BridgeMD
New treatment option for Dermatomyositis, Juvenile
Official title Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)
ClinicalTrials.gov ID: NCT06686524
What this study is testing
What is Anti-CD19 UCAR-T cells?
Anti-CD19 UCAR-T cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for dermatomyositis, juvenile.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 and older
You may be able to join if
- (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...
- Rapid progression of the disease and/or involvement of organs such as lungs, heart, and gastrointestinal tract;
You likely can't join if
- people 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...
- (4) Known to have active malignant diseases or confirmed malignancies before screening (including hematological malignancies and solid tumors, except...
- (5) Suffering from congenital heart disease or having a history of acute myocardial infarction within 6 months, or severe arrhythmia (including...
See the full eligibility 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 \ 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 people 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...
- people 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...
- (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...
- (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...
- (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.
The study team makes the final eligibility decision.
Where it's taking place
- Hangzhou, Zhejiang, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 5 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hangzhou, Zhejiang, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.