This is a Phase I, single-arm, open-label, dose-escalation and dose-expansion study. The primary objective is to evaluate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of YTS109 STAR-T cell therapy in patients with autoimmune hemolytic anemia who have failed ≥3 lines of therapy.
This study set up two dosage groups: 1e6 STAR+T cells/kg and 2e6 STAR+T cells/kg. With the starting dose of 1e6 STAR+T cells/kg, it was conducted according to the traditional 3+3 design rule. In this study, if the safety was good but the efficacy was not satisfactory at the dosage level of 2e6 STAR+T cells/kg, SRC could decide whether to continue increasing the dose to 3e6 STAR+T cells/kg based on the clinical preclinical data, cumulative safety, tolerance, preliminary efficacy, PK and other results.
If the subject shows no response (NR) or experiences recurrence after remission, a second infusion may be administered-provided the patient voluntarily consents and the investigator, after comprehensive assessment, determines that the potential benefits outweigh the risks. The investigator may decide on the timing of re-infusion, the use of lymphodepleting pretreatment, and the dose and number of re-infusions based on prior safety, efficacy, and pharmacokinetic (PK) data.
18 ≤ Age ≤ 60 years, regardless of gender.
A definitive diagnosis of Autoimmune Hemolytic Anemia (AIHA) or Evans Syndrome [including warm antibody-type, mixed warm-cold antibody-type, and cold antibody-type hemolytic anemia (cold agglutinin disease)] has been established, with diagnostic criteria referenced from the Chinese Clinical Practice Guidelines for the Diagnosis and Treatment of Autoimmune Hemolytic Anemia in Adults (2023 Edition).
Patients who have undergone at least three failed treatment attempts, whose anemia symptoms (hemoglobin < 100 g/L) persist despite conventional therapy, and who remain unresponsive or experience recurrence after disease remission. Definition of Conventional Therapy: Treatment with glucocorticoids and/or rituximab, combined with any one or more of the following interventions: splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, bendamustine, fludarabine, bortezomib, or other pharmacological agents, as well as biologic agents including anti-CD38 monoclonal antibodies, BTK inhibitors, Syk inhibitors, complement inhibitors, etc.
Oxygen Saturation (SpO₂): ≥ 92%.
Diagnosis of lymphoproliferative tumor
Other hereditary or acquired hemolytic diseases (Secondary AIHA caused by drugs or infection)
The platelet count in peripheral blood<30×10^9/L
Pregnant or breast-feeding subjects
Single group
YTS109 cell
DrugSubjects will receive YTS109 cell, and dose escalation will commence at 1E6 cells/kg or the starting dose may be adjusted based on accumulated data.
Dose Limiting Toxicity
The incidence and frequency of treatment-emergent adverse events
Safety assessments are conducted using the NCI-CTCAE version 5.0 standards
Best overall response rate (BOR) of each dose group
BOR is determined as the most favorable response observed after cell infusion, until either disease relapse or the completion of a specified observation period
Objective response rate (ORR) of each dose group
Time to response (TTR)
TTR is defined as the duration from cell infusion to the achievement of a hematological response
Peak Plasma Concentration (Cmax) of YTS109
To evaluate the metabolic characteristics of YTS109
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