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

15 de julio de 2026 actualizado por: 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).

Descripción general del estudio

Estado

Reclutamiento

Descripción detallada

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.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

10

Fase

  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Ubicaciones de estudio

    • Sichuan
      • Chengdu, Sichuan, Porcelana, 610041
        • Reclutamiento
        • West China Hospital, Sichuan University
        • Contacto:

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto

Acepta Voluntarios Saludables

No

Descripción

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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: N / A
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Infusión biológica de un solo brazo
Anti-BCMA, Anti-CD19 Compound CAR-T cells

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Número de Eventos Adversos (AEs) después de la infusión de ICG318 CAR-T.
Periodo de tiempo: Starting day 0 and up to 2 years after ICG318 CAR-T infusion.
Número de participantes con eventos adversos (EA), eventos adversos graves (EAG), eventos adversos emergentes del tratamiento (EAET), eventos adversos de especial interés (EAEI) y toxicidades limitantes de dosis (DLT).
Starting day 0 and up to 2 years after ICG318 CAR-T infusion.

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Determine the recommended phase 2 dose (RP2D) regimen.
Periodo de tiempo: 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.
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-28d
Periodo de tiempo: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-∞
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

20 de marzo de 2025

Finalización primaria (Estimado)

31 de diciembre de 2027

Finalización del estudio (Estimado)

31 de diciembre de 2027

Fechas de registro del estudio

Enviado por primera vez

6 de julio de 2026

Primero enviado que cumplió con los criterios de control de calidad

15 de julio de 2026

Publicado por primera vez (Actual)

20 de julio de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

20 de julio de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

15 de julio de 2026

Última verificación

1 de julio de 2026

Más información

Términos relacionados con este estudio

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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