IDECABTAGENE VICLEUCEL for Systemic lupus erythematosus | Idiopathic inflammatory myopathy | Systemic sclerosis | Sjögren's syndrome

Inclusion criteria

• {"criterion_text":"- Patient must understand and voluntarily sign an informed consent form including written consent for data protection"}
• {"criterion_text":"- Adults aged ≥ 18 years at time of consent"}
• {"criterion_text":"- Adequate renal (GFR > 30 ml/min/1.73m2), liver (AST/ALT ≤ 1.5 x ULN, unless deemed to be due to myositis CK-elevation or SLE related hepatitis; no Child Pough C), heart (no NYHA IV, EF ≥ 45 %) and pulmonary (FV and DLCO ≥ 35 %) function"}
• {"criterion_text":"- Disease specific inclusion criteria for SLE: 1. Diagnosis of SLE defined as follows: a. Fulfilling the 2019 ACR/EURLAR classification criteria of SLE b. Presence of anti-dsDNA, anti-histone, anti-nucleosome, or anti-Sm antibodies at screening or medical history 2. SLE disease activity a. Active disease at screening, defined as ≥ 1 major organ system with a BILAG A score (severe disease activity) or ≥ 2 organ systems with a BILAG B score (moderate activity) b. Insufficient response to, or recurrence of disease, after at least two of the following treatments: cyclophosphamide, mycophenolate mofetil or its derivatives, belimumab, azathioprine, anifrolumab, methotrexate, rituximab, obinutuzumab, hydroxychloroquine, cyclosporin, tracrolimus, voclosporin, IVIG or JAK inhibitors"}
• {"criterion_text":"- Disease specific inclusion criteria for IIM: 1. Diagnosis of IIM defined as follows: a. Fulfilling the 2017 ACR/EULAR classification criteria for probable or definite DM or PM b. Presence of one or more myositis-specific antibody (aminoacyl tRNA synthetases, Mi2, MDA5, SAE, SRP, ARS, HMGCR, MJ, TIF1gamma) at screening or medical history. Patients must have undergone a sufficient cancer screening prior screening. 2. IIM disease activity a. Presence or signs of active myositis in muscle biopsy or imaging (MRI or PET) and/or signs of interstitial lung disease related to IIM b. In patients with active myositis and muscle weakness as defined by MMT < 142 and 2 of the following criteria: i. VAS patients global ≥ 2 cm ii. VAS physician Global ≥ 2 cm iii. HAQ > 0.25 iv. minimum of one muscle enzyme > 1.3 x ULN v. VAS global extra muscular activity ≥ 2 cm c. Insufficient response to, or recurrence of disease, after at least two of the following treatments: IVIG, methotrexate, azathioprine, cyclophosphamide, tacrolimus, JAK inhibitors, rituximab, mycophenolate mofetil or its derivatives or cyclosporin"}
• {"criterion_text":"- Disease specific inclusion criteria for SSc: 1.\tDiagnosis of SSc defined as follows a. Fulfilling the 2013 ACR/EULAR classification criteria of SSc with diffuse skin involvement b. Presence of one or more of the following autoantibodies: Scl-70, anti-RNA polymerase, anti-Th/To, anti-RP11/12 or anti-U3RNP or a tissue biopsy confirming SSc at screening or medical history 2. SSc disease activity a. Signs of fast disease progression including i. disease duration of ≤ 5 years (from onset of first non-Raynaud manifestation) ii. mRSS score ≥ 15 at screening iii. worsening of disease within 6 months defined as mRSS increase ≥ 3 units or involvement of 1 or more new body area(s) or mRSS increase ≥ 2 units in one body area or ≥ 1 tendon friction rub iv. elevated acute phase reactant levels (CRP ≥ 6 mg/L, ESR ≥ 28 mm/h, or platelet count ≥ 330 G/L) b. b.\tInsufficient response to, or recurrence of disease, after at least two of the following treatments: mycophenolate mofetil or its derivatives, methotrexate, tocilizumab, rituximab, nintedanib, cyclophosphamide or azathioprine and not eligible or denying AHCT"}
• {"criterion_text":"- Disease specific inclusion criteria for PSS: PSS patients 1. Diagnosis of PSS defined as follows a. Fulfilling the 2016 ACR/EULAR classification criteria of PSS b. Presence of anti-SS-A/Ro and anti-SS-B/La antibodies or a tissue biopsy confirming PSS at screening or medical history 2. PSS disease activity a. Active disease with EULAR Sjögren’s Syndrome Disease Activity Index [ESSDAI] score > 6 b. Serological activity defined as hypocomplementemia or one of the follows: elevated CRP, ESR, IgG or RF c. c.\tInsufficient response to, or recurrence of disease, after at least two of the following treatments: cyclophosphamide, azathioprine, mycophenolate mofetil or its derivatives, methotrexate, belimumab, rituximab or hydroxychloroquine"}
• {"criterion_text":"- Patient with insufficient response to, or recurrence of disease, after at least one anti-CD19 / anti-CD20 targeted B cell treatment: rituximab, ocrelizumab, obinutuzumab, CD19 / CD20 T-cell engager or CD19 CAR T-cell therapy"}

Exclusion criteria

• {"criterion_text":"- Clinically suitability for a less burdensome and/or approved therapeutic approach, as judged by the investigator"}
• {"criterion_text":"- Current therapy with cytotoxic drugs"}
• {"criterion_text":"- Diagnosis of severe neuropsychiatric SLE, inclusion body myositis or limited SSc"}
• {"criterion_text":"- Requirement for immunization with live vaccine during the study period or within 14 days preceding leukapheresis"}
• {"criterion_text":"- Participants who are younger than 18 years or participants who are incapable to understand the aim, importance and consequences of the study and to give legal informed consent"}
• {"criterion_text":"- Have a history of alcohol or substance abuse within the preceding 6 months that, in the opinion of the investigator, may increase the risks associated with study participation or study agent administration, or may interfere with interpretation of results"}
• {"criterion_text":"- Patients who possibly are dependent on the sponsor, the principal investigator or investigator (e.g., family members)"}
• {"criterion_text":"- ANC < 1000/mm3, ALC < 200/mm3 or hemoglobin < 8 g/dl, absolute CD3+ T cell count ≥ 100/μl"}
• {"criterion_text":"- Uncontrolled severe concomitant disease, such as cancer (except basal or squamous cell skin cancer) or severe hepatic insufficiency defined as Child-Pugh score ≥ 10 (C) or unstable coronary artery disease"}
• {"criterion_text":"- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study"}
• {"criterion_text":"- Severely Impaired organ function, renal (eGFR < 30 ml/min/m2), liver (AST/ALT > 3 x ULN, Child Pough C), heart (NYHA IV, EF < 40 %) and lungs (FV and DLCO < 30 %)"}
• {"criterion_text":"- Any concomitant severe active infection (e.g., HIV, hepatitis B or C or active tuberculosis as defined by a positive QuantiFERON TB-test; if presence of latent tuberculosis is established then treatment according to local guidelines must have been initiated prior to enrollment)"}
• {"criterion_text":"- Pregnant or lactating females"}
• {"criterion_text":"- Known hypersensitivity to any drug components"}
• {"criterion_text":"- Malignancy in the last 5 years before screening, except stage 1 prostate cancer, DCIS, skin SCC and BCC"}

Primary endpoints

• {"endpoint_text":"- Incidence of any grade ≥ 3 CRS or grade ≥ 3 ICANS, any grade ≥ 3 organ toxicity that does not resolve to grade ≤ 2 within 72 hours, any grade ≥ 3 immune effector cell-associated hemophagocytosis, grade ≥ 2 ICANS and CRS that does not resolve to grade 1 within 7 days following adequate therapy","definition_or_measurement_approach":""}

Germany

Earliest CTIS Part Ii Submission Date

08-10-2025

Latest Decision Or Authorization Date

17-11-2025

Processing Time Days

40

Number Of Sites

1

Number Of Participants

15

Primary sponsor

Full Name

Universitaetsklinikum Erlangen AöR

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Germany