This is a Phase I clinical trial to evaluate the efficacy and safety of CD19-targeted CAR-T in the treatment of refractory juvenile dermatomyositis (RJDM).The experiment was divided into two phases: dose exploration (Part A) and dose extension (Part B).
Age: ≥5 years and <17 years old
To meet the diagnostic criteria of JDM, four or five of the following criteria must be met:① symmetrical proximal muscle weakness; ②Characteristic skin changes, including positive dermatitis (purplish red rash on upper eyelid with periorbital edema) and Gottron papules (red patchy squamous papules on the back of knuckles); ③ The level of one muscle enzyme in serum was increased; ④ Positive myositis antibody; ⑤Electromyography shows denervation and myopathy; ⑥ Muscle biopsies showed necrosis and inflammation.
The classification criteria of RJDM must meet ① and any of the criteria②-④: ① Patients who are intolerant or unresponsive to glucocorticoids and at least 2 immunosuppressants, adequate hormone therapy and duration of at least 6 months; ② The disease progresses rapidly and/or involves organs such as lungs, heart and gastrointestinal tract; ③ Calcification of subcutaneous or muscle and joint tissues; ④ Repeated rashes or skin ulcers.
myositis specific antibody positive, defined as MDA-5, NXP2, TIF-1γ, Ro-52 and any other positive;
Had previously received CAR T cell therapy;
Have other autoimmune or rheumatic diseases other than JDM;
primary immunodeficiency or severe secondary immunodeficiency that has not been corrected;
accompanied by serious infectious diseases, including but not limited to active tuberculosis, latent tuberculosis infection, active viral hepatitis, etc.;
Single group
CD19-targetd CAR-T
Biological/VaccineSubjects underwent lymphocyte clearance chemotherapy and then received a single intravenous cell infusion
To evaluate the safety of CAR-T in the treatment of refractory juvenile dermatomyositis [Safety and Tolerability]
The incidence of adverse events after CAR-T cell infusion was assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).including the type, frequency and severity of adverse events
To evaluate the efficacy of CAR-T in the treatment of refractory juvenile dermatomyositis [Effectiveness]
Disease improvement rate: The number of subjects who achieved disease improvement as a percentage of all subjects who received transfusions.
Duration of Response (DOR) of CAR-T treatment in the treatment of refractory juvenile dermatomyositis [Effectiveness]
DOR will be assessed from the first assessment of remission to the first assessment of recurrence or progression of the disease or death from any cause
AUCS of CD19 CAR-T cells [Cell dynamics]
AUCS is defined as the area under the curve in 90 days
CMAX of CD19 CAR-T cells [Cell dynamics]
CMAX is defined as the highest concentration of CEA CAR-T cells expanded in peripheral blood
TMAX of CD19 CAR-T cells[Cell dynamics]
TMAX is defined as the time to reach the highest concentration
To evaluate the effects of CD19-targeting CAR T cell preparations on the weight of children
Weight in kilograms were monitored after transfusion until subjects reached 18 years of age
To evaluate the effects of CD19-targeting CAR T cell preparations on the height of children
Height in meters were monitored after transfusion until subjects reached 18 years of age
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