ClinicalTrials.gov record NCT07715136
BCMA-CD19 cCAR-T for the Treatment of Refractory Inflammatory Myopathy
A Single-arm, Open-label Phase I Clinical Study to Evaluate ICG318 CAR-T in Adults With Refractory Inflammatory Myopathy
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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 a Phase 1, single-arm, open-label study testing the safety and tolerability of ICG318 CAR‑T (BCMA‑CD19‑IL‑15/IL‑15sushi) in adults with refractory idiopathic inflammatory myopathy. Participants receive cyclophosphamide lymphodepletion followed by one CAR‑T infusion. Safety, laboratory tests, and disease assessments are collected and participants are followed for up to 2 years.
What it is studying. The study is testing the safety and tolerability of ICG318 CAR‑T, a compound CAR‑T cell product that targets CD19 and BCMA, given once after cyclophosphamide lymphodepletion in adults with refractory inflammatory myopathy.
What the researchers hope to learn. To evaluate safety and tolerability (record adverse events up to 2 years), determine a recommended Phase 2 dose, and collect data on disease activity, muscle strength, laboratory markers, pharmacokinetics, and immune cell and antibody measures.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This single-arm, open-label, phase I trial evaluates the safety and tolerability of ICG318 CAR-T (BCMA-CD19-IL-15/IL15sushi cCAR T cells) in patients with refractory Idiopathic inflammatory myopathy (IIM).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- ICG318, BCMA-CD19-IL-15/IL-15 sushi Compound 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 18-60 (age ≥18 years at first symptom onset), gender not limited;
- The diagnosis meets the criteria for IIM according to the 2017 EULAR/ACR criteria and is confirmed to be of the following subtype:
- a. Dermatomyositis: i. Presence of Gottron's rash or sunspot rash; ii. At least one serological result is positive: anti-transcriptional mediator (TIF1-gamma/P155) antibody, anti-matrix protein 2 (NXP2/P140) antibody, anti-Mi2 antibody, anti-melanoma differentiation-associated gene 5 (MDA5) antibody, anti-small ubiquitin-like modifier-1 activator (SAE1) antibody and/or SAE2 antibody, anti-histyl-tRNA synthetase (Jo-1) antibody, anti-alanyl-tRNA synthetase (PL-12) antibody, anti-threonyl-tRNA synthetase (PL-7) antibody, anti-glycyl-tRNA synthetase (EJ) antibody, anti-leucyl-tRNA synthetase (0J) antibody; b. Immune-mediated necrotizing myopathy: i. No characteristic skin manifestations of dermatomyositis were observed (i.e. Gottron's rash or sun-facing rash); ii. Muscle weakness characterized by IIM (e.g., symmetrical proximal muscle weakness of the upper/lower limbs; or more pronounced weakness of the neck flexors than the neck extensors; or more pronounced proximal muscle weakness of the lower limbs than distal muscle weakness).
- iii. At least one serological result must be positive: anti-human signal recognition particle (SRP) antibody, or trihydroxytrimethylcoenzyme A reductase (HMGCR) antibody; c. Antisynthetic enzyme syndrome: i. At least one of the following clinical manifestations must be present: Raynaud's phenomenon, arthritis, interstitial lung disease, fever (with no other cause of fever found), or technician's hand (thickened and cracked skin on the hands, especially the fingertips).
- ii. At least one serological result is positive: anti-histyl-tRNA synthetase (Jo-1) antibody, anti-threonyl-tRNA synthetase (PL-7) antibody, anti-alanyl-tRNA synthetase (PL-12) antibody, anti-glycyl-tRNA synthetase (EJ) antibody, anti-leucyl-tRNA synthetase (OJ) antibody, anti-aspartyl-tRNA synthetase (KS) antibody, anti-phenylalanyl-tRNA synthetase (Zo) antibody, anti-glutamyl-tRNA synthetase (JS) antibody, anti-lysyl-tRNA synthetase (SC) antibody, anti-tyrosyl-tRNA synthetase (YRS) antibody;
- Refractory IIM: At least one round of high-dose corticosteroid therapy and/or intravenous immunoglobulin, and at least two immunosuppressants (including but not limited to cyclophosphamide, mycophenolate mofetil, tacrolimus, cyclosporine, and azathioprine) have been used in adequate doses for more than 6 months;
- At the time of enrollment, participants must be receiving background medication at a stable dose, defined as: receiving background therapy with one oral glucocorticoid and/or one immunosuppressant at a stable dose for ≥ 12 weeks;
- Severity of enrollment met the following criteria:
- MMT-8 score ≤141 (out of 150);
- Students must meet at least one of the following CSM numerical scale criteria based on abnormal visual analog scale (VAS) scores: Overall activity assessed by study participants ≥2; overall disease activity assessed by physicians ≥2; extra-muscular activity (MDAAT) ≥2; HAQ-DI ≥0.25;
- At least one muscle enzyme level >1.5 times ULN;
- Through examinations such as tumor markers, lung and abdominal CT scans, thyroid ultrasound, breast ultrasound, and gastroscopy and colonoscopy, possible potential tumors have been ruled out.
Exclusion criteria
- During the screening period, individuals with active viral, bacterial, or other infections requiring systemic treatment are excluded.
- The presence of a serious, poorly controlled comorbidity that the investigator considers clinically significant, such as (but not limited to) neurological, cardiovascular, renal, hepatic, endocrine, or gastrointestinal disorders;
- Patients with severe organ dysfunction:
- The estimated glomerular filtration rate (eGFR) using the MDRD formula is <40 ml/min/1.73m² ; [eGFR = 186 × (age) - 0.203 × SCr - 1.154 (mg/dl), for women, the result is multiplied by 0.742].
- Study participants with total bilirubin >1.5 times the upper limit of normal (no specific restrictions are made for liver enzymes because inflammatory myopathy can cause elevated ALT/AST).
- Cardiovascular: Assessed by echocardiography (ECHO) or cardiac radionuclide angiography (MUGA), with a left ventricular ejection fraction (LVEF) <50% and oxygen saturation <94%. Alternatively, patients may have poorly controlled hypertension or other conditions, or have NYHA class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant heart disease.
- Bone marrow function: Absolute neutrophil count (ANC) <1×10⁹/L; Absolute lymphocyte count (ALC) <0.1×10⁹/L; Platelet count <50×10⁹/L; Hemoglobin <8.0 g/dL;
- Research participants who have a history of or current malignancy;
- Infectious diseases: Research participants with active hepatitis B (defined as positive hepatitis B surface antigen or positive hepatitis B core antibody, with hepatitis B virus DNA detection value >1000 copies/mL) or hepatitis C (HCV RNA positive); research participants with positive HIV antibody or positive treponema pallidum antibody and positive non-treponemal syphilis antibody test; active tuberculosis (negative interferon release; if positive, chest X-ray or CT scan is required to rule out active infection);
- Central nervous system diseases, including cerebrovascular diseases, epilepsy, etc.;
- Allergies to components of treatment or pre-conditioning.
- Those with elective surgery planned during the study period;
- Those who do not wish to take necessary contraceptive measures during the research period;
- Any other situation that researchers deem unsuitable for participation in the study including poor compliance and being involved in another clinical study.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
Idiopathic inflammatory myopathy (IIM) is a group of chronic autoimmune diseases with the most common types in adults being dermatomyositis, immune-mediated necrotizing myopathy, polymyositis, and antisynthetase syndrome. IIM is driven by humoral immune cell dysfunction including B cells, plasma cells and long-lived plasma cells. ICG318 CAR-T, the investigational agent in this clinical trial, is an armored, compound chimeric antigen receptor (cCAR) composed of two independently functioning CARs that target the CD19 and BCMA surface antigens on B cells and plasma/long-lived plasma cells, respectively. This study is being conducted to evaluate the safety and tolerability of ICG318 CAR-T in patients with refractory IIM. A single dose of ICG318 CAR-T will be evaluated after cyclophosphamide lymphodepletion.
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 have screening tests to confirm diagnosis and to rule out tumors and infections. Eligible participants receive cyclophosphamide lymphodepletion followed by a single infusion of ICG318 (BCMA‑CD19‑IL‑15/IL‑15 sushi) CAR‑T cells. After infusion they will have close monitoring with frequent blood tests and clinical assessments (including muscle strength testing [MMT‑8], skin assessment [CDASI], disease activity measures, and quality‑of‑life questionnaires) during the first month and at scheduled visits up to 24 months, with safety follow‑up for adverse events up to 2 years. Use of effective contraception during the study period is required.
How the study is structured. Phase 1 interventional study with a single treatment group and no masking. After pre‑treatment (cyclophosphamide) participants receive one infusion of ICG318 CAR‑T. The trial uses sequential dose‑escalation cohorts (starting from 1×10^6/kg) to establish a recommended Phase 2 dose. Participants have regular clinic visits, blood tests, and disease assessments over about 2 years.
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 polymyositis
This trial is also listed on this website under anti-synthetase syndrome and dermatomyositis.
Important dates
- Study start
- Mar 20, 2025
- Primary completion
- Dec 31, 2027 (estimated)
- Study completion
- Dec 31, 2027 (estimated)
- Target enrollment
- 10 participants (estimated)
- Last updated on registry
- Jul 20, 2026
- 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
- iCell Gene Therapeutics
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.
- Qibing Xie 📞 +86-28-85422391 ✉ xieqibing1971@163.com
Participating sites (1)
- West China Hospital, Sichuan UniversityRecruiting
Chengdu, Sichuan, 610041 · China
- Qibing Xie · +86-28-85422391 · xieqibing1971@163.com
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- 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.
- corticosteroid
- 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.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- stable dose
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- 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.
- CT
- An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
- screening period
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- echocardiography
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- 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.
- investigational agent
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
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 website; 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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