AIHA - Cold Autoimmune Hemolytic AnemiaPhase 1NCT07091370
Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia After Three or More Lines of Therapy
A Phase 1 study testing Inaticabtagene Autoleucel Injection in people with AIHA - Cold Autoimmune Hemolytic Anemia, run by Juventas Cell Therapy Ltd..
Trial summary
This is a multicenter, open-label, single-arm Phase I clinical trial to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection treatment in autoimmune hemolytic anemia that has failed at least three lines of treatment. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- AIHA - Cold Autoimmune Hemolytic Anemia
- What is being tested?
- Inaticabtagene Autoleucel Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- 27 Dec 2025to 30 Mar 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
- Age range: 18-75 years (including 18 and 75 years), regardless of gender.
- Clinically diagnosed with AIHA or Evans, with hemoglobin lower than 100g/L and symptoms related to anemia according to the diagnostic criteria of the "Diagnosis and Treatment Guidelines for Autoimmune Hemolytic Anemia in Chinese Adults (2023 Edition)" and the "Chinese Expert Consensus on the Diagnosis and Treatment of Evans Syndrome (2024 Edition)". And meet the diagnostic criteria of failure in at least three lines of treatment as follows:
- Patients diagnosed with warm resistance type AIHA (wAIHA)/mixed AIHA (mAIHA) /Evans syndrome, the following conditions must be met: first-line glucocorticoid treatment failed, second-line rituximab treatment failed and failure of third-line treatment measures (including any one or more of splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, fludarabine, bortezomib, etc.) Patients diagnosed with cold-resistant type AIHA (cAIHA), that is, cold agglutinin disease (CAD), the following conditions must be met: first-line rituximab treatment failed, second-line rituximab ± bendamustine/fludarabine failed and failure of third-line treatment measures (including any one or more of bortezomib, cyclosporine, cyclophosphamide, mycophenolate mofetil, azathioprine, etc.)
- Women of childbearing potential must have a negative blood pregnancy test during the screening period. Any male and female patients of childbearing potential must agree to use an effective method of contraception throughout the study and for at least 2 year following infusion of CAR-T cells. Childbearing potential is biologically capable of bearing a living baby and sexually active. Female patients who were not of childbearing potential (ie, met at least 1 of the following criteria):
- Hysterectomy or oophorectomy, or medically confirmed ovarian failure, or medically confirmed postmenopausal (cessation of menses for at least 12 consecutive months in the absence of pathological or physiological causes).
- Adequate organ function according to the following criteria:
- Aspartate aminotransferase (AST) ≤ 3 times of upper limit of normal (ULN); Alanine aminotransferase (ALT) ≤ 3 times ULN; Serum creatinine ≤ 1.5 times ULN, or creatinine clearance ≥ 60 mL/min (Cockcroft and Gault formula); International normalized ratio (INR) ≤ 1.5 times ULN, and activated partial thromboplastin time (APTT) ≤ 1.5 times the ULN; Must have minimal pulmonary reserve and oxygen saturation > 93% in a nonoxygenated state;
- The Eastern Cancer Consortium (ECOG) performance status score is 0-2.
Who cannot join
- Previously diagnosed proliferative tumors of the lymphatic system;
- Secondary AIHA caused by drugs or infections;
- Pregnancy or breastfeeding;
- Previously undergone organ or hematopoietic stem cell transplantation;
- A history of new thrombosis or organ infarction within 6 months prior to screening;
- The active stage diagnosed as a connective tissue disease;
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- Suffering from hereditary hemolytic diseases or other acquired hemolytic diseases;
- Combined with active infections (such as sepsis, bacteremia, mycosis, uncontrolled pulmonary infection and active tuberculosis, etc.);
- Positive for hepatitis B surface antigen (HBsAg) and/or hepatitis Be antigen (HBeAg); Hepatitis Be antibody (HBe-Ab) and/or hepatitis B core antibody (HBc-Ab) are positive, and the HBV-DNA copy number is greater than the measurable lower limit; Positive for hepatitis C (HCV) antibody; Positive for human immunodeficiency virus (HIV) antibody; Those with positive syphilis (TP) test;
- Major surgery that was assessed as unsuitable by the investigators within 4 weeks before screening;
- Patients with concurrent active malignancy within the past five years, those with a history of malignancy but cuired are eligible.
- The patient's heart meets any of the following conditions:
- Left ventricular ejection fraction (LVEF) ≤45%;
- New York Heart Association (NYHA) grade III or IV congestive heart failure or active heart disease;
- Severe arrhythmias requiring treatment (excluding atrial fibrillation and paroxysmal supraventricular tachycardia);
- The QTcB interval was ≥450ms for males and ≥470ms for females (QTcB=QT/ R1/2);
- Had myocardial infarction, bypass surgery or stent surgery within 6 months before the study;
- Other heart diseases judged by the researchers as unsuitable for inclusion in the group;
- Have received live vaccines within 6 weeks before screening.
- Patients participating in any other interventional clinical study or receiving treatment of an active investigational drug within 3 months or 5 half-lives for launched drugs prior to Inaticabtagene Autoleucel Injection infusion.
- Patients with a history of epilepsy, cerebral ischemia/hemorrhage, cerebellar diseases, or other active central nervous system disorders;
- Patients with hypersensitivity reactions to the components of Inaticabtagene Autoleucel Injection;
- Patients previously received CAR-T cell therapy.
- Other circumstances where the researcher deems it inappropriate to participate in this clinical trial.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Xiaofeng Pan, Dr. contact
About this study
Official title: A Multicenter, Open-label, Single-arm Phase I Clinical Trial on the Safety and Tolerability of Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia That Has Failed at Least Three Lines of Treatment
This is a multicenter, open-label, single-arm Phase I clinical trial to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection treatment in autoimmune hemolytic anemia that has failed at least three lines of treatment.
Read the detailed description
This study will be conducted in Chinese hospitals and to include 6 to 12 subjects with autoimmune hemolytic anemia .
The main purpose of the study is to evaluate the safety and tolerability of Inaticabtagene Autoleucel 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 Inaticabtagene Autoleucel 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 Inaticabtagene Autoleucel 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
- AIHA - Cold Autoimmune Hemolytic Anemia
- Being tested
- Inaticabtagene Autoleucel Injection
- Keywords
- AIHA, CarT
- 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.
Inaticabtagene Autoleucel InjectionDrug
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: 0.25×10^6 CAR-T live cells /kg body weight
Group A (initial dose) : 0.5×10^6 CAR-T live cells /kg body weight
Group B: 1.0×10^6 CAR-T live cells /kg body weight
Group C: 1.5×10^6 CAR-T live cells /kg body weight
| Study group | Role | What they receive |
|---|---|---|
| Inaticabtagene Autoleucel Injection | Experimental | A conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by investigational treatment, Inaticabtagene Autoleucel Injection. Receives: Drug: Inaticabtagene Autoleucel 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 (CTCAE, Version 5.0)
Measured: Up to 28 days post-infusion
The safe dosage for a single infusion of Inaticabtagene Autoleucel 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 Inaticabtagene Autoleucel 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: Sixs months after cell reinfusion
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 posted29 Jul 2025
- Study start27 Dec 2025 (estimated)
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AIHA - Cold Autoimmune Hemolytic AnemiaStudies 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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Juventas Cell Therapy Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jun Shi, Dr., Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07091370
- Sponsor's protocol ID
- HY001106
- Status last verified
- Sep 2025
- First submitted
- 21 Jul 2025
- Last updated
- 22 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- 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 22 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Single-arm Phase I Clinical Trial on the Safety and Tolerability of Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia That Has Failed at Least Three Lines of Treatment
- ClinicalTrials.gov ID
- NCT07091370
- Acronym / sponsor ID
- HY001106
- Conditions
- AIHA - Cold Autoimmune Hemolytic Anemia
- Keywords
- AIHA, CarT
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Juventas Cell Therapy Ltd.Industry (pharma, biotech, devices)
- Study start
- 27 Dec 2025 (estimated)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Mar 2028 (estimated)
- First posted
- 29 Jul 2025 (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.
This is a multicenter, open-label, single-arm Phase I clinical trial to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection treatment in autoimmune hemolytic anemia that has failed at least three lines of treatment.
Detailed description
This study will be conducted in Chinese hospitals and to include 6 to 12 subjects with autoimmune hemolytic anemia .
The main purpose of the study is to evaluate the safety and tolerability of Inaticabtagene Autoleucel 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 Inaticabtagene Autoleucel 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 Inaticabtagene Autoleucel 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 |
|---|---|---|
| Inaticabtagene Autoleucel Injection | Experimental | Drug: Inaticabtagene Autoleucel InjectionA conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by investigational treatment, Inaticabtagene Autoleucel Injection. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age range: 18-75 years (including 18 and 75 years), regardless of gender.
- Clinically diagnosed with AIHA or Evans, with hemoglobin lower than 100g/L and symptoms related to anemia according to the diagnostic criteria of the "Diagnosis and Treatment Guidelines for Autoimmune Hemolytic Anemia in Chinese Adults (2023 Edition)" and the "Chinese Expert Consensus on the Diagnosis and Treatment of Evans Syndrome (2024 Edition)". And meet the diagnostic criteria of failure in at least three lines of treatment as follows:
- Patients diagnosed with warm resistance type AIHA (wAIHA)/mixed AIHA (mAIHA) /Evans syndrome, the following conditions must be met: first-line glucocorticoid treatment failed, second-line rituximab treatment failed and failure of third-line treatment measures (including any one or more of splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, fludarabine, bortezomib, etc.) Patients diagnosed with cold-resistant type AIHA (cAIHA), that is, cold agglutinin disease (CAD), the following conditions must be met: first-line rituximab treatment failed, second-line rituximab ± bendamustine/fludarabine failed and failure of third-line treatment measures (including any one or more of bortezomib, cyclosporine, cyclophosphamide, mycophenolate mofetil, azathioprine, etc.)
- Women of childbearing potential must have a negative blood pregnancy test during the screening period. Any male and female patients of childbearing potential must agree to use an effective method of contraception throughout the study and for at least 2 year following infusion of CAR-T cells. Childbearing potential is biologically capable of bearing a living baby and sexually active. Female patients who were not of childbearing potential (ie, met at least 1 of the following criteria):
- Hysterectomy or oophorectomy, or medically confirmed ovarian failure, or medically confirmed postmenopausal (cessation of menses for at least 12 consecutive months in the absence of pathological or physiological causes).
Key exclusion criteria (24 listed)
- Previously diagnosed proliferative tumors of the lymphatic system;
- Secondary AIHA caused by drugs or infections;
- Pregnancy or breastfeeding;
- Previously undergone organ or hematopoietic stem cell transplantation;
- A history of new thrombosis or organ infarction within 6 months prior to screening;
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 |
|---|---|---|
| Inaticabtagene Autoleucel InjectionThe 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: 0.25×10^6 CAR-T live cells /kg body weight Group A (initial dose) : 0.5×10^6 CAR-T live cells /kg body weight Group B: 1.0×10^6 CAR-T live cells /kg body weight Group C: 1.5×10^6 CAR-T live cells /kg body weight | Drug | Inaticabtagene Autoleucel 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 (CTCAE, Version 5.0)
The safe dosage for a single infusion of Inaticabtagene Autoleucel 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: Sixs months after cell reinfusion
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 22 Sep 2025.
- 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.
Xiaofeng Pan, Dr. contact
Jun Shi, Dr. principal investigator
Institute of Hematology & Blood Diseases Hospital, China
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Inaticabtagene Autoleucel Injection in people with AIHA - Cold Autoimmune Hemolytic Anemia, run by Juventas Cell Therapy Ltd..
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 8 inclusion and 24 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: opening soon, as of the sponsor's last update on 22 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
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 Junyin Yu (13920424844, Yujunyin@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 22 Sep 2025; AzTrials last refreshed its copy on 8 Nov 2026.