BridgeMD
New treatment option for Juvenile Dermatomyositis (JDM)
Official title Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases
ClinicalTrials.gov ID: NCT07184450
What this study is testing
What is BCMA/CD70-targetd CAR-T?
BCMA/CD70-targetd CAR-T is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for juvenile dermatomyositis (jdm).
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 is an investigator-initiated trial to evaluate the efficacy and safety of BCMA/CD70-targeted CAR-T in the treatment of refractory pediatric rheumatic diseases.
- 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
- Age ≥5 years old.
- To meet the diagnostic criteria of refractory B-cell-related pediatric rheumatic diseases, including but not limited to juvenile dermatomyositis...
- Diagnosed as juvenile dermatomyositis(JDM) according to the criteria of Bohan and Peter, and meeting the following conditions:
- The classification criteria of RJDM must meet (1) and any one of (2)-(5): (1) Patients who are intolerant or unresponsive to glucocorticoids and at...
- RJDM with anti-synthetase syndrome who are positive for anti-synthetase antibody and those with immune-mediated necrotizing myopathy who are positive...
You likely can't join if
- Severe major organ involvement related to the primary disease, such as severe pulmonary hypertension (PHA) (mean arterial pressure \> 45mmHg).
- primary immunodeficiency or severe secondary immunodeficiency that has not been corrected.
- accompanied by serious or active or uncontrollable infectious diseases, including but not limited to active tuberculosis, latent tuberculosis...
- Evidence of active malignant disease or diagnosis of malignant tumor(including hematological malignancies and solid tumors, except resected and cured...
- Congenital heart disease or severe arrhythmias (including multisource frequent supraventricular tachycardia, ventricular tachycardia,etc.); Or...
- suffering from other diseases that require long-term use of glucocorticoids or immunosuppressants.
See the full eligibility criteria
Who can join
- Age ≥5 years old.
- To meet the diagnostic criteria of refractory B-cell-related pediatric rheumatic diseases, including but not limited to juvenile dermatomyositis, polyarticular juvenile idiopathic arthritis, systemic sclerosis, and...
- Diagnosed as juvenile dermatomyositis(JDM) according to the criteria of Bohan and Peter, and meeting the following conditions:
- The classification criteria of RJDM must meet (1) and any one of (2)-(5): (1) Patients who are intolerant or unresponsive to glucocorticoids and at least 2 immunosuppressants, and the duration of adequate hormone...
- RJDM with anti-synthetase syndrome who are positive for anti-synthetase antibody and those with immune-mediated necrotizing myopathy who are positive for SRP or HMGCR antibody can be included.
- Meet the classification criteria for polyarticular juvenile idiopathic arthritis as defined by the International League of Associations for Rheumatology(ILAR) classification in 2001, and meeting the following...
- Meet the classification criteria for Systemic sclerosis (SSc) as defined by the 2013ACR/EULAR standards, and meeting the following conditions:
- Meet the definition of intractable disease: Glucocorticoids (≥0.5mg/kg/d) and cyclophosphamide, as well as one or more of the following immunomodulators (including antimalarial drugs, azathioprine,mycophenolate mofetil...
- Modified Rodnan Skin Score (mRSS) ≥15 points (total 51 points).
- Meet the classification criteria for primary Sjogren's syndrome as defined by the 2002 ACEG classification criteria /2016 EULAR/ACR classification criteria, and meeting the following conditions:
- Meet (1) and any one of (2)-(6): (1) For those who are intolerant or have an insufficient response to glucocorticoid (prednisone 1-2 mg/kg/d or an equivalent dose of other hormones) and at least two immunosuppressants...
- Positive for anti-SSA /Ro antibody;
- ESSDAI score ≥5 points or clinESSDAI score ≥5 points.
- Positive expression of CD19 in peripheral blood B cells determined by flow cytometry, and B cells \> 5 per/uL.
- Previously not treated with CAR-T; or recurrence or poor how well it works after previous autologous or universal CD19-targeted CAR-T treatment (evaluated by the researcher).
- The functions of important organs are basically normal:
- Cardiac function: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram;
- Renal function: eGFR≥30mL/min/1.73m2;
- Liver function: AST and ALT≤3.0 ULN, total bilirubin ≤2.0×ULN;
- Lung function: SpO2≥92%.
- Meet standards for leukapheresis or intravenous blood collection, and no other contraindications for leukapheresis.
- 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...
What rules you out
- Severe major organ involvement related to the primary disease, such as severe pulmonary hypertension (PHA) (mean arterial pressure \> 45mmHg).
- primary immunodeficiency or severe secondary immunodeficiency that has not been corrected.
- accompanied by serious or active or uncontrollable 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 severe arrhythmias (including multisource frequent supraventricular tachycardia, ventricular tachycardia,etc.); Or combined with a large number of pericardial effusion, serious myocarditis...
- suffering from other diseases that require long-term use of glucocorticoids or immunosuppressants.
- Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months before screening; Acute graft-versushost 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...
- Had received live vaccine within 4 weeks prior to screening.
- Positive blood pregnancy test.
- Situations in which other investigators consider it inappropriate to participate 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.