ClinicalTrials.gov record NCT06686524
Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is an open-label, single-arm Phase 1 dose‑escalation study testing CD19-targeted universal CAR‑T cells in people with refractory juvenile dermatomyositis (RJDM). The trial enrolls a total of 9–18 patients across three escalating dose groups using a 3+3 design. Participants receive lymphodepletion chemotherapy followed by one intravenous CAR‑T cell infusion and are followed for safety and disease activity outcomes.
What it is studying. The study is testing the safety and effects of a single intravenous infusion of CD19-targeted universal CAR‑T cells in people with refractory juvenile dermatomyositis. It also aims to measure how the cells behave in the body (cell dynamics), how the immune system recovers, and impacts on disease activity and survival-related measures.
What the researchers hope to learn. To evaluate the safety and tolerability of anti‑CD19 UCAR‑T cells in RJDM; to assess changes in disease activity and muscle inflammation; to characterize cell kinetics (pharmacodynamics/pharmacokinetics); and to explore effects on immune reconstitution and longer‑term survival measures.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This study is an open-label, single-arm, dose-escalation trial primarily designed to evaluate the safety and efficacy of a universal CAR-T cell therapy targeting CD19 in the treatment of patients with RJDM. Additionally, the study aims to characterize its pharmacokinetic and pharmacodynamic properties, explore its role in immune system reconstitution, and assess long-term survival benefits.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Anti-CD19 UCAR-T cells
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This study adopts the "3+3" design, with a total of three dosage groups (1 × 107/kg, 3 × 107/kg, 6 × 107/kg). The study will start from the low dose group. Only when the present dose group has completed enrollment and no DLT has been observed, the study could escalate to the next dose group based on the evaluation of efficacy and cell kinetic data. If no DLT is observed even in the highest dose group, the escalation will be terminated.Each dose group is expected to enroll 3 to 6 patients, with a total of 9 to 18 patients anticipated in the trial.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After giving informed consent, participants will receive lymphodepletion chemotherapy followed by a single intravenous infusion of anti‑CD19 UCAR‑T cells. They will have repeated clinic visits for safety monitoring, blood samples to measure the infused cells and lab markers, MRI scans to assess muscle inflammation, and disease‑activity and muscle‑strength assessments (for example CMAS, DAS, TIS) over follow‑up. Female participants of childbearing potential must undergo pregnancy testing and use effective contraception for one year after infusion.
How the study is structured. Open-label, single-group Phase 1 trial using a 3+3 dose‑escalation design. There are three planned dose levels; each dose group will enroll about 3–6 patients (total 9–18). After receiving lymphodepletion chemotherapy, each participant gets one IV infusion of anti‑CD19 UCAR‑T cells. No masking is used; safety, disease activity scores, imaging, antibody and enzyme levels, and cell dynamics are measured during follow-up.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on dermatomyositis
This trial is also listed on this site under polymyositis.
Important dates
- Study start
- Nov 11, 2024 (estimated)
- Primary completion
- Nov 10, 2026 (estimated)
- Study completion
- Nov 10, 2027 (estimated)
- Target enrollment
- 18 participants (estimated)
- Last updated on registry
- Nov 13, 2024
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Chongqing Precision Biotech Co., Ltd
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Lu Mei ping, M.D 📞 +86 13685773988 ✉ meipinglu@zju.edu.cn
Participating sites (1)
- Children's Hospital Zhejiang University School of Medicine, Hangzhou, zhejiangRecruiting
Hangzhou, Zhejiang · China
- Meiping Lu, M.D · +86 13685773988 · meipinglu@zju.edu.cn
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- dose-escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- CAR-T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- pharmacokinetic
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- pharmacodynamic
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- magnetic resonance imaging
- A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- electrocardiogram
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- eGFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- DLT
- A side effect, defined in advance, that is severe enough to limit how far the dose can be raised in a dose-escalation study. How many such events stop escalation is set by the study's rules.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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