[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"juvenile-dermatomyositis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:juvenile-dermatomyositis":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,49,89,114,141,179],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":31,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":40,"startDateStruct":43,"completionDateStruct":4,"leadSponsor":45,"locationsCount":48},"100153996","environmental-risk-factors-for-the-anti-synthetase-syndrome-100153996",false,"NCT01276470","Environmental Risk Factors for the Anti-synthetase Syndrome","Environmental Risk Factors for the Anti-Synthetase Syndrome","* INCLUSION CRITERIA:\n\nThere are no gender or ethnic restrictions to enrollment in the study. Age restrictions for children do apply to enrollment in the study.\n\nThe inclusion criteria for enrollment of myositis subjects are:\n\n1. Diagnosis of myositis based on criteria for possible, probable or definite PM or DM, with or without other connective tissue diseases, documented within 24 months of enrollment (using the most recent diagnosis date to define the 24-month period).\n2. CXR to assess possible ILD and assign the subject to the presumptive anti-synthetase positive or negative category if clinically indicated.\n3. Children must be greater than two years of age.\n4. Able and willing to give informed consent, to complete the questionnaires and to donate blood samples (in case of children greater than 2 years of age but \\\u003C18 years of age, parent\u002Flegal guardian must be willing and able to provide informed consent and child will provide assent according to child s maturity level and understanding).\n\nThe exclusion criteria for myositis subjects are:\n\n1. Cancer-associated myositis (cancer diagnosed within 2 years of the diagnosis of myositis).\n2. Inclusion body myositis.\n3. Myositis that has clearly developed as the result of a drug, toxin or other exposure and has resolved after discontinuation of the exposure to that agent.\n4. Children less than 2 years of age.\n\nThe inclusion criteria for controls are:\n\n1. Friends or, if friends are not available, cousins of the anti-synthetase-positive myositis patient, or, if friends or cousins are not available, volunteers from the general community (such as the NIH Normal volunteer program), race- gender- and age- (within 5 years for minors and within 10 years for adults) matched, and when possible who is living as close as possible to the geographic area of the myositis patient.\n2. Controls should be without a recognized autoimmune disease or ILD.\n3. Able and willing to give informed consent, to complete the questionnaires and to donate blood samples (in case of children greater than 2 years of age but \\\u003C18 years of age, parent\u002Flegal guardian must be willing and able to provide informed consent) and child will provide assent according to child maturity level and understanding).\n\nThe exclusion criteria for all protocol subjects are:\n\n1. Medical illness that in the judgment of the investigators does not allow safe blood draws or other clinical evaluations needed for study participation.\n2. Cognitive impairment.\n3. Not able or willing to give informed assent or consent.\n4. Children less than 2 years of age.\n5. Patients who at their reference date were not in the US or Canada\n6. Individuals currently incarcerated\n\nHIV considerations:\n\nHIV is not an exclusion for affected participants in this study for the two following reasons:\n\n1. It has no impact on study procedures or tests.\n2. It may be one of the viral risk factors we are investigating.",true,"ALL","2 Years","100 Years",{"count":21,"type":22},580,"ESTIMATED","OBSERVATIONAL","Background:\n\n* Like other complex diseases, autoimmune diseases are the result of numerous causes, including genetic and environmental factors. Some researchers believe that people who are susceptible to autoimmune disorders develop them when the body reacts to environmental or other factors by creating white blood cells that attack the body s own tissues, which then progresses to autoimmune diseases. These immune-triggered disorders can overlap with one another to some extent, but most autoimmune diseases have certain distinct triggers.\n* The autoimmune disorder myositis weakens the muscles and may cause other health problems. Environmental exposures associated with myositis include ultraviolet radiation, stressful life events and muscle overexertion, collagen implants, infections such as retroviruses and streptococci bacteria, and certain drugs and chemicals. Some individuals with myositis also produce proteins in the blood called autoantibodies that react with certain parts of the person s own cells, called synthetases, which are involved in making new proteins. A syndrome called the anti-synthetase syndrome, which includes myositis and lung disease, is associated with having the anti-synthetase autoantibodies. Researchers are interested in studying differences in environmental exposures in individuals with myositis. This study is being conducted to determine if persons with the anti-synthetase syndrome have had different environmental exposures before disease onset compared with other patients with myositis who do not have this syndrome and also compared with healthy volunteers.\n\nObjectives:\n\n\\- To determine whether selected infectious and noninfectious environmental exposures are more common in individuals who have myositis with the anti-synthetase syndrome, compared with healthy volunteers.\n\nEligibility:\n\n\\- Individuals who have been diagnosed with myositis (with or without anti-synthetase autoantibodies), and healthy volunteers without autoimmune disorders.\n\nDesign:\n\n* Participants will be screened with a full medical history and physical examination, and will provide blood, urine and house dust samples.\n* Participants will complete questionnaires about their medical history and the types of exposures they have had at work, at home, and elsewhere. Participants who have myositis will also be asked about certain infections, heavy exercise or physical exertion, sun exposure, tobacco and alcohol use, and stressful events prior to being diagnosed with the disease. Healthy volunteers will be asked about the same exposures before the date of diagnosis of disease of the myositis subject to which they have been matched.\n* Participants will receive a kit that contains instructions and a filter to be put onto their vacuum cleaner to collect house dust in the bedroom. This dust will be kept for possible future analyses of infectious or toxic agents based on the other results from the study.\n* Individuals with myositis will have other tests as clinically indicated, including lung function tests and imaging studies.",[26,27,28,29,30],"Myositis","Dermatomyositis","Polymyositis","Juvenile Dermatomyositis","Juvenile Polymyositis",[32,33,34,35,36,37],"Adult and Pediatric Autoimmune Disease","Antibody","Autoimmune Diseases","Autoimmunity Pathogenesis","Environmental Risk Factors","Natural History","RECRUITING","2026-06-11",{"date":41,"type":42},"2026-06-12","ACTUAL",{"date":44,"type":42},"2011-02-09",{"name":46,"class":47},"National Institute of Environmental Health Sciences (NIEHS)","NIH",8,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":17,"minAge":56,"maxAge":57,"enrollmentInfo":58,"targetDuration":4,"studyType":60,"phases":61,"briefSummary":64,"conditions":65,"keywords":71,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":78,"lastUpdatePostDateStruct":79,"startDateStruct":81,"completionDateStruct":83,"leadSponsor":85,"locationsCount":88},"100527937","phase-2-reset-myositis-an-open-label-study-to-evaluate-the-safety-and-efficacy-of-caba-201-in-subjects-with-active-idiopathic-inflammatory-myopathy-or-juvenile-idiopathic-inflammatory-myopathy-100527937","NCT06154252","RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy","A Phase 1\u002F2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy","Adult Cohorts\n\nInclusion Criteria:\n\n* Age ≥18 and ≤75\n* A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism\u002FAmerican College of Rheumatology classification criteria\n* Diagnosis of DM, ASyS, or IMNM\n* Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated creatine kinase (CK), DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography\n* Presence of muscle weakness\n\nOther protocol-defined criteria apply.\n\nExclusion Criteria:\n\n* Contraindication to leukapheresis\n* History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites\n* Active infection requiring medical intervention at screening\n* Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections\n* Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures\n* Significant lung or cardiac impairment\n* Previous CAR T cell therapy\n* Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant\n\nOther protocol-defined criteria apply.\n\nJuvenile Cohort\n\nInclusion Criteria:\n\n* Age ≥6 and ≤17 years at enrollment\n* A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism\u002FAmerican College of Rheumatology classification criteria\n* Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated muscle enzymes, DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography\n\nOther protocol-defined criteria apply.\n\nExclusion Criteria:\n\n* Contraindication to leukapheresis\n* History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites\n* Active infection requiring medical intervention at screening\n* Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.\n* Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures\n* Significant lung or cardiac impairment\n* Previous CAR T cell therapy\n* Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant\n\nOther protocol-defined criteria apply.","6 Years","75 Years",{"count":59,"type":22},74,"INTERVENTIONAL",[62,63],"PHASE2","PHASE3","RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy",[66,27,67,68,29,30,69,70],"Idiopathic Inflammatory Myopathy","Anti-Synthetase Syndrome","Immune-Mediated Necrotizing Myopathy","Juvenile Idiopathic Inflammatory Myopathy (JIIM)","Juvenile Myositis",[72,73,74,75,66,26,27,76,77,29,30,69,70],"CABA-201","Autoimmune Disease","Anti-CD19 CAR-T therapy","Cellular Therapy","Anti-synthetase Syndrome","Immune-mediated Necrotizing Myopathy","2026-06-08",{"date":80,"type":42},"2026-06-10",{"date":82,"type":42},"2023-12-20",{"date":84,"type":22},"2028-07",{"name":86,"class":87},"Cabaletta Bio","INDUSTRY",35,{"id":90,"slug":91,"hasResults":11,"nctId":92,"briefTitle":93,"officialTitle":94,"acronym":4,"eligibilityCriteria":95,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":96,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":98,"conditions":99,"keywords":101,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":106,"lastUpdatePostDateStruct":107,"startDateStruct":109,"completionDateStruct":4,"leadSponsor":111,"locationsCount":113},"100062399","studies-of-the-natural-history-pathogenesis-and-outcome-of-autoinflammatory-diseases-including-juvenile-dermatomyositis-100062399","NCT00059748","Studies of the Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases Including Juvenile Dermatomyositis","Studies of Natural History, Pathogenesis, and Outcomes in Autoimmune and Inflammatory Diseases Including Juvenile Dermatomyositis","* INCLUSION CRITERIA:\n* Patients with CANDLE, SAVI, and JDM who are mutation positive for the disease or fulfill clinical criteria of the disease.\n* Patients who fulfill criteria for definite or probable JDM\n* Patients with other suspected autoinflammatory diseases\n* There is:\n\n  * 2 years old or older\n  * Patients or their legal guardians need to be able and willing to give informed consent and a pediatric patient needs to be willing to assent to the protocol whenever possible.\n\nRelatives of patients with autoinflammatory diseases or healthy volunteers may be included for genetic testing. The genetic evaluations will be conducted in collaboration with Dr. Fleisher's laboratory at the Clinical Center laboratory and other groups. We may also collect blood for serum and RNA analyses to establish a cohort of healthy controls that is matched in age, gender and ethnicity to the study patients. Skin biopsies for research may be requested from patients, patient relatives and healthy volunteers\n\nEXCLUSION CRITERIA:\n\n* Active malignancy or any medical condition that in the opinion of the investigator would warrant exclusion\n* Inability to return for follow up visits\n* Pregnancy\n* Adults without the capacity to consent",{"count":97,"type":22},5000,"Purpose:\n\nThe purpose of this protocol is 1. To comprehensively evaluate patients with autoinflammatory diseases clinically, genetically and immunologically at the autoinflammatory disease clinic at the NIH. 2. To follow patients with autoinflammatory Diseases that are genetically defined including Neonatal-Onset Multisystem Inflammatory Disease (NOMID), the most severe clinical phenotype of Cryopyrin-Associated Periodic Syndromes (CAPS), Deficiency of IL-1 Receptor Antagonist (DIRA), Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated temperatures (CANDLE), and STING-Associated Vasculopathy with onset in Infancy (SAVI), and those with genetically undefined autoinflammatory disorders to determine long-term disease outcomes. 3. To develop biomarkers that help us assess disease activity and response to treatment. 4. To assess the eligibility of affected patients for inclusion in ongoing and planned treatment protocols.\n\nGoal: The goals of our studies are to understand the underlying immune dysregulation, to identify the genetic cause and to translate our findings into novel treatments that improve patients disease outcome.\n\nEligibility:\n\n* Patients with known NOMID\u002FCAPS, DIRA, CANDLE, SAVI, CRMO, Still's Disease, and with other yet undifferentiated autoinflammatory diseases.\n* Healthy adult and pediatric relatives.\n* Volunteers\n\nDesign:\n\nParticipants will be evaluated at the NIH for 2-5 days. All participants will have a detailed medical history, physical exam, blood tests and other evaluations depending on the extend of their autoinflammatory disease.\n\nParticipants may also expect the following assessments:\n\n1. Clinical test that help assess organ damage and functional impact such as hearing vision, memory and learning tests.\n2. Imaging studies to characterize the organ involvement of the inflammatory disease including: X-rays, CT scans, special MRIs, bone scans.\n3. Laboratory evaluations including clinical markers of disease activity, research samples for genetic studies, and blood samples for cytokine\u002Fbiomarker assessment, and gene expression profiling.\\\u003CTAB\\>\n4. Completion of questionnaires to assess disease activity and quality of life.\n5. If indicated, other procedures may be administered that include: a lumbar puncture if CNS inflammation is suspected and a skin biopsy if skin inflammation is present. other gastrointestinal procedures as they are clinically indicated.\n6. Patients my have a research skin biopsy taken.\n\nParticipants may return for a single follow-up visits or for long term-follow up depending on their disease and willingness to be followed long-term.",[100,29],"Autoinflammatory Disease",[26,102,103,104,105,37],"JDM","Immune Dysregulation","Skin Rash","Inflammation","2026-06-04",{"date":108,"type":42},"2026-06-05",{"date":110,"type":42},"2003-05-09",{"name":112,"class":47},"National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)",1,{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":17,"minAge":121,"maxAge":4,"enrollmentInfo":122,"targetDuration":4,"studyType":60,"phases":124,"briefSummary":126,"conditions":127,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":132,"startDateStruct":134,"completionDateStruct":136,"leadSponsor":138,"locationsCount":140},"100599794","phase-1-descartes-08-for-children-adolescents-and-young-adults-with-autoimmune-disorders-100599794","NCT07089121","Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders","Descartes-08 for Children, Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus, ANCA-associated Vasculitis, Juvenile Myasthenia Gravis, and Juvenile Dermatomyositis","Inclusion Criteria:\n\n* At least age 12\n* definitive diagnosis of childhood-onset systemic lupus erythematous, juvenile Myasthenie gravis, juvenile dermatomyositis and AAV\n* Signs and symptoms of moderate disease\n* History of systemic treatment\n* Parent\u002FGuardian\u002FPatient must be able to give written informed consent\n\nExclusion Criteria:\n\n* Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient;\n* Abnormal PT\u002FINR or PTT increased \\> 1.5-fold or patient is on anticoagulation therapy (except in cases of elevated PTT with documented lupus anticoagulant; or in patients who have been on stable doses of anticoagulation therapy for more than 6 months of VTE diagnosis; or in patients on stable doses of anticoagulation therapy for at least 8 weeks of atrial fibrillation diagnosis; these conditions will not be exclusionary unless, in the investigator's opinion, they make participation in the study unsafe);\n* ANC \\\u003C 1000 cells\u002Fmicroliter ;\n* Hemoglobin \\\u003C 8.0 g\u002FdL ;\n* Platelets \\\u003C 50,000\u002Fmm3 (NOTE: platelet transfusions are permissible);\n* ALT and\u002For AST with GGT ≥ 3× upper limit of normal\n* Creatine Clearance less than 30mL\u002Fmin \u002F1.73 m2;\n* History of primary immunodeficiency, organ, or allogeneic bone marrow transplant;\n* Patients must be seronegative for hepatitis B surface antigen;\n* Patients must be seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patients must be tested for the presence of viremia by RT-PCR and must be HCV RNA negative;\n* History of positive HIV or positive HIV at screening;\n* Active tuberculosis or positive QuantiFERON test at screening;\n* Any other laboratory abnormality that, in the opinion of the investigator, may jeopardize the subject's ability to participate in the study; 23. Any active significant cardiac or pulmonary disease not related to the primary indication as determined by principal investigator and medical monitor Note: Patients with asthma and COPD controlled with inhaled medications are allowed; 24. Any arterial or venous thromboembolic events in the past 3 months; 25. History of malignancy that required treatment in the past 3 years except for successfully-treated squamous cell and\u002For basal cell carcinoma of the skin and\u002For breast or colon cancer that is surgically removed and did not require adjuvant chemotherapy or radiotherapy; 26. Treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer); 27. Receipt of a live vaccination within 4 weeks prior to baseline (Day 1) or intent to receive live vaccination during the study (Note: mRNA-based vaccines such as those against SARS-CoV-2 are not considered live; likewise, the Janssen Covid-19 vaccine is not live); 28. History of significant recurrent infections or any active infection that may interfere with the patient's participation in the opinion of the investigator; 29. Any known psychiatric illness that may interfere with the patient's participation in the study in the opinion of the investigator.","12 Years",{"count":123,"type":22},50,[125,62],"PHASE1","Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis",[128,129,130,29],"Childhood-onset Systemic Lupus Erythematous","ANCA-Associated Vasculitis (AAV)","Juvenile Myasthenia Gravis","2026-04-07",{"date":133,"type":42},"2026-04-08",{"date":135,"type":42},"2026-01-14",{"date":137,"type":22},"2028-12",{"name":139,"class":87},"Cartesian Therapeutics",2,{"id":142,"slug":143,"hasResults":11,"nctId":144,"briefTitle":145,"officialTitle":146,"acronym":147,"eligibilityCriteria":148,"healthyVolunteers":11,"sex":17,"minAge":149,"maxAge":4,"enrollmentInfo":150,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":152,"conditions":153,"keywords":159,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":169,"lastUpdatePostDateStruct":170,"startDateStruct":172,"completionDateStruct":174,"leadSponsor":176,"locationsCount":113},"100621706","mihra---patient-rooted-insights-for-shaping-myositis-science-prisms-100621706","NCT07374107","MIHRA - Patient-Rooted Insights for Shaping Myositis Science (PRISMS)","MIHRA - Patient-Rooted Insights for Shaping Myositis Science (PRISMS) - A Qualitative Study of Patient-voiced Research Priorities Across Rare Myositis Diseases","MIHRA-PRISMS","Inclusion Criteria:\n\n* Ability to provide informed consent\n* Have a clinician diagnosis of an idiopathic inflammatory myopathy or be a care partner or parent of a person living with an idiopathic inflammatory myopathy.\n* Participants who may have signed up through the MIHRA Patient Contact Registry https:\u002F\u002Fmihrafoundation.org\u002Fmihra-programs\u002Fmihra-patient-contact-registry\u002F\n\nExclusion Criteria:\n\n* Under the age of 7 years old\n* Do not have a diagnosis of an inflammatory myopathy","7 Years",{"count":151,"type":22},700,"Myositis diseases are each rare diseases. As in other rare diseases, people living with myositis diseases face physical and psychosocial challenges that may not be recognized in current research priorities. The PRISMS study is a global investigation that collects patient perspectives through (mostly online) methods of open-ended questions, community forums and survey to identify the most pressing research concerns as identified by patients. Findings will be analyzed to create a patient-voiced set of research priorities that can guide the direction of research and help inform funding decisions across myositis diseases.\n\nPotential participants can express interest via https:\u002F\u002Fmihrafoundation.org\u002Fmihra-programs\u002Fmihra-patient-contact-registry\u002F",[154,155,26,156,27,157,76,68,28,158,70,29],"IBM","IIM","Inflammatory Myopathy","Dermatomyositis, Juvenile","Inclusion Body Myositis",[160,161,162,163,164,165,166,167,168],"myositis","rare diseases","patient priorities","patient engagement","patient initiated","patient research partners","qualitative research","mixed methods research","research priorities","2026-01-20",{"date":171,"type":42},"2026-01-28",{"date":173,"type":42},"2025-06-25",{"date":175,"type":22},"2030-12",{"name":177,"class":178},"Myositis International Health & Research Collaborative Alliance Foundation","OTHER",{"id":180,"slug":181,"hasResults":11,"nctId":182,"briefTitle":183,"officialTitle":184,"acronym":4,"eligibilityCriteria":185,"healthyVolunteers":11,"sex":17,"minAge":186,"maxAge":187,"enrollmentInfo":188,"targetDuration":4,"studyType":60,"phases":190,"briefSummary":191,"conditions":192,"keywords":193,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":196,"lastUpdatePostDateStruct":197,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":113},"100559848","phase-1-clinical-trial-of-cd19-targeted-car-t-therapy-for-refractory-juvenile-dermatomyositis-100559848","NCT06569472","Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis","Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Juvenile Dermatomyositis (RJDM)","Inclusion Criteria:\n\n1. Age: ≥5 years and \\\u003C17 years old\n2. 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.\n3. 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\u002For involves organs such as lungs, heart and gastrointestinal tract; ③ Calcification of subcutaneous or muscle and joint tissues; ④ Repeated rashes or skin ulcers.\n4. myositis specific antibody positive, defined as MDA-5, NXP2, TIF-1γ, Ro-52 and any other positive;\n5. 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.\n6. The functions of important organs are basically normal:\n\n   ① Cardiac function: left ventricular ejection fraction (LVEF) ≥55%, no obvious abnormality in electrocardiogram;\n\n   ② Renal function: eGFR≥30ML\u002Fmin\u002F1.73m2;\n\n   ③ Liver function: AST and ALT≤3.0 ULN, total bilirubin ≤2.0×ULN;\n\n   ④ Lung function: Lung function is basically normal, SpO2≥92%;\n7. Have the criteria for simple or intravenous blood collection, and no other contraindications for cell collection;\n8. 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;\n9. The patient or his\u002Fher guardian agrees to participate in this clinical trial and signs an informed consent indicating that he\u002Fshe understands the purpose and procedure of this clinical trial and is willing to participate in the study.\n\nExclusion Criteria:\n\n1. Had previously received CAR T cell therapy;\n2. Have other autoimmune or rheumatic diseases other than JDM;\n3. primary immunodeficiency or severe secondary immunodeficiency that has not been corrected;\n4. accompanied by serious infectious diseases, including but not limited to active tuberculosis, latent tuberculosis infection, active viral hepatitis, etc.;\n5. Evidence of active malignant disease or diagnosis of malignant tumor (including hematological malignancies and solid tumors, except resected and cured skin basal cell carcinoma)\n6. 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;\n7. suffering from other diseases that require long-term use of glucocorticoids or immunosuppressants;\n8. There is an active or uncontrollable infection that requires systemic treatment within 1 week prior to screening;\n9. 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;\n10. 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;\n11. Had received live vaccine within 4 weeks prior to screening;\n12. Positive blood pregnancy test;\n13. Patients with known malignant diseases such as tumors before screening;\n14. Patients who had participated in other clinical trials within 3 months prior to enrollment;\n15. Situations in which other investigators consider it inappropriate to participate in the study.","5 Years","17 Years",{"count":189,"type":22},10,[125],"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).",[29],[194,195],"CAR-T","CD19","2024-08-23",{"date":198,"type":42},"2024-08-26",{"date":200,"type":42},"2024-07-02",{"date":202,"type":22},"2028-07-31",{"name":204,"class":87},"Chongqing Precision Biotech Co., Ltd"]