Treatment of Refractory Juvenile Dermatomyositis with CD19 Targeted CAR-T Cells
Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
This study is testing a new CAR-T cell therapy to see if it can help kids with tough-to-treat juvenile dermatomyositis feel better and improve their health.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 18 (estimated) |
| Ages | 5 Years and up |
| Sex | All |
| Sponsor | Chongqing Precision Biotech Co., Ltd Industry-sponsored |
| Drugs / interventions | CAR-T, prednisone |
| Locations | 1 site (Hangzhou, Zhejiang) |
| Trial ID | NCT06686524 on ClinicalTrials.gov |
What this trial studies
This open-label, single-arm, dose-escalation trial evaluates the safety and efficacy of a universal CAR-T cell therapy targeting CD19 in patients with refractory juvenile dermatomyositis (RJDM). The study employs a '3+3' design with three dosage groups, starting from a low dose and escalating based on safety and efficacy data. Each group will enroll 3 to 6 patients, aiming to assess pharmacokinetic and pharmacodynamic properties, immune system reconstitution, and long-term survival benefits.
Who should consider this trial
Good fit: Ideal candidates include children aged 5 years and older diagnosed with refractory juvenile dermatomyositis who have not responded adequately to standard treatments.
Not a fit: Patients with mild juvenile dermatomyositis or those who have not yet undergone standard treatment options may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from refractory juvenile dermatomyositis.
How similar studies have performed: While CAR-T therapies have shown promise in other conditions, this specific application for juvenile dermatomyositis is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
* (1) Age ≥ 5 years old;
* (2) Diagnosis of juvenile dermatomyositis (JDM) according to Bohan and Peter criteria;
* (3) Meeting the classification criteria for RJDM, and meeting condition ① and any one of ② to ⑤ conditions:
* Intolerance or inadequate response to glucocorticoids (prednisone 1-2mg/kg/d or equivalent dose of other hormones) and at least two immunosuppressants, with hormone therapy lasting for at least 6 months;
* Rapid progression of the disease and/or involvement of organs such as lungs, heart, and gastrointestinal tract;
* Calcification of subcutaneous or muscular and articular tissues;
* Repeated skin rashes or ulcers;
* Repeated or persistent muscle weakness (muscle magnetic resonance imaging indicating widespread, diffuse edema or a Children's Myositis Assessment Scale (CMAS) score \< 48, and at least two abnormal results among the following five core measurement indicators: Physician's Global Assessment (PhGA) ≥ 2cm, Patient's Global Assessment (PtGA) ≥ 2cm, Disease Activity Score (DAS) ≥ 2 points, Child Health Assessment Questionnaire (C-HAQ) total score ≥ 0.25 points, and muscle enzyme level \> 1.5 times the upper limit of normal (ULN));
* (4)Patients with immune-mediated necrotizing myopathy who are positive for SRP or HMGCR antibodies meet the criteria for RJDM and can be directly included;
* (5) Basic normal function of important organs:
* Cardiac function: Left ventricular ejection fraction (LVEF) ≥55%, with no significant abnormalities observed in the electrocardiogram;
* Renal function: eGFR ≥ 30mL/min/1.73m2;
* Liver function: AST and ALT ≤3.0 ULN, total bilirubin ≤2.0×ULN (excluding those caused by primary diseases);
* Pulmonary function: SpO2 ≥92%;
* (6) Female subjects of childbearing age have a negative result in the urine pregnancy test and agree to take effective contraceptive measures during the trial until one year after infusion;
* (7) The patient or their guardian agrees to participate in this clinical trial and signs an informed consent form, indicating their understanding of the purpose and procedures of the clinical trial and their willingness to participate in the study.
Exclusion Criteria:
Subjects meeting any of the following criteria will be excluded from this trial:
* (1) Previously received CAR-T cell therapy (except for those whose safety risks have been judged as eliminated by the investigator);
* (2) Accompanied by primary immunodeficiency disease or severe secondary immunodeficiency disease that has not been corrected;
* (3) Accompanied by severe, active, or uncontrolled infectious diseases, including but not limited to active tuberculosis, latent tuberculous infection, active viral hepatitis, etc.;
* (4) Known to have active malignant diseases or confirmed malignancies before screening (including hematological malignancies and solid tumors, except for resected and cured cutaneous basal cell carcinoma);
* (5) Suffering from congenital heart disease or having a history of acute myocardial infarction within 6 months, or severe arrhythmia (including multifocal frequent ventricular supraventricular tachycardia, ventricular tachycardia, etc.); or complicated with moderate to large pericardial effusion, severe myocarditis, etc.; or unstable vital signs requiring vasopressors to maintain blood pressure;
* (6) Accompanied by other diseases that require long-term use of glucocorticoids or immunosuppressants;
* (7) Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; or presence of acute graft-versus-host disease (GVHD) of grade 2 or above within 2 weeks before screening;
* (8) Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and peripheral blood hepatitis B virus (HBV) DNA titer detection is greater than the normal reference range; or positive for hepatitis C virus (HCV) antibody and peripheral blood hepatitis C virus (HCV) RNA titer detection is greater than the normal reference range; or positive for human immunodeficiency virus (HIV) antibody; or positive for syphilis test;
* (9) Received live vaccines within 4 weeks before screening;
* (10) Positive blood pregnancy test;
* (11) Participated in other clinical trials within 3 months before enrollment;
* (12) Other conditions that the investigator considers unsuitable for participation in the study.
Where this trial is running
Hangzhou, Zhejiang
- Children's Hospital Zhejiang University School of Medicine, Hangzhou, zhejiang — Hangzhou, Zhejiang, China (Recruiting)
Study contacts
- Study coordinator: Lu Mei ping, M.D
- Email: meipinglu@zju.edu.cn
- Phone: +86 13685773988
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.