- ICH GCP
- US-Register für klinische Studien
- Klinische Studie NCT07596680
CD19/BCMA-Targeted Universal CAR-T Cell Injection for the Treatment of Autoimmune Diseases
Clinical Study on the Safety, Efficacy and Pharmacokinetics of Universal CD19/BCMA-Targeted CAR-T Cell Injection in Patients With Autoantibody-Mediated Autoimmune Diseases
This is a single-arm, open-label, investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RD06-05 in patients with autoantibody-mediated autoimmune diseases. The enrolled population consists of patients with active autoimmune diseases, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), ANCA-associated vasculitis (AAV), idiopathic inflammatory myopathies (IIM), Sjögren's syndrome (SS), among others.
The CAR-T cell dose used in this study is 6×10⁶ CAR⁺ T cells/kg. Six subjects will be enrolled for each indication, with a total of 30 subjects to be enrolled.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Studientyp
Einschreibung (Geschätzt)
Phase
- Frühphase 1
Kontakte und Standorte
Studienkontakt
- Name: Ming Gao, Master
- Telefonnummer: +86 177 1418 8689
- E-Mail: ming.gao@imvivabio.com
Studienorte
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Shanghai Municipality
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Shanghai, Shanghai Municipality, China, 220127
- Renji Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
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Kontakt:
- Qiong Fu, Doctor
- Telefonnummer: +86 13585603288
- E-Mail: Fuqiong5@163.com
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-
Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
- Erwachsene
- Älterer Erwachsener
Akzeptiert gesunde Freiwillige
Beschreibung
Inclusion Criteria
General Inclusion Criteria (All Patients)
- Voluntarily provides written informed consent.
- Age ≥18 and ≤70 years, any gender.
Adequate organ function:
- ALT and AST ≤3×ULN; total bilirubin ≤2×ULN (excluding Gilbert syndrome).
- Creatinine ≤1.5×ULN or creatinine clearance ≥40 mL/min.
- Neutrophils ≥1×10⁹/L; hemoglobin ≥60 g/L; platelets ≥20×10⁹/L; lymphocytes >0.3×10⁹/L.
- INR ≤1.5×ULN or PT ≤1.5×ULN.
- Resting room-air SpO₂ ≥92%.
- LVEF ≥50% on echocardiogram.
- Negative serum or urine pregnancy test for females of childbearing potential at screening.
- Highly effective contraception required from 28 days before lymphodepletion until 12 months after RD06-05 infusion for females; effective barrier contraception required from lymphodepletion until 12 months after RD06-05 infusion for males, with no sperm donation during the study.
For SLE Patients
- Diagnosis of SLE per 2019 EULAR/ACR or 2012 SLICC criteria.
- Active disease despite ≥2 months of stable (≥2 weeks) treatment with glucocorticoids plus immunosuppressants and/or biologics; prednisone ≥7.5 mg/day or equivalent.
- Positive ANA, anti-dsDNA antibody, and/or anti-Smith antibody at screening.
- SLEDAI-2K >6 and clinical SLEDAI-2K ≥4 at screening. Patients with lupus nephritis (proteinuria >0.5 g/24h, UPCR >500 mg/g, or active urinary sediment) are exempt from clinical SLEDAI-2K requirement.
- Physician Global Assessment (PGA) ≥1.0 (0-3 VAS) at screening.
For SSc Patients
- Diagnosis of SSc per 2013 ACR/EULAR criteria.
- Diffuse cutaneous SSc at screening.
- Active disease defined by at least one of: new SSc within 2 years; new/worsening skin or thoracic/abdominal involvement within 6 months; worsening skin thickening (mRSS ≥2); tendon friction rubs within 3 months; worsening respiratory symptoms with FVC decline ≥5% predicted or DLCO decline ≥10% predicted; or ILD progression on HRCT compared to 12 months prior.
- Refractory or relapsing disease after >6 months of conventional therapy including glucocorticoids, cyclophosphamide, immunosuppressants, and/or biologics.
For AAV Patients
- Diagnosis of ANCA-associated vasculitis (MPA, GPA, EGPA) per 2022 ACR/EULAR criteria.
- Positive MPO-ANCA or PR3-ANCA.
- BVAS with at least 1 major item, 3 minor items, or 2 renal items.
- Failure of standard of care: no remission after ≥4 months of glucocorticoids plus cyclophosphamide/rituximab; relapse after prior remission; or persistent active disease despite ≥6 months of SOC.
For IIM Patients
- Diagnosis of IIM (DM, ASS, IMNM) per 2017 ACR/EULAR criteria (probability ≥55%).
- Active disease defined by ≥2 abnormal core measures, or active myositis on muscle MRI, or active inflammation on muscle biopsy within 16 weeks.
- Positive myositis-specific autoantibodies.
- Refractory or relapsing disease after ≥6 months of conventional therapy including glucocorticoids, immunosuppressants, and/or biologics.
For pSS Patients
- Diagnosis of primary Sjögren's syndrome per 2016 ACR/EULAR criteria.
- Positive anti-SSA/Ro antibody.
- ESSDAI ≥6 at screening.
- Refractory or relapsing disease after ≥6 months of conventional therapy including glucocorticoids, immunosuppressants, and/or biologics.
Exclusion Criteria:
General Exclusion Criteria (All Patients):
- Coexisting autoimmune disease confounding disease activity/safety (stable ≥3 months may be eligible with approval).
- Anti-CD20 mAb/T-cell engager within 3 months; CD19/BCMA-targeted therapy within 6 months (exception with CD19⁺ B-cell > LLN and approval).
- Rapidly progressive glomerulonephritis (RPGN).
- NYHA III/IV heart failure; severe cardiac disease within 12 months.
- Severe CNS disease impairing compliance/assessments.
- Malignancy history (except cured non-melanoma skin cancer/carcinoma in situ, disease-free ≥3 years).
- Primary immunodeficiency.
- Uncontrolled infection (uncomplicated UTI/upper respiratory infection permitted).
- Positive HIV; positive HCV (except undetectable RNA); positive syphilis.
- Positive HBsAg; positive HBcAb (except undetectable HBV DNA).
- Positive EBV/CMV DNA/IgM at screening.
- Active/recurrent tuberculosis.
- Prior CAR-T or genetically modified immune cell therapy.
- Live attenuated vaccine within 4 weeks before enrollment.
- Hypersensitivity to cell therapy product components.
- Tacrolimus hypersensitivity or ≥Grade 3 toxicity requiring hospitalization.
- Other clinical trial participation within 30 days before screening.
- Pregnant/breastfeeding; childbearing potential unwilling to use effective contraception.
- Any other ineligible condition (investigator judgment).
Exclusion Criteria for SLE
- Active/unstable neuropsychiatric SLE requiring intervention within 90 days.
- Anti-BAFF/APRIL therapy within required washout period; multiple NSAIDs within 14 days; inability to hold NSAIDs; intra-articular glucocorticoids within 6 weeks; immunosuppressants exceeding dose limits; hydroxychloroquine dose adjustment within 8 weeks; ACEI/ARB/SGLT2i adjustment within 4 weeks.
Exclusion Criteria for AAV
- Alveolar hemorrhage requiring invasive ventilation beyond screening.
- Dialysis/plasmapheresis within 12 weeks.
- Renal transplantation history.
- Cyclophosphamide within 12 weeks; immunosuppressant discontinuation required 1 week before lymphodepletion.
- High-dose IV glucocorticoids within 4 weeks.
- Oral glucocorticoids >60mg prednisone equivalent daily for >6 weeks.
- Specific immunosuppressants/biologics within 4 weeks.
- Concomitant strong CYP3A4 inducers.
Exclusion Criteria for IIM
- Severe rhabdomyolysis or CK ≥120×ULN at screening.
- FVC ≤50% predicted or DLCO ≤40% predicted at screening.
Exclusion Criteria for SSc
- Significant respiratory disease other than ILD.
- FVC <50% or DLCO <40% predicted at screening/baseline.
- Lung transplantation listing/expected within 12 months.
- Scleroderma renal crisis within 6 months.
- Scleroderma-like disorders.
- Prior chlorambucil, bone marrow transplantation, or total lymphoid irradiation.
Exclusion Criteria for pSS
- Active fibromyalgia interfering with assessment/requiring medication adjustment (stable permitted).
- Cyclophosphamide within 12 weeks; immunosuppressant discontinuation required 1 week before lymphodepletion.
- High-dose glucocorticoids (≥60mg/day) within 4 weeks.
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: N / A
- Interventionsmodell: Einzelgruppenzuweisung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
|---|---|
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Experimental: RD06-05
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CAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.
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Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
SLE (systemic lupus erythematosus)
Zeitfenster: 2 years
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Proportion of patients achieving lupus low disease activity state (LLDAS) and DORIS(Definition Of Remission In SLE) remission, as well as the proportion of patients achieving drug-free remission
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2 years
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SLE (systemic lupus erythematosus)
Zeitfenster: 2 years
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Proportion of SLE patients with renal involvement who achieved complete renal response (CRR) and partial renal response
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2 years
|
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SLE (systemic lupus erythematosus)
Zeitfenster: 2 years
|
Change in UPCR(Urine Protein/Creatinine Ratio) from baseline in SLE patients with renal involvement
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2 years
|
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SLE (systemic lupus erythematosus)
Zeitfenster: 2 years
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Change in eGFR(estimated Glomerular Filtration Rate) from baseline in SLE patients with renal involvement
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2 years
|
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SLE (systemic lupus erythematosus)
Zeitfenster: 2 years
|
Changes in SLEDAI-2K, Physician Global Assessment (PGA), and British Isles Lupus Assessment Group (BILAG) score from baseline
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2 years
|
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SLE (systemic lupus erythematosus)
Zeitfenster: 2 years
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Changes in anti-dsDNA antibody, C3 and C4 levels from baseline
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2 years
|
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AAV (ANCA-associated vasculitis)
Zeitfenster: 2 years
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Change in Birmingham Vasculitis Activity Score (BVAS) from baseline.
The Birmingham Vasculitis Activity Score (BVAS) ranges from a minimum of 0 to a maximum of 63, with higher scores indicating worse disease activity and clinical outcomes.
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2 years
|
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AAV (ANCA-associated vasculitis)
Zeitfenster: 2 years
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Proportion of patients with vasculitis relapse (including major flare and minor flare)
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2 years
|
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IIM (idiopathic inflammatory myopathies)
Zeitfenster: 2 years
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Major clinical remission assessed according to the 2016 ACR/EULAR myopathy remission criteria
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2 years
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SSc (systemic sclerosis)
Zeitfenster: 2 years
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Changes in modified Rodnan Skin Score (mRSS) from baseline
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2 years
|
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SSc (systemic sclerosis)
Zeitfenster: 2 years
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Changes in European Scleroderma Trials and Research group (EUSTAR) Activity Index from baseline
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2 years
|
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SSc (systemic sclerosis)
Zeitfenster: 2 years
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Changes in Forced Vital Capacity (FVC) and Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) from baseline in patients complicated with interstitial lung disease
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2 years
|
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SS (Sjögren's syndrome )
Zeitfenster: 2 years
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Change in ESSDAI(EULAR Sjögren's Syndrome Disease Activity Index) score from baseline.
ESSDAI has a score range from 0 to 105, with higher scores indicating greater disease activity and poorer clinical outcomes.
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2 years
|
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SS (Sjögren's syndrome )
Zeitfenster: 2 years
|
Change in ESPRI(EULAR Sjögren's Syndrome Patient Reported Index) score from baseline.
ESSPRI ranges from a minimum of 0 to a maximum of 10, with higher scores indicating worse symptoms and poorer patient-reported outcomes.
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2 years
|
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SS (Sjögren's syndrome )
Zeitfenster: 2 years
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Change in STAR(Sjögren's Tool for Assessing Response) score from baseline.
STAR score ranges from 0 to 28, with higher scores indicating more severe disease status and poorer therapeutic response.
|
2 years
|
Mitarbeiter und Ermittler
Ermittler
- Hauptermittler: Qiong Fu, Doctor, Renji Hospital
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Geschätzt)
Primärer Abschluss (Geschätzt)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Erkrankungen des Bewegungsapparates
- Mundkrankheiten
- Stomatognathe Erkrankungen
- Erkrankungen des Nervensystems
- Gefäßerkrankungen
- Herz-Kreislauf-Erkrankungen
- Muskelerkrankungen
- Neuromuskuläre Erkrankungen
- Arthritis
- Gelenkerkrankungen
- Rheumatische Erkrankungen
- Bindegewebserkrankungen
- Autoimmunerkrankungen
- Erkrankungen des Immunsystems
- Augenkrankheiten
- Hautkrankheiten
- Hautkrankheiten, Gefäß
- Arthritis, Rheuma
- Xerostomie
- Speicheldrüsenerkrankungen
- Syndrome des trockenen Auges
- Erkrankungen des Tränenapparates
- Vaskulitis
- Systemische Vaskulitis
- Haut- und Bindegewebserkrankungen
- Lupus erythematodes, systemisch
- Sjögren-Syndrom
- Sklerodermie, systemisch
- Myositis
- Anti-Neutrophilen-Zytoplasma-Antikörper-assoziierte Vaskulitis
Andere Studien-ID-Nummern
- BHCT-RD06-05-03
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