YTS109 in Pediatric Relapsed/Refractory Autoimmune Diseases

Starting soon · Phase 1

Conditions studied: Systemic Lupus Erythematosus (SLE), Diffuse Systemic Sclerosis, Idiopathic Inflammatory Myopathies (IIMs), ANCA Associated Vasculitis (AAV), Antiphospholipid Syndrome (APS), Sjogren's Syndrome (SS)

In brief

This exploratory, single-arm, open-label study will evaluate the safety and preliminary efficacy of YTS109 cell therapy in pediatric patients with relapsed/refractory autoimmune diseases, including systemic lupus erythematosus, diffuse systemic sclerosis, idiopathic inflammatory myopathies, and Sjögren's syndrome, as well as other eligible autoimmune diseases defined by the protocol eligibility criteria. Approximately 12 patients aged 5 to \<18 years will be enrolled at Children's Hospital of Fudan University and will receive a single intravenous infusion of YTS109 cells. Dose escalation will follow a standard 3+3 design starting at 1.5 × 10\^6 cells/kg. The primary objective is to assess the safety and preliminary efficacy of YTS109 cell therapy in this population. Secondary objectives include characterizing the pharmacokinetic and pharmacodynamic profiles of YTS109 cells. Primary endpoints include the type, severity, and frequency of adverse events, along with efficacy assessments.

Key facts

Study ID

NCT07439029

Run by

Children's Hospital of Fudan University

People needed

12

Starts

2026-03-01

Expected to finish

2030-07-01

Last updated by the study team

2026-02-27

Who can join

Age: 5 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 5 to <18 years at screening; sex not restricted.
• CD19 positivity: Presence of CD19-positive B cells in peripheral blood, confirmed by flow cytometry.
• Adequate major organ function, meeting all of the following criteria:
• 1）Bone marrow function:
• Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L (no colony-stimulating factor use within 2 weeks prior to testing; neutropenia attributable to the underlying disease may be allowed);
• Hemoglobin ≥ 60 g/L. 2）Hepatic function: ALT ≤ 3 × ULN (exceptions allowed for elevations attributable to the underlying disease); AST ≤ 3 × ULN (exceptions allowed for elevations attributable to the underlying disease); Total bilirubin (TBIL) ≤ 1.5 × ULN (exceptions allowed for elevations attributable to the underlying disease).
• 3）Renal function: Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m², calculated using the Schwartz formula (exceptions allowed for reduced renal function attributable to the underlying disease).
• 4）Coagulation: International normalized ratio (INR) ≤ 1.5 × ULN and prothrombin time (PT) ≤ 1.5 × ULN.
• 5）Cardiac status: Hemodynamically stable. 4. Females of childbearing potential must be not pregnant and not breastfeeding during screening and throughout the study period.
• The participant and the legal guardian are willing to participate, provide written informed consent, and can comply with study procedures and follow-up.
• Specific inclusion criteria:
• Recurrent refractory systemic lupus erythematosus
• Meets the 2019 EULAR/ACR classification criteria for systemic lupus erythematosus (SLE).
• Active disease, defined as either: SELENA-SLEDAI ≥ 6 and at least one BILAG-2004 organ domain score of A (severe activity) or two domains scored B (moderate activity), or a combination thereof; or SELENA-SLEDAI ≥ 8.
• Relapsed/refractory or intolerant to conventional therapy, defined as one of the following: Inadequate response after >3 months of conventional therapy; or intolerance to treatment-related adverse effects; or Disease flare/recurrence after achieving remission based on the LLDAS criteria. Conventional therapy is defined as treatment with glucocorticoids and cyclophosphamide, and one or more of the following immunomodulatory agents: antimalarials, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, or any biologic agent, including rituximab, belimumab, and telitacicept.
• If renal involvement is present, a kidney biopsy must have been performed within 12 months prior to treatment, demonstrating active lesions or predominantly active lesions on pathology.
• Relapsing refractory/progressive diffuse systemic sclerosis
• Meets the 2013 ACR classification criteria for systemic sclerosis and is consistent with the diffuse cutaneous subtype (dcSSc).
• Positive for any antinuclear antibody (ANA) or systemic sclerosis-associated autoantibody.
• Evidence of diffuse cutaneous skin sclerosis and/or active interstitial lung disease (ILD), defined as ground-glass opacities on high-resolution computed tomography (HRCT).
• Inadequate response to conventional therapy for >3 months or disease relapse/recurrence after achieving remission. Conventional therapy is defined as treatment with glucocorticoids and cyclophosphamide, and one or more of the following immunomodulatory agents: antimalarials, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, JAK inhibitors, or any biologic agent, including rituximab, tocilizumab, TNF-α inhibitors (etanercept, adalimumab, infliximab), and telitacicept.
• Progressive disease, defined as either:
• 1） Rapid skin progression: mRSS increase >25%; or 2） Progressive lung disease: FVC decline ≥10%, or FVC decline ≥5% accompanied by DLCO decline ≥15%.
• Note: Criterion 4 or 5 must be met (either one is sufficient).
• Recurrent refractory/progressive inflammatory myopathy:

You may not qualify if…

• History of severe drug allergy or a known allergic predisposition.
• Presence of, or suspected uncontrolled infection requiring treatment, including fungal, bacterial, viral, or other infections.
• Central nervous system (CNS) disorders, except for prior seizures, psychosis, organic brain syndrome, cerebrovascular accident, encephalitis, or CNS vasculitis attributable to the underlying disease, as determined by the investigator.
• Cardiac dysfunction deemed unable to tolerate study treatment (i.e., inadequate cardiac function at the investigator's discretion).
• Known congenital immunoglobulin deficiency.
• Presence of severe congenital structural malformations or syndromic birth defects (e.g., severe cardiovascular malformations, severe CNS malformations), or a confirmed diagnosis of a severe inherited metabolic disorder that, in the investigator's judgment, may significantly increase trial-related risk or interfere with compliance and interpretation of results.
• History of malignancy within the past 5 years.
• End-stage renal disease.
• Evidence of certain chronic/active infections, including: HBsAg-positive, or HBcAb-positive with peripheral blood HBV DNA above the upper limit of detection; Anti-HCV antibody positive with detectable HCV RNA; HIV antibody positive; Positive syphilis test.
• Psychiatric illness or severe cognitive impairment.
• Participation in another clinical trial within 3 months prior to enrollment.
• Use of immunosuppressive agents with therapeutic effects on the underlying disease within five half-lives prior to enrollment, or use of biologic agents within 4 weeks prior to enrollment.
• Pregnant or planning pregnancy (females).
• Any other condition that, in the investigator's opinion, makes the participant unsuitable for enrollment in this study.

Where it is running

• Children's Hospital of Fudan University — Shanghai, China

Full record on ClinicalTrials.gov

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