HY001N for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy.

NCT07748533 | Recruiting Phase 1 DMC

• 1 other identifier: HY001N101
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autoimmune Hemolytic Anemia (AIHA)

Outcome Measures

Primary Outcomes (2)

• Incidence of Treatment-related Adverse Events

  Therapy-related adverse events (AE), including severe adverse events (SAE) and laboratory outliers with clinical significance, will be recorded and assessed according to the Common Terminology Criteria for Adverse Events (CTCA, Version 6.0)

  Up to 28 days post-infusion

• The safe dosage for a single infusion of HY001N Cell Injection

  The safe dosage for AIHA patients will be evaluated by comprehensively assessing the Overall Response Rate (ORR) and the incidence of adverse events (AEs).

  Up to 28 days post-infusion

Secondary Outcomes (3)

• Overall Remission Rate (ORR)

  Six months post-infusion

• Changes in laboratory test indicators

  Up to 24 months post-infusion

• Drug-free Remission (DFR)

  Up to 24 months post-infusion

Study Arms (1)

Participants who take HY001N cell injection

EXPERIMENTAL

A conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by investigational treatment, HY001N Cell Injection.

Drug: HY001N Cell Injection

Interventions

HY001N Cell Injection DRUG

Drug: HY001N Cell Injection The dose was incremented according to the "3+3" principle, and the three dose levels A, B and C were given in sequence at one time, which were respectively: A-1 dose group: 0.25×10\^5 CAR-T live cells /kg body weight Group A (initial dose) : 0.5×10\^5 CAR-T live cells /kg body weight Group B: 1.0×10\^5 CAR-T live cells /kg body weight Group C: 1.5×10\^5 CAR-T live cells /kg body weight

Participants who take HY001N cell injection

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Juventas Cell Therapy Ltd.: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, China

RECRUITING

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

Xiaofeng PAN

CONTACT

+86 13295841367; panxiaofeng@juventas.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2026
• First Posted: August 5, 2026
• Study Start: July 2, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): October 1, 2028
• Last Updated: August 5, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)