Autoimmune Hemolytic AnemiaPhase 1NCT06231368
CNCT19 for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy.
A Phase 1 study testing CNCT19 CAR-T cell therapy in people with Autoimmune Hemolytic Anemia and Autologous CD19 CAR-T, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a Phase 1, single-arm, open-label, dose-escalation and dose-expansion study. The main purpose is to evaluate the safety and tolerability, efficacy of CNCT19 CAR T-cell therapy in patients with autoimmune hemolytic anemia after failure of three or more lines of therapy. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Autoimmune Hemolytic Anemia, Autologous CD19 CAR-T, Failure of Three or More Lines of Therapy
- What is being tested?
- CNCT19 CAR-T cell therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 4 Feb 2024to 31 Aug 2025
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms
- Male or female age ≥ 12 years
- Subjects with autoimmune hemolytic anemia or Evans syndrome after Failure ≥3 lines of therapy. The Failure of ≥3 lines of therapy meets 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.)
- Female subjects of childbearing potential must have a negative Serum HCG test within 7 days before enrollment. Subjects of childbearing potential will be required to follow contraception requirements from the time of enrollment until the 1-year follow-up after cell infusion
- Laboratory tests of adequate organ function: Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3×ULN; and have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and the blood oxygen saturation in a non-oxygenated state is >93%
- ECOG performance status ≤2
- Subject with a life expectancy of more than 3 months
Who cannot join
- History of other lymphoproliferative neoplasms
- Secondary AIHA caused by drugs or infection
- Platelets in subjects with Evans syndrome<30×10^9/L
- Pregnant or breast-feeding subjects
- Treatment with any of the following within the noted period prior to study entry: 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
- Previously received organ or stem cell transplantation
Show all 20Show fewer
- History of new thrombosis or organ infarction in the past 6 months
- Diagnosis of the active stage of the 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 (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
- 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 men and ≥470ms for women, e.have myocardial infarction, bypass surgery, or stent placement within the 6 months before the study, 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 interventional clinical studies during CNCT19 CAR T-Cell therapy, and the drug has a half-life of <5. Subjects treated with active investigational drugs or intend to participate in another clinical trial or receive treatment other than that specified in the protocol throughout the study period
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Safety and Efficacy of CNCT19 for Patients With Autoimmune Hemolytic Anemia After Failure of Three or More Lines of Therapy
This is a Phase 1, single-arm, open-label, dose-escalation and dose-expansion study. The main purpose is to evaluate the safety and tolerability, efficacy of CNCT19 CAR T-cell therapy in patients with autoimmune hemolytic anemia after failure of three or more lines of therapy. Participants will receive CNCT19 cell infusion after preconditioning, and they will receive a 1-year follow-up.
- Conditions
- Autoimmune Hemolytic Anemia, Autologous CD19 CAR-T, Failure of Three or More Lines of Therapy
- Being tested
- CNCT19 CAR-T cell therapy
- Keywords
- Autoimmune Hemolytic Anemia, CNCT19 CAR T-Cell therapy
- 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.
CNCT19 CAR-T cell therapyBiological
CNCT19 was a second-generation CAR T-cell with scFv derived from clone HI19α and 4-1BB/CD3-ζ costimulatory domain.
| Study group | Role | What they receive |
|---|---|---|
| CNCT19 CAR T-Cell Therapy | Experimental | Participants will receive CNCT19 cell infusion after preconditioning, and they need to be closely monitored for 24 hours following CAR-T cell infusion. Participants are advised to remain in the hospital for a minimum of 14 days following cell infusion. The duration of hospitalization and observation will be determined based on the researcher's comprehensive assessment of the subject's condition. Receives: Biological: CNCT19 CAR-T cell therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and the severity of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Measured: Within 12 months
Secondary outcomes (1)
Percentage of patients with hematological response
Hematological response is mainly evaluated by hemoglobin (Hb), laboratory Indicators of hemolysis (serum bilirubin and lactate dehydrogenase) and blood transfusion.
Measured: Within 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2024
- Study start4 Feb 2024
- Primary completion18 Nov 2024
- Study completion31 Aug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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
- 6 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Juventas Cell Therapy Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06231368
- Sponsor's protocol ID
- IIT2024003
- Status last verified
- Nov 2024
- First submitted
- 22 Jan 2024
- Last updated
- 16 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Li R, Pan H, Zhang L, Ma J, Li W, Gao Z, Fang L, Tian L, Shen Y, Yang F, Zhao J, Nie N, Li J, Wang W, Pan X, Lian Y, Li X, Peng G, Li L, Yu X, Xu C, Liu Y, Kuang Z, Huang J, Zhao X, Ge M, Liu L, Chen S, Feng Y, Chang AH, Deng B, Dai M, Huang L, Lv L, Zheng Y, Luo Y, Xiong H, Shi J. CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia. N Engl J Med. 2026 Jan 15;394(3):253-267. doi: 10.1056/NEJMoa2509820.
PubMed 41534043
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Safety and Efficacy of CNCT19 for Patients With Autoimmune Hemolytic Anemia After Failure of Three or More Lines of Therapy
- ClinicalTrials.gov ID
- NCT06231368
- Acronym / sponsor ID
- IIT2024003
- Conditions
- Autoimmune Hemolytic Anemia, Autologous CD19 CAR-T, Failure of Three or More Lines of Therapy
- Keywords
- Autoimmune Hemolytic Anemia, CNCT19 CAR T-Cell therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Juventas Cell Therapy Ltd.
- Study start
- 4 Feb 2024 (actual)
- Primary completion
- 18 Nov 2024 (actual)
- Study completion
- 31 Aug 2025 (estimated)
- First posted
- 30 Jan 2024 (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 Phase 1, single-arm, open-label, dose-escalation and dose-expansion study. The main purpose is to evaluate the safety and tolerability, efficacy of CNCT19 CAR T-cell therapy in patients with autoimmune hemolytic anemia after failure of three or more lines of therapy. Participants will receive CNCT19 cell infusion after preconditioning, and they will receive a 1-year follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CNCT19 CAR T-Cell Therapy | Experimental | Biological: CNCT19 CAR-T cell therapyParticipants will receive CNCT19 cell infusion after preconditioning, and they need to be closely monitored for 24 hours following CAR-T cell infusion. Participants are advised to remain in the hospital for a minimum of 14 days following cell infusion. The duration of hospitalization and observation will be determined based on the researcher's comprehensive assessment of the subject's condition. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms
- Male or female age ≥ 12 years
- Subjects with autoimmune hemolytic anemia or Evans syndrome after Failure ≥3 lines of therapy. The Failure of ≥3 lines of therapy meets 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.)
- Female subjects of childbearing potential must have a negative Serum HCG test within 7 days before enrollment. Subjects of childbearing potential will be required to follow contraception requirements from the time of enrollment until the 1-year follow-up after cell infusion
- Laboratory tests of adequate organ function: Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3×ULN; and have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and the blood oxygen saturation in a non-oxygenated state is >93%
Key exclusion criteria (20 listed)
- History of other lymphoproliferative neoplasms
- Secondary AIHA caused by drugs or infection
- Platelets in subjects with Evans syndrome<30×10^9/L
- Pregnant or breast-feeding subjects
- Treatment with any of the following within the noted period prior to study entry: 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
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 |
|---|---|---|
| CNCT19 CAR-T cell therapyCNCT19 was a second-generation CAR T-cell with scFv derived from clone HI19α and 4-1BB/CD3-ζ costimulatory domain. | Biological | CNCT19 CAR T-Cell Therapy |
Primary outcome
Incidence and the severity of the adverse event
Time frame: Within 12 months
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
1 secondary outcome
Percentage of patients with hematological response
Time frame: Within 24 weeks
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 16 May 2025.
- Derived from this study: PubMed 41534043Li R, Pan H, Zhang L, Ma J, Li W, Gao Z, Fang L, Tian L, Shen Y, Yang F, Zhao J, Nie N, Li J, Wang W, Pan X, Lian Y, Li X, Peng G, Li L, Yu X, Xu C, Liu Y, Kuang Z, Huang J, Zhao X, Ge M, Liu L, Chen S, Feng Y, Chang AH, Deng B, Dai M, Huang L, Lv L, Zheng Y, Luo Y, Xiong H, Shi J. CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia. N Engl J Med. 2026 Jan 15;394(3):253-267. doi: 10.1056/NEJMoa2509820.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
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 CNCT19 CAR-T cell therapy in people with Autoimmune Hemolytic Anemia and Autologous CD19 CAR-T, run by Institute of Hematology & Blood Diseases Hospital, China 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 7 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: under way, not looking for participants, as of the sponsor's last update on 16 May 2025. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and the severity of the adverse event (time frame: Within 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06231368, which also lists 1 linked publication.
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 16 May 2025.