Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis

Trial details

Who runs it

Chongqing Precision Biotech Co., Ltd

Trial code

NCT06569472

Record updated

26 August 2024

Original description

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).Read full original descriptionShow less

Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Juvenile Dermatomyositis (RJDM)

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).

Inclusion: 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; If the patient has SRP or HMGCR antibody positive immune-mediated necrotizing myopathy equivalent to RJDM, the inclusion criteria of (2) - (4) can be met; The functions of important organs are basically normal:; ① Cardiac function: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram; ② Renal function: eGFR≥30ML/min/1.73m2; ③ Liver function: AST and ALT≤3.0 ULN, total bilirubin ≤2.0×ULN; ④ Lung function: Lung function is basically normal, SpO2≥92%; Have the criteria for simple or intravenous blood collection, and no other contraindications for cell collection; The subject of childbearing age has a negative urine pregnancy test result and agrees to take effective contraceptive measures during the test period until 1 year after the infusion; The patient or his/her guardian agrees to participate in this clinical trial and signs an informed consent indicating that he/she understands the purpose and procedure of this clinical trial and is willing to participate in the study.

Exclusion: Had previously received CAR T cell therapy; Congenital heart disease or history of acute myocardial infarction within 6 months before screening, or severe arrhythmias (including multi-source frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with a large number of pericardial effusion, serious myocarditis, etc.; Or patients with unstable vital signs who need hypertensive drugs to maintain their blood pressure; There is an active or uncontrollable infection that requires systemic treatment within 1 week prior to screening; Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; Acute graft-versus-host disease (GVHD) of grade 2 or above was present within 2 weeks prior to screening; Had received live vaccine within 4 weeks prior to screening; Patients who had participated in other clinical trials within 3 months prior to enrollment; Have other autoimmune or rheumatic diseases other than JDM; accompanied by serious infectious diseases, including but not limited to active tuberculosis, latent tuberculosis infection, active viral hepatitis, etc; Evidence of active malignant disease or diagnosis of malignant tumor (including hematological malignancies and solid tumors, except resected and cured skin basal cell carcinoma); suffering from other diseases that require long-term use of glucocorticoids or immunosuppressants; Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal reference value range; Or hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Or positive for human immunodeficiency virus (HIV) antibodies; Or syphilis test positive; Or cytomegalovirus (CMV) DNA test positive; Patients with known malignant diseases such as tumors before screening; primary immunodeficiency or severe secondary immunodeficiency that has not been corrected; Positive blood pregnancy test; Situations in which other investigators consider it inappropriate to participate in the study.

ClinicalTrials.gov NCT06569472