A Study of YTS109 Cell in Subjects With Relapsed/Refractory Autoimmune Hemolytic Anemia

NCT07287930 | Not Yet Recruiting Phase 1

• 1 other identifier: YTS109-011
• Study Type: interventional
• Target: 13
• Locations: 0 countries
• Sites: N/A

Brief Summary

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 1E6 cells/kg or the starting dose may be adjusted based on accumulated data.

Conditions

Autoimmune Hemolytic Anemia

Outcome Measures

Primary Outcomes (2)

• • Dose Limiting Toxicity [Time Frame: Within 28 days after infusion]

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

  Within 28 days

• • The incidence and frequency of treatment-emergent adverse events [Time Frame: Within 6 months after infusion]

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

  Within 6 months

Secondary Outcomes (7)

• • Best overall response rate (BOR) of each dose group [Time Frame: Within 12 weeks after infusion]

  Within 12 weeks

• • Objective response rate (ORR) of each dose group [Time Frame: Within 4 weeks after infusion]

  Within 4 weeks

• • Time to response (TTR) [Time Frame: Within 6 months after infusion]

  Within 6 months

• • 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.

Study Arms (1)

Experimental: YTS109 cell

EXPERIMENTAL

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

Interventions

YTS109 cell DRUG

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.

Experimental: YTS109 cell

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 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:
• 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).
• Renal Function:
• Creatinine clearance (CrCl) ≥ 60 ml/minute (calculated using the Cockcroft-Gault formula).
• Oxygen Saturation (SpO₂): ≥ 92%.
• Bone Marrow Function Requirements:
• Absolute neutrophil count (ANC) ≥ 1.0 × 10⁹/L;
• Platelet count ≥ 30 × 10⁹/L;
• International normalized ratio (INR) ≤ 1.5 × ULN;
• Activated partial thromboplastin time (APTT) ≤ 1.5 × ULN.
• ECOG performance status≤2.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: lead
• China Immunotech (Beijing) Biotechnology Co., Ltd.: collaborator

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Heng Mei Professor Heng Mei

CONTACT

+86 13886180811; hmei@hust.cdu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: December 4, 2025
• First Posted: December 17, 2025
• Study Start: December 25, 2025
• Primary Completion (Estimated): December 25, 2026
• Study Completion (Estimated): December 26, 2027
• Last Updated: December 17, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share