YTS109 in Pediatric Relapsed/Refractory Autoimmune Diseases

NCT07439029 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: IIT2025030
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Outcome Measures

Primary Outcomes (7)

• Incidence of Treatment-Emergent Adverse Events

  Safety assessments are conducted using the NCI-CTCAE version 5.0 standards.

  12weeks for safety measurements during the treatment assessment period

• Efficacy outcomes for SLE

  SLE Response index 4(SR-4) response: Min/Max Value: Not specife: a decrease in score indicates improvement: hicher scores indicate worse outcome

  12 weeks for efficacy measurements during the treatment assessment period

• Efficacy outcomes for Systemic Sclerosis

  ACR-CRISS score (CRISS score ≥0.6 improvement, \< 0.6 no improvement) and modified CRISS score (rCRISS score) (percentage of patients with at least 3 of the 5 core items of ACR-CRISS improved by a certain percentage (e.g. 25%, except FVC (5%))

  12 weeks for efficacy measurements during the treatment assessment period

• Efficacy outcomes for Inflammatory Myopathy

  Total Improvement Score (TlS): Min/Max Value: Not specified; an increase in score indicates improvement, higher scores indicate better outcomes.

  12 weeks for efficacy measurements during the treatment assessment period

• Efficacy outcomes for Sjogren's Syndrome

  Sjogren's tool for assessing response (STAR): Min/Max Value: Not specified: a decrease n score indicates improvement: higher scores indicate worse outcome

  12 weeks for efficacy measurements during the treatment assessment period

• Efficacy outcomes for Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

  Pediatric Vasculitis Activity Score Min/Max Value: 0 to 63: an increase in score indicates worsening condition. Higher cores indicate: Worse Outcome

  12 weeks for efficacy measurements during the treatment assessment period

• Efficacy outcomes for antiphospholipid syndrome

  Incidence of newly confirmed thrombotic events. New thrombotic events will be confirmed by objective imaging studies (e.g., Doppler ultrasonography, CT angiography, magnetic resonance imaging, or venography) and adjudicated according to the revised Sydney classification criteria for antiphospholipid syndrome.

  12 weeks for efficacy measurements during the treatment assessment period

Secondary Outcomes (12)

• Peak Plasma Concentration (Cmax) of YTS109

  12 and 24 weeks

• Time to Peak (Tmax) of YTS109

  12 and 24 weeks

• Area under the plasma concentration versus time curve (AUC) of YTS109

  12 and 24 weeks

• Efficacy outcomes for SLE

  24 weeks for efficacy measurements during the treatment assessment period

• Efficacy outcomes for Systemic Sclerosis

  24 weeks for efficacy measurements during the treatment assessment period

Study Arms (1)

YTS109

EXPERIMENTAL

Single intravenous infusion of YTS109 cells; dose escalation per 3+3 design starting at 0.75×10\^6 cells/kg.

Biological: YTS109 cell; Other: YTS109 cell

Interventions

YTS109 cell BIOLOGICAL

Single intravenous infusion of YTS109 cells; dose escalation per 3+3 design starting at 0.75×10\^6 cells/kg.

YTS109

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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:
• ）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).
• ）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).
• ）Coagulation: International normalized ratio (INR) ≤ 1.5 × ULN and prothrombin time (PT) ≤ 1.5 × ULN.
• ）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.
• 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.

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.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead
• China Immunotech (Beijing) Biotechnology Co., Ltd.: collaborator

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, China

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Scleroderma, Diffuse; Myositis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Antiphospholipid Syndrome; Sjogren's Syndrome

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Scleroderma, Systemic; Skin Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Arthritis, Rheumatoid; Arthritis; Joint Diseases; Rheumatic Diseases; Xerostomia; Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Dry Eye Syndromes; Lacrimal Apparatus Diseases; Eye Diseases

Central Study Contacts

Professor Hong Xu

CONTACT

+86 02164932929; hxu@shmu.edu.cn

Study Design

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

Study Record Dates

• First Submitted: February 8, 2026
• First Posted: February 27, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): July 1, 2030
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)