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

2026年7月15日 更新者: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).

調査の概要

詳細な説明

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.

研究の種類

介入

入学 (推定)

10

段階

  • フェーズ 1

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

研究場所

    • Sichuan
      • Chengdu、Sichuan、中国、610041
        • 募集
        • West China Hospital, Sichuan University
        • コンタクト:

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人

健康ボランティアの受け入れ

いいえ

説明

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.

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:処理
  • 割り当て:なし
  • 介入モデル:順次割り当て
  • マスキング:なし(オープンラベル)

武器と介入

参加者グループ / アーム
介入・治療
実験的:シングルアーム生物学的注入
Anti-BCMA, Anti-CD19 Compound CAR-T cells

この研究は何を測定していますか?

主要な結果の測定

結果測定
メジャーの説明
時間枠
ICG318 CAR-T注入後の有害事象(AE)の数。
時間枠:0日目からICG318 CAR-T注入後2年まで。
有害事象、重篤な有害事象(SAE)、治療下で発現した有害事象(TEAE)、特に関心のある有害事象(AESI)、および用量制限毒性(DLT)が認められた参加者の数。
0日目からICG318 CAR-T注入後2年まで。

二次結果の測定

結果測定
メジャーの説明
時間枠
Determine the recommended phase 2 dose (RP2D) regimen.
時間枠: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.
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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)
時間枠: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
時間枠: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)
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠:Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-28d
時間枠:Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-∞
時間枠: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
時間枠: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
時間枠: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
時間枠: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

協力者と研究者

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研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2025年3月20日

一次修了 (推定)

2027年12月31日

研究の完了 (推定)

2027年12月31日

試験登録日

最初に提出

2026年7月6日

QC基準を満たした最初の提出物

2026年7月15日

最初の投稿 (実際)

2026年7月20日

学習記録の更新

投稿された最後の更新 (実際)

2026年7月20日

QC基準を満たした最後の更新が送信されました

2026年7月15日

最終確認日

2026年7月1日

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