Actively Recruiting
Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis
Led by Chongqing Precision Biotech Co., Ltd · Updated on 2024-08-26
10
Participants Needed
1
Research Sites
212 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 5 and 17 years old
- Diagnosis of juvenile dermatomyositis meeting at least four of the following: symmetrical proximal muscle weakness; characteristic skin changes including purple-red eyelid rash with swelling and Gottron papules; elevated serum muscle enzyme; positive myositis antibody; electromyography showing denervation and myopathy; muscle biopsy showing necrosis and inflammation
- Refractory juvenile dermatomyositis defined by intolerance or non-response to glucocorticoids and at least two immunosuppressants with adequate therapy for at least 6 months, or rapid disease progression, or involvement of organs like lungs, heart, gastrointestinal tract, or calcification in tissues, or repeated rashes or skin ulcers
- Positive myositis-specific antibodies such as MDA-5, NXP2, TIF-1γ, Ro-52 or others
- If positive for SRP or HMGCR antibody indicating immune-mediated necrotizing myopathy equivalent to RJDM, must meet above diagnostic criteria
- Normal important organ functions including cardiac (LVEF ≥55%, normal ECG), renal (eGFR ≥30 ML/min/1.73m2), liver (AST and ALT ≤3.0 ULN, total bilirubin ≤2.0×ULN), and lung function (SpO2 ≥92%)
- Suitable for simple or intravenous blood collection with no contraindications
- Negative pregnancy test for childbearing age subjects and agreement to use effective contraception during and for 1 year after the study
- Signed informed consent from patient or guardian understanding the study purpose and procedures
You will not qualify if you...
- Previous receipt of CAR T cell therapy
- Presence of autoimmune or rheumatic diseases other than juvenile dermatomyositis
- Primary immunodeficiency or severe uncorrected secondary immunodeficiency
- Serious infectious diseases such as active or latent tuberculosis, active viral hepatitis
- Active malignant disease or diagnosis of malignant tumors except cured skin basal cell carcinoma
- Congenital heart disease, recent myocardial infarction within 6 months, severe arrhythmias, large pericardial effusion, serious myocarditis, or unstable vital signs requiring blood pressure drugs
- Other diseases requiring long-term use of glucocorticoids or immunosuppressants
- Active or uncontrolled infections needing systemic treatment within 1 week before screening
- Received solid organ or hematopoietic stem cell transplantation within 3 months before screening; recent acute graft-versus-host disease
- Positive tests for hepatitis B, hepatitis C, HIV, syphilis, or CMV with elevated viral loads
- Live vaccine received within 4 weeks before screening
- Positive pregnancy test
- Known malignant diseases before screening
- Participation in other clinical trials within 3 months prior
- Any condition deemed inappropriate for study participation by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
M
Meiping Lu, M.D
CONTACT
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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