Not Yet RecruitingPhase 1

YTS109 in Pediatric Relapsed/Refractory Autoimmune Diseases

China12 participantsStarted 2026-03

Plain-language 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.

Who can participate

Age range5 Years – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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:
✓. 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).
✓. 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).

Exclusion criteria

What they're measuring

Incidence of Treatment-Emergent Adverse Events

Timeframe: 12weeks for safety measurements during the treatment assessment period

Efficacy outcomes for SLE

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Systemic Sclerosis

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Inflammatory Myopathy

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Sjogren's Syndrome

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for antiphospholipid syndrome

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Trial details

NCT IDNCT07439029

SponsorChildren's Hospital of Fudan University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-03

Contact for this trial

Professor Hong Xu

+86 02164932929 hxu@shmu.edu.cn

View on ClinicalTrials.gov More Systemic Lupus Erythematosus (SLE) trials

Plain-language summary

Who can participate

Age range5 Years – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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:
✓. 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).
✓. 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).

Exclusion criteria

What they're measuring

Incidence of Treatment-Emergent Adverse Events

Timeframe: 12weeks for safety measurements during the treatment assessment period

Efficacy outcomes for SLE

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Systemic Sclerosis

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Inflammatory Myopathy

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Sjogren's Syndrome

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period

Efficacy outcomes for antiphospholipid syndrome

Timeframe: 12 weeks for efficacy measurements during the treatment assessment period