ClinicalTrials.gov record NCT07287930
A Study of YTS109 Cell in Subjects With Relapsed/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.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 1, single-arm, open-label study gives escalating doses of YTS109 cell therapy to adults with AIHA whose disease has not responded to three or more prior treatments. It will evaluate safety, early signs of response, how the cells behave in the body, and immune-cell recovery. An exploratory approach will test non-lymphodepleting conditioning and single versus fractionated infusions.
What it is studying. The study is testing YTS109 cell therapy in adults with relapsed or refractory AIHA to measure safety and tolerability, look for preliminary clinical responses, study how the therapy is processed in the body (pharmacokinetics/pharmacodynamics), and observe effects on B cell reconstitution. It also explores infusing cells without full lymphodepletion and whether one infusion versus 1–3 fractionated infusions affects outcomes.
What the researchers hope to learn. To evaluate the safety and tolerability of YTS109 cell therapy; to gather early evidence about clinical responses in heavily pretreated AIHA; to characterize pharmacokinetics and pharmacodynamics of the infused cells; and to study how B cells and immune cells recover after treatment. The study also aims to explore the effects of non-lymphodepleting conditioning and different infusion schedules.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- YTS109 cell
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
- 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 description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
Not provided
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After screening and informed consent, participants will receive YTS109 cell therapy by infusion (dose-escalation starting around 1×10⁶ cells/kg; the starting dose may be adjusted). An exploratory non-lymphodepleting conditioning may be given before infusion, and the cell dose may be given as a single infusion or divided into 1–3 infusions completed within 7 days (no later than 15 days). Expect regular clinic visits with blood tests and safety monitoring (including close observation for early toxicities in the first 28 days) and follow-up visits with blood sampling to assess response, drug levels, and immune cell recovery for up to at least 12 months. Participants must follow contraception requirements if applicable and comply with study procedures and follow-up.
How the study is structured. Phase 1, interventional treatment study that is open-label and non-randomized. All participants receive YTS109 cell infusion in a single-group, dose-escalation followed by dose-expansion format; dose escalation starts at 1×10^6 cells/kg (adjustable based on accumulated data). The study records dose-limiting toxicities within 28 days and treatment-emergent adverse events up to 6 months; secondary measures include response rates, time to response, PK measures (Cmax, Tmax, AUC), and B cell reconstitution up to 12 months. An exploratory non-lymphodepleting infusion can be given as one infusion or divided into 1–3 infusions completed within 7 days (no later than 15 days).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
Important dates
- Study start
- Dec 25, 2025 (estimated)
- Primary completion
- Dec 25, 2026 (estimated)
- Study completion
- Dec 26, 2027 (estimated)
- Target enrollment
- 13 participants (estimated)
- Last updated on registry
- Dec 17, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
- Principal investigator
- MEI HENG, Professor
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology - Collaborators
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Heng Mei Professor Heng Mei 📞 +86 13886180811 ✉ hmei@hust.cdu.cn
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- dose-escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- pharmacokinetics
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- pharmacodynamics
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- lines of therapy
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- Creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- ECOG performance status
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- safety follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- CAR-T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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