Research on YTS109 Cell in Patients With Recurrent/Refractory Autoimmune Hemolytic Anemia

The Safety and Efficacy of YTS109 Cell for Relapsed/Refractory Autoimmune Hemolytic Anemia Patients After Receiving Three or More Lines of Therapy.

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Autoimmune Hemolytic Anemia
• Intervention / Treatment: Drug: YTS109 cell

• Study Type: Interventional
• Enrollment (Estimated): 13
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Liping Jing; Phone Number: 13820381160; Email: jlp1160@126.com

Study Locations

• China
  • Beijing, China
    • Beijing GoBroad Boren Hospital
    • Contact: Liping Jing; Phone Number: 13820381160; Email: jlp1160@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

• Adequate Organ Function:

  1. Liver Function:

     Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 × upper limit of normal (ULN); Total bilirubin ≤ 2.0 × ULN (excluding Gilbert syndrome, where total bilirubin ≤ 3.0 × ULN).

  2. Renal Function:

     Creatinine clearance (CrCl) ≥ 60 ml/minute (calculated using the Cockcroft-Gault formula).

  3. Oxygen Saturation (SpO₂): ≥ 92%.
• ECOG performance status≤2.
• Subjects of childbearing potential will be required to follow contraception requirements from the time of enrollment until the end of the 12-month safety follow-up period.
• The subjects voluntarily participate in the study, sign the informed consent, demonstrate good compliance, and cooperate with follow-up.

Exclusion Criteria:

• 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
• Receive any of the following treatments within the specified time before cell infusion: a.anti-CD20 monoclonal antibodies <12 weeks, b.sutimlimab or other marketed biologics <5 half-lives,c.plasma exchange <4 weeks, d.post-splenectomy <12 weeks, e. BTK inhibitors, anti-CD38 monoclonal antibody, Syk inhibitors, BAFF inhibitors < 5 half-lives.
• Previously received organ or stem cell transplantation
• History of new thrombosis or organ infarction in the past 6 months
• Diagnosis of the active stage of the connective tissue disease.
• Have active infections, such as sepsis, bacteremia, fungemia, uncontrolled pulmonary infection and active tuberculosis, etc.
• Positive hepatitis B surface antigen (HBsAg) or hepatitis B e antigen (HBeAg); positive hepatitis B e antibody (HBe-Ab) or hepatitis B core antibody (HBc-Ab), and the HBV-DNA copy number is above the lower limit of the measurable capacity; positive hepatitis C (HCV) antibody; positive human immunodeficiency virus (HIV) antibody; positive syphilis test.
• Underwent major surgery within 4 weeks before screening, as determined by the investigator to be unsuitable for enrollment.
• Have malignant tumors within 5 years before enrollment, except tumors with negligible risk of metastasis or death and curable tumors, such as adequately treated cervical carcinoma in situ, cutaneous basal cell carcinoma, etc.
• Have any of the following cardiovascular diseases: a.Left ventricular ejection fraction (LVEF) ≤45%, b. presence of active heart disease or congestive heart failure (New York Heart Association [NYHA] Class III or IV)), c.severe arrhythmias requiring treatment, d.have myocardial infarction, bypass surgery, or stent placement within the 6 months before the study, e.other heart diseases judged by the researcher to be unsuitable for enrollment.
• Have a history of live attenuated vaccines within 6 weeks before enrollment.
• Have a history of epilepsy or other active central nervous system diseases.
• Have an allergy to the ingredients of the medicine used in this study.
• Previously received CAR-T cell therapy.
• Patients considered to be ineligible for the study by the investigator for reasons other than the above.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: YTS109 cell; Subjects will receive YTS109 cell, and dose escalation will commence at 1E6 cells/kg or the starting dose may be adjusted based on accumulated data.	Drug: YTS109 cell; Subjects will receive YTS109 cell, and dose escalation will commence at 1E6 cells/kg or the starting dose may be adjusted based on accumulated data.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose Limiting Toxicity		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	Within 6 months after infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective response rate (ORR) of each dose group		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	Within 6 months after infusion
Peak Plasma Concentration (Cmax) of YTS109	To evaluate the metabolic characteristics of YTS109	Within 12 months after infusion
Time to Peak (Tmax) of YTS109	To evaluate the metabolic characteristics of YTS109	Within 12 months after infusion
Area under the plasma concentration versus time curve (AUC) of YTS109	To evaluate the metabolic characteristics of YTS109	Within 12 months after infusion
The reconstitution of B cell in peripheral blood	Changes in B cells quantification and phenotypic in peripheral blood	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	Within 12 weeks after infusion

Collaborators and Investigators

• Sponsor: China Immunotech (Beijing) Biotechnology Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): March 18, 2026
• Primary Completion (Estimated): March 18, 2027
• Study Completion (Estimated): March 18, 2028

Study Registration Dates

• First Submitted: March 2, 2026
• First Submitted That Met QC Criteria: March 2, 2026
• First Posted (Actual): March 6, 2026

Study Record Updates

• Last Update Posted (Actual): March 6, 2026
• Last Update Submitted That Met QC Criteria: March 2, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Autoimmune Diseases; Immune System Diseases; Hematologic Diseases; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Anemia, Hemolytic, Autoimmune
• Other Study ID Numbers: YTS109-016

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No