Study Investigating the Safety of Anti-CD19 CAR-T Cells Therapy Produced at Gustave Roussy for Adults With Severe and Refractory Systemic Autoimmune Rheumatic Diseases

NCT07544225 | Not Yet Recruiting Phase 1 DMC

• 2 other identifiers: 2026-526552-32-002026/4318
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open-label, single-dose, prospective, monocenter, Phase I interventional study (not first-in-human) to assess the safety, tolerability, and preliminary efficacy of autologous anti-CD19 CAR-T cells as an advanced therapy medicinal product in adult patients; with severe and refractory systemic autoimmune rheumatic diseases, including rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), Sjogren's disease (SjD), systemic sclerosis (SSc) and idiopathic inflammatory myositis (IIM).

Conditions

Rheumatoid Arthritis (RA); Systemic Lupus Erythematosus (SLE); Sjogren's Disease With Dry Eyes; Systemic Sclerosis (SSc); Idiopathic Inflammatory Myositis (IIM)

Outcome Measures

Primary Outcomes (1)

• Unacceptable toxicities over a period of 4 weeks after the single CAR-T cell infusion

  Unacceptable toxicities are defined over a period of 4 weeks after the single CAR-T cell infusion as either: * Cytokine Release Syndrome (CRS) ≥ grade 3 or, * Immune-effector Cell Associated Neurotoxicity Syndrome (ICANS) ≥ grade 3 or, * Organ toxicity (cardiac, dermatologic, gastrointestinal, hepatic, pulmonary, renal/genitourinary, or neurologic) ≥ grade 3

  from CAR-T cells infusion to 30 days post-treatment

Study Arms (1)

Treatment

EXPERIMENTAL

The anti-CD19 CAR-T cells will be administered as a single infusion at the dose of 1x106 cells/kg body weight

Biological: anti-CD19 CAR-T cells

Interventions

anti-CD19 CAR-T cells BIOLOGICAL

The anti-CD19 CAR-T cells will be administered as a single infusion at the dose of 1x106 cells/kg body weight

Treatment

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ECOG Performance status 0 to 2.
• Signed informed consent (or legal representative) must be obtained from the patient prior to participation in the study.
• Patients must have valid health insurance coverage in the country where the study is conducted.
• A diagnosis of treatment-refractory systemic autoimmune rheumatic disease (rheumatoid arthritis (RA), severe lupus erythematosus (SLE), Sjogren's disease (SjD), Systemic sclerosis (SSc) or idiopathic inflammatory myositis (IIM)) with a severe and progressive disease according to respective criteria below.
• Eligible patients must have failed at least two lines of conventional or biologic immunosuppressive therapies
• Calculated creatinine clearance (calculated using CKD-EPI 2021 Formula) \> 30 mL/min
• Women of childbearing potential must agree to use highly effective contraception or practice true sexual abstinence for at least 1 month before study start and until 12 months after CAR-T cells infusion. (see section 7.7 " Pregnancy and contraception ").
• Male patients must agree to practice true sexual abstinence or use condoms from 1 month before study start until 12 months after CAR-T cells infusion. Also, it is recommended the childbearing potential female partner uses a highly effective method of contraception for the same duration (cf. section 7.7 " Pregnancy and contraception ").
• Patients shall be eligible to undergo CAR-T cells infusion
• Patients consents to give blood samples.
• Patients Patients must be located within one hour's driving distance from Gustave Roussy during the first 30 days after the injection and must be accompanied, as they should not drive during these initial 30 days
• Specific criteria for SLE:
• Diagnosis of SLE according to the 2019 ACR/EULAR classification criteria.
• Patient must be positive for at least one of the following autoantibodies at screening:
• anti-dsDNA (above the upper limit of normal, ULN) or

You may not qualify if:

• Any significant active infection, including:
• Active hepatitis B (defined by detectable HBV DNA) or C infection (defined by detectable HCV RNA),
• Known HIV infection,
• Previous treatment with any gene therapy product or CAR-T cells therapy.
• Inadequate organ function during screening
• Inadequate renal function is defined as eGFR ≤30mL/min/1.73m2 using the the Cockcroft-Gault Formula
• Inadequate hepatic function is defined as any of the following:
• defined by Child-Pugh class B or C
• ALT and AST above 2 × ULN
• TBL \> 1.5 × ULN with the exception of participants with Gilbert's syndrome or patients with a positive Coombs test and evidence of hemolysis, who may be included if their TBL is ≤ 3.0 × ULN and direct bilirubin ≤ 1.5 × ULN
• International normalized ratio (INR) \> 1.5
• Inadequate cardiac function is defined as LVEF \< 50% as determined by ultra-sonography performed at screening
• Inadequate lung function requiring continuous oxygenotherapy
• Active malignancy or history of cancer within the past 2 years (except successfully treated localized skin basal or squamous cell carcinoma, or cervical carcinoma in situ).
• History of allogeneic hematopoietic tem cell transplantation or solid organ transplantation.

Sponsors & Collaborators

• Gustave Roussy, Cancer Campus, Grand Paris: lead

Study Sites (1)

Gustave Roussy

Villejuif, Val de Marne, 94805, France

Location

MeSH Terms

Conditions

Arthritis, Rheumatoid; Lupus Erythematosus, Systemic; Scleroderma, Systemic; Myositis

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases

Central Study Contacts

Cristina CASTILLA-LLORENTE

CONTACT

+33(0)142114355; Cristina.castilla-llorente@gustaveroussy.fr

Timothé DENAËS

CONTACT

+33(0)142115554; timothe.denaes@gustaveroussy.fr

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2026
• First Posted: April 22, 2026
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): February 1, 2029
• Study Completion (Estimated): February 1, 2043
• Last Updated: September 2, 2026

Record last verified: 2026-08

Locations

FR (1)