody levels

    For idiopathic inflammatory myopathies

    Time frame: Screening period, baseline, Day 14, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 post-infusion.

  48. The proportion of subjects who maintained remission

    For AAV

    Time frame: Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  49. duration of response

    For AAV

    Time frame: Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  50. Changes in Vasculitis Damage Index (VDI) scores compared to baseline

    For AAV

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  51. Changes in quality of life (SF-36 V2 scale) scores compared to baseline

    For AAV. The min-max score (0-100, the higher score represents the better result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  52. Changes in Disease Scope Index (DEI) compared to baseline

    For AAV. The min-max score (0-21, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

  53. Changes in Five Factor Score (FFS) compared to baseline

    For AAV. The min-max score (0-5, the higher score represents the worse result)

    Time frame: At Baseline, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 after infusion

Other outcomes

  1. Virus Leak Detection

    For all subjects. Collect blood, saliva, urine, and fecal samples for cryogenic storage and detect any potential leaks. Collect blood, saliva, urine, and fecal samples to detect virus levels and determine if there is a carrier virus leak

    Time frame: baseline、Day2、Day4、Day6、Day8、Day10、Day14、Day21、Day28

  2. immunogenicity

    For all subjects. Detecting anti CAR antibodies in patient serum using bridging ELISA or flow cytometry

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

  3. Single-cell sequencing

    For all subjects. Collect peripheral blood from patients and perform single-cell sequencing. Droplet platforms (such as 10 × Genomics) are the most commonly used scRNA seq platforms. The platform places single cells into water in oil droplets containing gel beads through the microfluidic chamber. gel beads have mRNA capture primers, unique molecular barcodes, and enzyme/reagent mixtures required for cell lysis and reverse transcription. After reverse transcription of transcription information to obtain cDNA, high-throughput sequencing can be performed through PCR amplification and library preparation.

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

07

Study locations

1 site
  • Tongji Hospital, Affiliated to Tongji Medical College of Huazhong University of Science and Technology
    Wuhan, Hubei 430000, China
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 17, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT07339540
Lead sponsor
Tongji Hospital
Responsible party
Lingli Dong (professor, Tongji Hospital) — Principal investigator
First posted
Jan 14, 2026
Start date
Nov 4, 2025
Primary completion
Oct 30, 2028 (estimated)
Completion
Oct 30, 2028 (estimated)
Last update
Sep 17, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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