ClinicalTrials.gov record NCT06569472

Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Juvenile Dermatomyositis (RJDM)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.The registry record was last updated on Aug 26, 2024, about 2 years ago, so the status shown may be out of date. The study team or the registry can confirm.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age5 years old and older
SexAll
LocationChina
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 Phase 1 interventional study tests CD19-targeted CAR-T cells in children aged 5 to 17 with refractory juvenile dermatomyositis. The study has two parts: dose exploration (Part A) and dose extension (Part B). Participants receive lymphocyte‑depleting chemotherapy followed by a single intravenous CAR‑T infusion and are monitored for safety and early signs of disease improvement, with follow-up for up to 2 years.

What it is studying. The study is evaluating the safety, tolerability, and initial effectiveness of a single infusion of CD19-targeted CAR-T cells in people with refractory juvenile dermatomyositis. It also measures how the infused CAR-T cells behave in the blood over time.

What the researchers hope to learn. To assess safety and tolerability of CD19 CAR-T in refractory juvenile dermatomyositis using standard adverse-event criteria; to estimate the proportion of participants who show disease improvement and measure duration of response up to 2 years; and to characterize CAR‑T cell dynamics (area under the curve, peak level, and time to peak) over the first months after infusion.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • CD19-targetd CAR-T

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Age: ≥5 years and <17 years old
  • To meet the diagnostic criteria of JDM, four or five of the following criteria must be met:① symmetrical proximal muscle weakness; ②Characteristic skin changes, including positive dermatitis (purplish red rash on upper eyelid with periorbital edema) and Gottron papules (red patchy squamous papules on the back of knuckles); ③ The level of one muscle enzyme in serum was increased; ④ Positive myositis antibody; ⑤Electromyography shows denervation and myopathy; ⑥ Muscle biopsies showed necrosis and inflammation.
  • The classification criteria of RJDM must meet ① and any of the criteria②-④: ① Patients who are intolerant or unresponsive to glucocorticoids and at least 2 immunosuppressants, adequate hormone therapy and duration of at least 6 months; ② The disease progresses rapidly and/or involves organs such as lungs, heart and gastrointestinal tract; ③ Calcification of subcutaneous or muscle and joint tissues; ④ Repeated rashes or skin ulcers.
  • myositis specific antibody positive, defined as MDA-5, NXP2, TIF-1γ, Ro-52 and any other positive;
  • If the patient has SRP or HMGCR antibody positive immune-mediated necrotizing myopathy equivalent to RJDM, the inclusion criteria of (2) - (4) can be met.
  • The functions of important organs are basically normal:
  • ① Cardiac function: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram;
  • ② Renal function: eGFR≥30ML/min/1.73m2;
  • ③ Liver function: AST and ALT≤3.0 ULN, total bilirubin ≤2.0×ULN;
  • ④ Lung function: Lung function is basically normal, SpO2≥92%;
  • Have the criteria for simple or intravenous blood collection, and no other contraindications for cell collection;
  • The subject of childbearing age has a negative urine pregnancy test result and agrees to take effective contraceptive measures during the test period until 1 year after the infusion;
  • The patient or his/her guardian agrees to participate in this clinical trial and signs an informed consent indicating that he/she understands the purpose and procedure of this clinical trial and is willing to participate in the study.

Exclusion criteria

  • Had previously received CAR T cell therapy;
  • Have other autoimmune or rheumatic diseases other than JDM;
  • primary immunodeficiency or severe secondary immunodeficiency that has not been corrected;
  • accompanied by serious infectious diseases, including but not limited to active tuberculosis, latent tuberculosis infection, active viral hepatitis, etc.;
  • Evidence of active malignant disease or diagnosis of malignant tumor (including hematological malignancies and solid tumors, except resected and cured skin basal cell carcinoma)
  • Congenital heart disease or history of acute myocardial infarction within 6 months before screening, or severe arrhythmias (including multi-source frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with a large number of pericardial effusion, serious myocarditis, etc.; Or patients with unstable vital signs who need hypertensive drugs to maintain their blood pressure;
  • suffering from other diseases that require long-term use of glucocorticoids or immunosuppressants;
  • There is an active or uncontrollable infection that requires systemic treatment within 1 week prior to screening;
  • Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; Acute graft-versus-host disease (GVHD) of grade 2 or above was present within 2 weeks prior to screening;
  • Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal reference value range; Or hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Or positive for human immunodeficiency virus (HIV) antibodies; Or syphilis test positive; Or cytomegalovirus (CMV) DNA test positive;
  • Had received live vaccine within 4 weeks prior to screening;
  • Positive blood pregnancy test;
  • Patients with known malignant diseases such as tumors before screening;
  • Patients who had participated in other clinical trials within 3 months prior to enrollment;
  • Situations in which other investigators consider it inappropriate to participate in the study.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

juvenile dermatomyositis (JDM) is a non-suppurative chronic autoimmune disease and the most common type of juvenile idiopathic inflammatory myopathy.Despite the active treatment measures, some JDM patients are still intolerant or unresponsive to the treatment, resulting in a high disability and mortality rate.Because CD19 is widely expressed on the surface of B lymphocytes, CD19 CAR-T can also cause deep depletion of other CD19+ B cells while killing cancer cells, which is expected to achieve immune reconstruction in patients with autoimmune diseases, and completely change the status quo that such patients need to take hormones and immunosuppressants for a long time.

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.

Participants will provide blood for manufacturing CAR‑T cells, receive lymphocyte‑clearing (lymphodepleting) chemotherapy, then a single intravenous infusion of CD19‑targeted CAR‑T cells. They will have short-term safety monitoring (including checks in the first 28 days), disease assessments around 2–3 months, blood tests to measure CAR‑T cell levels, and longer follow‑up of response (up to about 2 years as described). Pregnancy testing and contraception monitoring are required for those of childbearing potential, and written informed consent/assent is required.

How the study is structured. Phase 1, interventional, single-group study with no masking. The trial is divided into dose‑finding (Part A) and dose‑extension (Part B). Participants undergo lymphocyte‑depleting chemotherapy and then receive one intravenous infusion of CD19-targeted CAR-T cells. Follow-up includes monitoring for adverse events, assessments of disease improvement, and measurements of CAR‑T cell levels.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Juvenile Dermatomyositis. Does it apply to my diagnosis?
  • 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 polymyositis

This trial is also listed on this site under dermatomyositis.

Clinical trials55 trials listed for polymyositisEvery trial the registry lists for polymyositis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in polymyositis researchLatest monthly update: September 2026. Includes: CD4 count risk. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jul 2, 2024
Primary completion
Jul 31, 2027 (estimated)
Study completion
Jul 31, 2028 (estimated)
Target enrollment
10 participants (estimated)
Last updated on registry
Aug 26, 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.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

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.
refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
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.
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.
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.
ULN
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.
contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
informed consent
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.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

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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