Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis
- Conditions
- Juvenile Dermatomyositis
- Registration Number
- NCT06569472
- Lead Sponsor
- Chongqing Precision Biotech Co., Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 10
Inclusion Criteria:<br><br> 1. Age: =5 years and <17 years old<br><br> 2. To meet the diagnostic criteria of JDM, four or five of the following criteria must<br> be met:? symmetrical proximal muscle weakness; ?Characteristic skin changes,<br> including positive dermatitis (purplish red rash on upper eyelid with periorbital<br> edema) and Gottron papules (red patchy squamous papules on the back of knuckles); ?<br> The level of one muscle enzyme in serum was increased; ? Positive myositis antibody;<br> ?Electromyography shows denervation and myopathy; ? Muscle biopsies showed necrosis<br> and inflammation.<br><br> 3. The classification criteria of RJDM must meet ? and any of the criteria?-?: ?<br> Patients who are intolerant or unresponsive to glucocorticoids and at least 2<br> immunosuppressants, adequate hormone therapy and duration of at least 6 months; ?<br> The disease progresses rapidly and/or involves organs such as lungs, heart and<br> gastrointestinal tract; ? Calcification of subcutaneous or muscle and joint tissues;<br> ? Repeated rashes or skin ulcers.<br><br> 4. myositis specific antibody positive, defined as MDA-5, NXP2, TIF-1?, Ro-52 and any<br> other positive;<br><br> 5. If the patient has SRP or HMGCR antibody positive immune-mediated necrotizing<br> myopathy equivalent to RJDM, the inclusion criteria of (2) - (4) can be met.<br><br> 6. The functions of important organs are basically normal:<br><br> ? Cardiac function: left ventricular ejection fraction (LVEF) =55%, no obvious<br> abnormality in electrocardiogram;<br><br> ? Renal function: eGFR=30ML/min/1.73m2;<br><br> ? Liver function: AST and ALT=3.0 ULN, total bilirubin =2.0×ULN;<br><br> ? Lung function: Lung function is basically normal, SpO2=92%;<br><br> 7. Have the criteria for simple or intravenous blood collection, and no other<br> contraindications for cell collection;<br><br> 8. The subject of childbearing age has a negative urine pregnancy test result and<br> agrees to take effective contraceptive measures during the test period until 1 year<br> after the infusion;<br><br> 9. The patient or his/her guardian agrees to participate in this clinical trial and<br> signs an informed consent indicating that he/she understands the purpose and<br> procedure of this clinical trial and is willing to participate in the study.<br><br>Exclusion Criteria:<br><br> 1. Had previously received CAR T cell therapy;<br><br> 2. Have other autoimmune or rheumatic diseases other than JDM;<br><br> 3. primary immunodeficiency or severe secondary immunodeficiency that has not been<br> corrected;<br><br> 4. accompanied by serious infectious diseases, including but not limited to active<br> tuberculosis, latent tuberculosis infection, active viral hepatitis, etc.;<br><br> 5. Evidence of active malignant disease or diagnosis of malignant tumor (including<br> hematological malignancies and solid tumors, except resected and cured skin basal<br> cell carcinoma)<br><br> 6. Congenital heart disease or history of acute myocardial infarction within 6 months<br> before screening, or severe arrhythmias (including multi-source frequent<br> supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with a<br> large number of pericardial effusion, serious myocarditis, etc.; Or patients with<br> unstable vital signs who need hypertensive drugs to maintain their blood pressure;<br><br> 7. suffering from other diseases that require long-term use of glucocorticoids or<br> immunosuppressants;<br><br> 8. There is an active or uncontrollable infection that requires systemic treatment<br> within 1 week prior to screening;<br><br> 9. Received solid organ transplantation or hematopoietic stem cell transplantation<br> within 3 months before screening; Acute graft-versus-host disease (GVHD) of grade 2<br> or above was present within 2 weeks prior to screening;<br><br> 10. Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive<br> and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal<br> reference value range; Or hepatitis C virus (HCV) antibody positive and peripheral<br> blood hepatitis C virus (HCV) RNA titer greater than the normal reference value<br> range; Or positive for human immunodeficiency virus (HIV) antibodies; Or syphilis<br> test positive; Or cytomegalovirus (CMV) DNA test positive;<br><br> 11. Had received live vaccine within 4 weeks prior to screening;<br><br> 12. Positive blood pregnancy test;<br><br> 13. Patients with known malignant diseases such as tumors before screening;<br><br> 14. Patients who had participated in other clinical trials within 3 months prior to<br> enrollment;<br><br> 15. Situations in which other investigators consider it inappropriate to participate in<br> the study.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate the safety of CAR-T in the treatment of refractory juvenile dermatomyositis [Safety and Tolerability];To evaluate the efficacy of CAR-T in the treatment of refractory juvenile dermatomyositis [Effectiveness];Duration of Response (DOR) of CAR-T treatment in the treatment of refractory juvenile dermatomyositis [Effectiveness]
- Secondary Outcome Measures
Name Time Method AUCS of CD19 CAR-T cells [Cell dynamics];CMAX of CD19 CAR-T cells [Cell dynamics];TMAX of CD19 CAR-T cells[Cell dynamics]