Refractory ANCA Associated Vasculitis and Lupus Nephritis Treated With BCMA-targeting CAR-T Cells
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-70
SponsorLingli Dong
About this trial
Lupus nephritis (LN) and ANCA-associated vasculitis are severe autoimmune diseases, which may lead to the death of patients, particularly when they are refractory to the conventional therapeutic agents. Based on the current knowledge, the autoantibodies against self-antigens may exert important pathological roles in the pathogenesis of both LN and ANCA-associated vasculitis, of which the origins are primarily plasmablasts and plasma cells. BCMA is the molecule expressed on memory B cells, plasmablasts and plasma cells, and therefore is an ideal target for the elimination of potential pathogenic antibody secreting cells. Chimeric antigen receptor (CAR) T cells against BCMA may provide a novel therapeutic way for the refractory LN and ANCA-associated vasculitis patients to eliminate the pathogenic autoantibody-secreting cells. In this study, the safety and efficacy of a novel CAR-T cell therapy using PRG-1801 cells, are evaluated in patients with refractory LN and ANCA-associated vasculitis.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥ 18 years old, not exceeding 70 years old (including 70 years old);
If the kidneys are involved, estimate the glomerular filtration rate (eGFR) to be ≥ 15 mL/minute/1.73 m2;
Absolute lymphocyte count: ≥ 0.5 × 10 ^ 9/L [The use of granulocyte colony-stimulating factor (G CSF) is allowed, but subjects are not allowed to receive this supportive treatment within 7 days before the screening period laboratory examination];
Absolute neutrophil count: ≥ 1.0 × 10 ^ 9/L [The use of granulocyte colony-stimulating factor (G-CSF) is allowed, but subjects are not allowed to receive this supportive treatment within 7 days before the screening period laboratory examination];
Disqualifiers
Pregnant or lactating women;
If combined with alveolar hemorrhage, invasive pulmonary ventilation is required;
Refractory GPA and MPA: Combined with other autoimmune diseases involving multiple organ systems, such as EGPA, SLE, IgA vasculitis (Henoch Schönlein), rheumatoid arthritis, inflammatory myositis, anti-glomerular basement membrane disease, or cryoglobulinemia vasculitis;
Difficult to treat LN: severe extrarenal clinical manifestations such as lupus encephalopathy, pulmonary hemorrhage, lupus myocarditis, lupus enteritis, and lupus crisis; Other autoimmune diseases other than combined SLE, including dermatomyositis/polymyositis, mixed connective tissue disease, systemic sclerosis, rheumatoid arthritis, etc;
Trial design
Design model
Single group
Treatments tested in this trial
PRG-1801 （CAR-T against BCMA）
DrugPatients with refractory LN and ANCA-associated vasculitis will be treated with PRG-1801
Treatment groups
24 Participants
are divided into 1 treatment groupGroup A: CAR-T treatmentExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
AE Incidence of PRG-1801 Single Infusion
AE Incidence of PRG-1801 Single Infusion
Time frame
24 months after PRG-1801 infusion2
Types and incidence of dose-limiting toxicity(DLT) after PRG-1801 cells infusion in subjects with refractory ANCA-associated vasculitis and refractory LN.
To evaluate the DLT occurred within 28 days and 3 months after PRG-1801 infusion
Time frame
28 days and 3 months after PRG-1801 infusionSecondary outcomes
1
Therapeutic effects-For refractory ANCA-associated vasculitis(The proportion of subjects who maintained remission after cell infusion)
The proportion of subjects who maintained remission after cell infusion
Time frame
Month1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24 after cell infusion2
Therapeutic effects-For refractory ANCA-associated vasculitis(Changes in VDI (Vasculitis Damage Index) scores from baseline after cell infusion)
Changes in VDI (Vasculitis Damage Index) scores from baseline after cell infusion
Time frame
Month1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24 after cell infusion3
Therapeutic effects-For refractory ANCA-associated vasculitis (Changes in glomerular filtration rate compared to baseline after cell infusion in subjects with renal involvement.)
Changes in glomerular filtration rate compared to baseline after cell infusion in subjects with renal involvement.
Time frame
Month1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24 after cell infusion4
Therapeutic effects-For refractory LN (Changes in SLEDAI-2000 score from baseline after cell infusion)
Changes in SLEDAI-2000 score from baseline after cell infusion
Time frame
Month1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24 after cell infusionSponsors and contacts
Click on the lead sponsor to view all of their trials.
Lingli Dong
Lead sponsor
Tongji Hospital
Sponsor institution
Shenzhen Pregene Biopharma Co., Ltd.
Collaborator
Source ClinicalTrials.gov