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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.
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Studietype
Inschrijving (Geschat)
Fase
- Vroege fase 1
Contacten en locaties
Studiecontact
- Naam: Ming Gao, Master
- Telefoonnummer: +86 177 1418 8689
- E-mail: ming.gao@imvivabio.com
Studie Locaties
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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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Contact:
- Qiong Fu, Doctor
- Telefoonnummer: +86 13585603288
- E-mail: Fuqiong5@163.com
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Beschrijving
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Experimenteel: 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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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
SLE (systemic lupus erythematosus)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
|
|
SLE (systemic lupus erythematosus)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 2 years
|
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)
Tijdsspanne: 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 )
Tijdsspanne: 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
|
|
SS (Sjögren's syndrome )
Tijdsspanne: 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
|
|
SS (Sjögren's syndrome )
Tijdsspanne: 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
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Medewerkers en onderzoekers
Onderzoekers
- Hoofdonderzoeker: Qiong Fu, Doctor, Renji Hospital
Studie record data
Bestudeer belangrijke data
Studie start (Geschat)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
- Musculoskeletale aandoeningen
- Mondziekten
- Stomatognatische ziekten
- Ziekten van het zenuwstelsel
- Vaatziekten
- Hart-en vaatziekten
- Spierziekten
- Neuromusculaire aandoeningen
- Artritis
- Gewrichtsziekten
- Reumatische aandoeningen
- Bindweefselziekten
- Auto-immuunziekten
- Ziekten van het immuunsysteem
- Oogziekten
- Huidziektes
- Huidziekten, vasculair
- Artritis, reumatoïde
- Xerostomie
- Speekselklierziekten
- Droge-ogen-syndroom
- Ziekten van het traanapparaat
- Vasculitis
- Systemische vasculitis
- Huid- en bindweefselaandoeningen
- Lupus erythematosus, systemisch
- Syndroom van Sjogren
- Sclerodermie, systemisch
- Myositis
- Anti-neutrofiele cytoplasmatische antilichaam-geassocieerde vasculitis
Andere studie-ID-nummers
- BHCT-RD06-05-03
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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