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. The objective is to evaluate the safety, preliminary efficacy, pharmacokinetics/pharmacodynamics (PK/PD), and immune cell reconstitution characteristics of YTS109 cell therapy in Multi-rAIHA subjects who have failed third-line or higher-line treatments. This study will also conduct an exploratory investigation into the impact of non-lymphodepleting conditioning prior to the infusion of STAR-T cells. For the non-lymphodepleting exploratory cell infusion, it can be administered as a single infusion or divided into 1 to 3 infusions (with the fractionated infusions to be completed within 7 days (and in any case no later than 15 days)). Dose escalation will commence at 5E6 cells/kg or the starting dose may be adjusted based on accumulated data.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
7
Subjects will receive YTS109 cell, and dose escalation will commence at 5E6 cells/kg or the starting dose may be adjusted based on accumulated data.
Institute of Hematology & Blood Diseases Hospital, China
Tianjin, Tianjin Municipality, China
RECRUITINGDose Limiting Toxicity
Time frame: Within 28 days after infusion
The incidence and frequency of treatment-emergent adverse events
Safety assessments are conducted using the NCI-CTCAE version 5.0 standards.
Time frame: Within 12 months after infusion
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.
Time frame: Within 12 weeks after infusion
Objective response rate (ORR) of each dose group
Time frame: Within 4 weeks after infusion
Time to response (TTR)
TTR is defined as the duration from cell infusion to the achievement of a hematological response.
Time frame: Within 6 months after infusion
Peak Plasma Concentration (Cmax) of YTS109
To evaluate the metabolic characteristics of YTS109
Time frame: Within 12 months after infusion
Time to Peak (Tmax) of YTS109
To evaluate the metabolic characteristics of YTS109
Time frame: Within 12 months after infusion
Area under the plasma concentration versus time curve (AUC) of YTS109
To evaluate the metabolic characteristics of YTS109
Time frame: Within 12 months after infusion
The reconstitution of B cell in peripheral blood
Changes in B cells quantification and phenotypic in peripheral blood
Time frame: Within 12 months after infusion
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