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BCMA-CD19 cCAR-T for the Treatment of Refractory Inflammatory Myopathy

2026년 7월 15일 업데이트: iCell Gene Therapeutics

A Single-arm, Open-label Phase I Clinical Study to Evaluate ICG318 CAR-T in Adults With Refractory Inflammatory Myopathy

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).

연구 개요

상세 설명

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.

연구 유형

중재적

등록 (추정된)

10

단계

  • 1단계

연락처 및 위치

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연구 연락처

연구 장소

    • Sichuan
      • Chengdu, Sichuan, 중국, 610041
        • 모병
        • West China Hospital, Sichuan University
        • 연락하다:

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

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아니

설명

Inclusion Criteria:

  1. Age 18-60 (age ≥18 years at first symptom onset), gender not limited;
  2. 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;

  3. 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;
  4. 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;
  5. Severity of enrollment met the following criteria:

    1. MMT-8 score ≤141 (out of 150);
    2. 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;
    3. At least one muscle enzyme level >1.5 times ULN;
  6. 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:

  1. During the screening period, individuals with active viral, bacterial, or other infections requiring systemic treatment are excluded.
  2. 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;
  3. Patients with severe organ dysfunction:

    1. 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].
    2. 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).
    3. 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.
    4. 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;
  4. Research participants who have a history of or current malignancy;
  5. 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);
  6. Central nervous system diseases, including cerebrovascular diseases, epilepsy, etc.;
  7. Allergies to components of treatment or pre-conditioning.
  8. Those with elective surgery planned during the study period;
  9. Those who do not wish to take necessary contraceptive measures during the research period;
  10. Any other situation that researchers deem unsuitable for participation in the study including poor compliance and being involved in another clinical study.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 순차적 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 단일군 생물학적 제제 주입
Anti-BCMA, Anti-CD19 Compound CAR-T cells

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
ICG318 CAR-T 주입 후 이상 반응(AE) 발생 건수
기간: ICG318 CAR-T 주입 후 0일부터 최대 2년까지.
이상 반응(AE), 중대한 이상 반응(SAE), 치료 관련 이상 반응(TEAE), 특별 관심 이상 반응(AESI) 및 용량 제한 독성(DLT)이 발생한 참가자 수.
ICG318 CAR-T 주입 후 0일부터 최대 2년까지.

2차 결과 측정

결과 측정
측정값 설명
기간
Determine the recommended phase 2 dose (RP2D) regimen.
기간: Starting day 0 and assessed 2 years after ICG318 CAR-T infusion.
The protocol is based on cohort schema with dose escalation from 1×10^6/kg.
Starting day 0 and assessed 2 years after ICG318 CAR-T infusion.
The proportion of subjects who achieved drug-free remission.
기간: At 6 months (M), 12M, 24M after ICG318 CAR-T infusion.
At 6 months (M), 12M, 24M after ICG318 CAR-T infusion.
Basic Metabolic Panel
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Sodium, Chloride, Potassium, Bicarbonate, BUN, Creatinine, and Glucose levels
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Coagulation studies
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
PT/INR and aPTT
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Cytokine testing
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
IL-6 and TNF-alpha
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Manual Muscle Testing 8 (MMT8) score
기간: Days 14, 28; Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Evaluation of muscle strength with MMT8
Days 14, 28; Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Evaluation of skin pathology with CDASI rating
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Myositis Disease Activity Assessment Tool (MDAAT) score
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Extra-muscular disease activity measured with MDAAT
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
36-item short form survey (SF-36) score. 0 (worst health) and 100 (best health)
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Patient reported quality of life using SF-36
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Creatinine kinase levels
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Lactate dehydrogenase levels
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Hydroxybutyrate dehydrogenase levels
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Transaminase levels
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
AST, ALT
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
C-reactive protein
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Procalcitonin levels
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Ferritin levels
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Tmax
기간: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Time to maximum serum concentration of ICG318.
Months 3, 6, 12, 24 after ICG318 CAR-T administration
Cmax
기간: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-28d
기간: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-∞
기간: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
Peripheral blood lymphocyte subsets (T, B, NK, CD4/CD8
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6 post-ICG318 CAR-T
Anti-myositis antibody levels
기간: Days 14, 28 ; Months 2, 3, 6, 9, 12, 18, 24 post-ICG318.
(e.g. anti-Jo-1, anti-nuclear etc.)
Days 14, 28 ; Months 2, 3, 6, 9, 12, 18, 24 post-ICG318.
Immunoglobulins
기간: Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T
Days 1-7, 10, 14, 21, and 28, Months 2, 3, 6, 9, 12, 18, 24 post-ICG318 CAR-T

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연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2025년 3월 20일

기본 완료 (추정된)

2027년 12월 31일

연구 완료 (추정된)

2027년 12월 31일

연구 등록 날짜

최초 제출

2026년 7월 6일

QC 기준을 충족하는 최초 제출

2026년 7월 15일

처음 게시됨 (실제)

2026년 7월 20일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 7월 20일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 7월 15일

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2026년 7월 1일

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염증성 근병증에 대한 임상 시험

ICG318, BCMA-CD19-IL-15/IL-15 sushi Compound CAR-T에 대한 임상 시험

구독하다