Actively Recruiting
Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for Treatment of Refractory Juvenile Dermatomyositis (RJDM)
Led by Chongqing Precision Biotech Co., Ltd · Updated on 2024-11-13
18
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of a universal CAR-T cell therapy targeting CD19 in treating patients with refractory juvenile dermatomyositis (RJDM). This Phase 1, open-label, single-arm dose-escalation trial also aims to study how the therapy behaves in the body, its role in immune system recovery, and potential long-term survival benefits. The study uses a "3+3" dose-escalation design with three dosing levels: 1 × 10^7/kg, 3 × 10^7/kg, and 6 × 10^7/kg. Patients receive a single intravenous infusion of anti-CD19 UCAR-T cells after lymphodepletion chemotherapy. Dose escalation to the next group occurs only if no dose-limiting toxicity is seen. Each dose group will enroll 3 to 6 patients, totaling 9 to 18 participants. Participants will be monitored for adverse events within 28 days and assessed over six months for disease activity, muscle inflammation by MRI, antibody and muscle enzyme changes, and physical function using the Childhood Myositis Assessment Scale. Pharmacokinetics of the infused cells will be tracked over one month, and muscle enzyme changes will be followed for one year. The study continues until long-term safety and efficacy information is collected.
CONDITIONS
Brief Title
Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 5 years or older
- Diagnosis of juvenile dermatomyositis according to Bohan and Peter criteria
- Meets classification criteria for refractory juvenile dermatomyositis with intolerance or inadequate response to glucocorticoids and at least two immunosuppressants
- Rapid disease progression or involvement of lungs, heart, or gastrointestinal tract, or calcification of tissues, or repeated skin rashes/ulcers, or persistent muscle weakness with specific assessment scores
- Patients with immune-mediated necrotizing myopathy positive for SRP or HMGCR antibodies meeting RJDM criteria
- Normal function of important organs: cardiac (LVEF ≥ 55%), renal (eGFR ≥ 30 mL/min/1.73m2), liver (AST and ALT ≤ 3.0 ULN, total bilirubin ≤ 2.0× ULN), pulmonary (SpO2 ≥ 92%)
- Female patients of childbearing age must have negative urine pregnancy test and agree to effective contraception during trial until one year after infusion
- Patient or guardian agrees to participate and signs informed consent
You will not qualify if you...
- Previous CAR-T cell therapy unless safety risks are judged eliminated
- Primary immunodeficiency or severe secondary immunodeficiency not corrected
- Severe, active, or uncontrolled infections including active tuberculosis, viral hepatitis, etc.
- Active malignant diseases or confirmed malignancies except resected basal cell carcinoma
- Congenital heart disease, recent myocardial infarction, severe arrhythmia, significant pericardial effusion, severe myocarditis, or unstable vital signs needing vasopressors
- Other diseases requiring long-term glucocorticoids or immunosuppressants
- Received solid organ or hematopoietic stem cell transplantation recently or acute graft-versus-host disease
- Positive for hepatitis B, C, HIV, or syphilis infections above normal levels
- Received live vaccines within 4 weeks before screening
- Positive blood pregnancy test
- Participation in other clinical trials within 3 months before enrollment
- Other conditions deemed unsuitable by the investigator
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment event followed by monitoring for up to 6 months
Participants receive a single infusion of CD19 UCAR-T cells intravenously after lymphodepletion chemotherapy.
Multiple visits for treatment administration and follow-up assessments over 6 months
Duration - Up to 1 year
Participants are monitored for safety, efficacy, and cell dynamics after treatment.
Regular visits during the first month and periodic visits up to 1 year
Trial Site Locations
Total: 1 location
1
Children's Hospital Zhejiang University School of Medicine, Hangzhou, zhejiang
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
L
Lu Mei ping, M.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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