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

15 juli 2026 uppdaterad av: 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).

Studieöversikt

Status

Rekrytering

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

10

Fas

  • Fas 1

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

    • Sichuan
      • Chengdu, Sichuan, Kina, 610041
        • Rekrytering
        • West China Hospital, Sichuan University
        • Kontakt:

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen

Tar emot friska volontärer

Nej

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Sekventiell tilldelning
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Enarmad biologisk infusion
Anti-BCMA, Anti-CD19 Compound CAR-T cells

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Antal biverkningar (AEs) efter ICG318 CAR-T-infusion.
Tidsram: Från dag 0 och upp till 2 år efter ICG318 CAR-T-infusion.
Antal deltagare med biverkningar (AEs), allvarliga biverkningar (SAEs), behandlingsutlösta biverkningar (TEAEs), biverkningar av särskilt intresse (AESI) och dosbegränsande toxiciteter (DLTs).
Från dag 0 och upp till 2 år efter ICG318 CAR-T-infusion.

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Determine the recommended phase 2 dose (RP2D) regimen.
Tidsram: 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.
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-28d
Tidsram: Months 3, 6, 12, 24 after ICG318 CAR-T administration
Months 3, 6, 12, 24 after ICG318 CAR-T administration
AUC 0-∞
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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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Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

20 mars 2025

Primärt slutförande (Beräknad)

31 december 2027

Avslutad studie (Beräknad)

31 december 2027

Studieregistreringsdatum

Först inskickad

6 juli 2026

Först inskickad som uppfyllde QC-kriterierna

15 juli 2026

Första postat (Faktisk)

20 juli 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

20 juli 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

15 juli 2026

Senast verifierad

1 juli 2026

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