Autoimmune Hemolytic Anemia (AIHA)Phase 1NCT07748533
HY001N for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy.
A Phase 1 study testing HY001N Cell Injection in people with Autoimmune Hemolytic Anemia (AIHA), run by Juventas Cell Therapy Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Autoimmune Hemolytic Anemia (AIHA)
- What is being tested?
- HY001N Cell Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 2 Jul 2026to 1 Oct 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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):
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- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin
Jun Shi, Doctor
About this study
Official title: 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
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.
Read more
- 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.
Read the detailed description
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.
- Conditions
- Autoimmune Hemolytic Anemia (AIHA)
- Being tested
- HY001N Cell Injection
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HY001N Cell InjectionDrug
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
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: HY001N Cell Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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).
Measured: Up to 28 days post-infusion
Secondary outcomes (3)
Overall Remission Rate (ORR)
The proportion of subjects achieving CR, CRi or PR after treatment by HY001N Cell Injection. CR is defined as clinical symptoms disappear, Hb≥110g/L (female) or Hb≥120g/L (male); the levels of hemolysis-related laboratory indicators (serum bilirubin and lactate dehydrogenase) return to normal. For CAD patients, CR criteria also include: undetectable clonal B cells and clonal IgM. Hematological complete remission with compensatory hemolytic state (CRi) is defined as Hb≥110g/L (female) or Hb≥120g/L (male); and hemolysis-related laboratory indicators (serum bilirubin and lactate dehydrogenase) improve but do not return to normal levels. Partial response (PR) is defined as Hb ≥ 100 g/L or an increase of ≥ 20 g/L from baseline; and at least 7 days without blood transfusion.
Measured: Six months post-infusion
Changes in laboratory test indicators
Changes in Hemoglobin,reticulocytes, serum bilirubin, lactate dehydrogenase, and haptoglobin; clonal B cells and monoclonal immunoglobulin.
Measured: Up to 24 months post-infusion
Drug-free Remission (DFR)
The duration from achieving CR, CRi, or PR to the time of relapse
Measured: Up to 24 months post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2026
- Study start2 Jul 2026
- Primary completion1 Sep 2028 (estimated)
- Study completion1 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune Hemolytic Anemia (AIHA)Studies of the same condition, open and under way.
- More trials run by Juventas Cell Therapy Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Juventas Cell Therapy Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07748533
- Sponsor's protocol ID
- HY001N101
- Status last verified
- Aug 2026
- First submitted
- 25 Jun 2026
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07748533
- Acronym / sponsor ID
- HY001N101
- Conditions
- Autoimmune Hemolytic Anemia (AIHA)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Juventas Cell Therapy Ltd.Industry (pharma, biotech, devices)
- Study start
- 2 Jul 2026 (actual)
- Primary completion
- 1 Sep 2028 (estimated)
- Study completion
- 1 Oct 2028 (estimated)
- First posted
- 5 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants who take HY001N cell injection | Experimental | Drug: HY001N Cell InjectionA conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by investigational treatment, HY001N Cell Injection. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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;
- 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.)
- Failure of first-line rituximab therapy; Failure of second-line rituximab ± Bendamostine/fludalabine; Failure of third-line therapy (bortezomib, Sutimlimab, Pegcetacoplan, Eculizumab, etc.)
Key exclusion criteria (20 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HY001N Cell InjectionDrug: 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 | Drug | Participants who take HY001N cell injection |
Primary outcome
Incidence of Treatment-related Adverse Events
Time frame: Up to 28 days post-infusion
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)
The safe dosage for a single infusion of HY001N Cell Injection
Time frame: Up to 28 days post-infusion
The safe dosage for AIHA patients will be evaluated by comprehensively assessing the Overall Response Rate (ORR) and the incidence of adverse events (AEs).
3 secondary outcomes
Overall Remission Rate (ORR)
Time frame: Six months post-infusion
Changes in laboratory test indicators
Time frame: Up to 24 months post-infusion
Drug-free Remission (DFR)
Time frame: Up to 24 months post-infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HY001N Cell Injection in people with Autoimmune Hemolytic Anemia (AIHA), run by Juventas Cell Therapy Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaofeng PAN (+86 13295841367, panxiaofeng@juventas.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-related Adverse Events (time frame: Up to 28 days post-infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2026.