ClinicalTrials.gov record NCT07748533
HY001N for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy.
A Multi-center, Open-label, Single-arm Phase I Study to Assess the Safety, and Tolerability of HY001N Cell Injection in Patients With Autoimmune Hemolytic Anemia After Failure of 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 I study tests HY001N cell injection in adults with autoimmune hemolytic anemia who have failed three or more prior lines of therapy. Participants undergo apheresis, a lymphodepletion regimen of fludarabine and cyclophosphamide, then a single infusion of HY001N at an assigned dose level. The study primarily looks at safety and tolerability and also collects early measures of blood response and lab changes.
What it is studying. The study is testing whether HY001N cell injection can be given safely and tolerated in adults with AIHA after at least three prior treatment failures. It also collects early evidence on remission and response rates (complete, complete with incomplete hemolysis, or partial), tracks changes in hemoglobin and hemolysis markers, and measures pharmacokinetic, pharmacodynamic, and immunogenicity signals.
What the researchers hope to learn. Scientific goals: evaluate safety and tolerability of HY001N; determine a recommended phase II dose; measure overall remission and response rates (CR, CRi, PR); characterize pharmacokinetics, pharmacodynamics, and immunogenicity; and track duration of drug-free remission and changes in hemolysis-related laboratory tests.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The goal of this clinical trial is to learn if HY001N cell injection safety and tolerability in adult patients with autoimmune hemolytic anemia after failure of 3 or more lines of therapy. It will also learn about the efficacy of HY001N cell injection to treat adult patients with autoimmune hemolytic anemia. The main questions it aims to answer are: Proportion of participants attaining a CR (defined as normalization of hemoglobin not attributed to transfusion effect and the normalization of hemolytic markers) or CRi (defined as normalization of hemoglobin not attributed to transfusion effect without normalization of hemolytic markers) after HY001N infusion? Proportion of participants attaining a PR (defined as hemoglobin ≥ 100 g/L or at least ≥ 20 g/L increase from baseline not attributed to transfusion effect) after HY001N infusion.
- What medical problems do participants have when taking HY001N cell injection?
Researchers will see if HY001N cell injection works to treat autoimmune hemolytic anemia. Participants will:
- Take apheresis, lymphodepletion regimen and HY001N cell injection.
- Visit the clinic on schedule.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- HY001N Cell Injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Participant and/or participant's legal representative fully understand and voluntarily sign informed consent forms;
- Aged 18 to 75 years, regardless of gender;
- Participants with autoimmune hemolytic anemia or Evans syndrome after Failure ≥3 lines of therapy. The Failure of ≥3 lines of therapy meet all the following conditions: Hemoglobin less than 10g/dL and symptoms of anemia;
- For participants diagnosed warm AIHA, or mixed AIHA, or Evans syndrome:
- Failure of first-line corticosteroid therapy; Failure of second-line rituximab therapy; Failure of any one or more of the third-line treatments (splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, fludarabine, bortezomib, etc.)
- For participants diagnosed cold AIHA (cold agglutinin disease):
- Failure of first-line rituximab therapy; Failure of second-line rituximab ± Bendamostine/fludalabine; Failure of third-line therapy (bortezomib, Sutimlimab, Pegcetacoplan, Eculizumab, etc.)
- Women of childbearing age must have a negative blood pregnancy test within 7 days prior to the initiation of conditioning. Any male or female subject of reproductive potential must agree to use effective contraception throughout the study period and for at least 2 year following the infusion of CAR T-cells. In the judgment of the investigator, a subject of reproductive potential refers to having the biological capacity to conceive or father a living child and being sexually active. Women who considered infertile (i.e., meeting at least one of the following criteria): Has undergone a hysterectomy or bilateral oophorectomy, or Medically confirmed ovarian failure, or is medically confirmed as postmenopausal (defined as at least 12 consecutive months of amenorrhea without pathological or physiological causes).
- Laboratory tests of adequate organ function: Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3×ULN; Coagulation profile: INR ≤ 1.5×ULN and aPTT ≤ 1.5×ULN; Serum creatinine ≤1.5×ULN or CCr ≥ 60mL/min; and have a minimum level of pulmonary reserve defined as the blood oxygen saturation in a non-oxygenated state is > 91%.
- Eastern Cooperative Oncology Group (ECOG) performance status 0~2.
Exclusion criteria
- History of lymphoproliferative neoplasms
- Secondary AIHA caused by drugs or infection
- Pregnancy or lactation
- Previously received organ or hematopoietic stem cell transplantation
- History of new thrombotic event or organ infarction in the past 6 months
- Diagnosis of the active stage of connective tissue disease
- Had other inherited or acquired hemolytic diseases
- Have active infections, such as sepsis, bacteremia, fungemia, uncontrolled pulmonary infection and active tuberculosis, etc.
- Positive hepatitis B surface antigen (HBs-Ag) or hepatitis B e antigen (HBe-Ag); 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; EBV-DNA or CMV-DNA copy number is above the lower limit of the measurable capacity;
- Received major surgery within 4 weeks before screening that was assessed by the researcher as 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 (except atrial fibrillation, paroxysmal supraventricular tachycardia), d. QTcB interval
- ≥450ms for male and ≥470ms for female, e. have myocardial infarction, bypass surgery, or stent placement within the 6 months before the trial, f. other heart diseases judged by the researcher to be unsuitable for enrollment
- Have a history of live attenuated vaccines within 6 weeks before enrollment
- Participate in other experimental studies within 30 days prior to apheresis or within 5 half-lives of the trial drug, whichever is longer. (Note: Parallel enrollment in studies is allowed)
- Have a history of epilepsy or other active central nervous system diseases
- Active bleeding
- Have an allergy to the ingredients of the medicine used in this trial
- Previously received CAR T-cell therapy
- Participants 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
This study will be conducted in Chinese hospitals and to include 9 to 12 subjects with refractory/relapsed autoimmune hemolytic anemia. The main purpose of the study is to evaluate the safety and tolerability of HY001N Cell Injection in the treatment of autoimmune hemolytic anemia that has failed at least three lines of treatment, and determine the recommended phase II dose (RP2D) of HY001N Cell Injection in subjects with autoimmune hemolytic anemia who have failed at least three lines of treatment. The Secondary purpose of the study is to evaluate the efficacy of HY001N Cell Injection in the treatment of autoimmune hemolytic anemia that has failed at least three lines of treatment and to evaluate the pharmacokinetic (PK) characteristics, pharmacodynamic (PD) characteristics and immunogenicity in the treated patients.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
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.
Participants undergo apheresis to collect cells, receive a short lymphodepletion (conditioning) chemotherapy regimen with fludarabine and cyclophosphamide, and then receive a single infusion of HY001N cell injection. Dosing will follow an escalating “3+3” design across predefined dose levels. You will have scheduled clinic visits for safety checks and blood tests; treatment-related adverse events are recorded up to 28 days after infusion, and some laboratory and response outcomes are assessed up to 24 months after infusion. Researchers will collect blood samples to measure drug levels (pharmacokinetics), immune responses and immunogenicity, and to track hemoglobin and other blood test changes.
How the study is structured. Open-label, single-group Phase I dose-escalation study using a "3+3" design. All participants receive apheresis, a conditioning (lymphodepletion) regimen of fludarabine plus cyclophosphamide, then a single infusion of HY001N at one of several escalating dose levels. Safety and treatment-related adverse events are monitored (primary safety window up to 28 days); response rates and laboratory changes are followed further (secondary outcomes up to 24 months). There is no masking and no comparison (control) group.
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 listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- 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
- Jul 2, 2026
- Primary completion
- Sep 1, 2028 (estimated)
- Study completion
- Oct 1, 2028 (estimated)
- Target enrollment
- 9 participants (estimated)
- Last updated on registry
- Aug 5, 2026
- 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
- Juventas Cell Therapy 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.
- Xiaofeng PAN 📞 +86 13295841367 ✉ panxiaofeng@juventas.cn
Participating sites (1)
- Institute of Hematology & Blood Diseases HospitalRecruiting
Tianjin · China
- Jun Shi, Doctor
Terms used on this page · explanations by MedicalUpdates.ai
- 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.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- legal representative
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- 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.
- corticosteroid
- 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.
- 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.
- ULN
- 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.
- ECOG
- 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.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- 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.
- 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.
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- relapsed
- A condition that improved with treatment and has since returned or worsened.
- phase II
- A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
- RP2D
- The dose chosen at the end of a phase 1 study for testing in later studies, based on safety and other measurements.
- pharmacokinetic
- 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.
- pharmacodynamic
- 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.
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 website; 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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