Autoimmune ThrombocytopeniaPhase 1NCT06826430
Inaticabtagene Autoleucel Injection in the Treatment of Refractory Systemic Lupus Erythematosus-related Immune Thrombocytopenia
A Phase 1 study testing Inaticabtagene autoleucel Injection in people with Autoimmune Thrombocytopenia, run by Juventas Cell Therapy Ltd. at 1 site in China.
Trial summary
This is a single-arm, open-label，phase I clinical study to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection in treatment of refractory systemic lupus erythematosus-related immune thrombocytopenia. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Autoimmune Thrombocytopenia
- 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
- 1 siteChina
- Study dates
- 1 Mar 2025to 31 Dec 2027
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age range: 18-70 years (including 18 and 70 years), regardless of gender.
- Clinically diagnosed with Refractory Systemic Lupus Erythematosus-related Immune Thrombocytopenia according to the revised criteria of ACR in 1997 or EULAR/ACR in 2019. At least two consecutive blood routine tests showing platelet counts <50×10^9/L; Peripheral blood smear microscopy showed no significant abnormalities in the morphology of blood cells; The morphological characteristics of bone marrow cells are consistent with immune thrombocytopenia. Treated at least 1 course of MP shock therapy or high-dose steroids, combined with one or more immunosuppressive agents (including biologics) for at least 3 months but not achieving partial remission, or the efficacy cannot be maintained during the steroid reduction process.
- During the study period, the use of corticosteroids at a dose not exceeding 10mg prednisone or its equivalent, all immunosuppressants (excluding hydroxychloroquine) should be discontinued.
- Women of childbearing potential must have a negative blood pregnancy test 7 days prior to trial conditioning therapy; 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).
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- 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;
- Total serum bilirubin ≤ 2 times ULN unless the patient has documented Gilbert's syndrome; patients with Gilbert's syndrome who have bilirubin ≤ 3.0 times ULN and direct bilirubin ≤ 1.5 times ULN may be included;
- Serum creatinine ≤ 1.5 times ULN, or creatinine clearance ≥ 60 mL/min (Cockcroft and Gault formula), Patients with lupus nephritis may relax the conditions appropriately according to the judgment of the investigator;
- Must have minimal pulmonary reserve and oxygen saturation > 91% in a nonoxygenated state;
- Lymphocyte count > 0.4 × 109/L.
Who cannot join
- Severe active central nervous system (CNS) lupus, including seizures, psychosis, cerebrovascular accidents, or CNS vasculitis requiring therapeutic intervention within 60 days after baseline.
- Dialysis patients or creatinine clearance rate less than 30mL/min.
- Pregnancy or breastfeeding.
- Merge active infections (such as sepsis, bacteremia, mycosis, uncontrolled lung infections, and active tuberculosis).
- Hepatitis B surface antigen (HBsAg) and/or hepatitis B e antigen (HBeAg) are positive; Hepatitis B e antibody (HBe Ab) and/or hepatitis B core antibody (HBcAb) are positive, and the number of HBV-DNA copies is greater than the measurable lower limit; Hepatitis C (HCV) antibody positive; positive for human immunodeficiency virus (HIV) antibodies; positive for syphilis antibody (TP Ab);
- Major surgery that was assessed as unsuitable by the investigators within 4 weeks before screening.
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- 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 that require treatment (excluding atrial fibrillation and paroxysmal supraventricular tachycardia); QTcB interval ≥ 450ms for males and ≥ 470ms for females (QTcB=QT/RR1/2); Have had myocardial infarction, bypass or stent surgery within the 6 months prior to the study; Other heart diseases that have been determined by researchers to be unsuitable for inclusion;
- Patients with clinically significant pleural effusion during screening.
- Patients vaccinated with a live vaccine within 6 weeks prior to screening.
- Patients with deep vein thrombosis within 6 months prior to screening, or a history of pulmonary embolism.
- Patients with a life expectancy of less than 6 months.
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
wenqiu Huang, Dr. contact
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality
Mengtao Li, Dr.
About this study
Official title: A Multi-center, Open-label, Single-arm Phase I Clinical Study to Evaluate the Safety and Tolerability of Inaticabtagene Autoleucel Injection in Treatment of Refractory Systemic Lupus Erythematosus-related Immune Thrombocytopenia
This is a single-arm, open-label，phase I clinical study to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection in treatment of refractory systemic lupus erythematosus-related immune thrombocytopenia.
Read the detailed description
This is a single arm, open-label, non-randomized, dose-escalation, phase I study to determine the safety and tolerability of Inaticabtagene Autoleucel Injection in treatment of refractory systemic lupus erythematosus-related immune thrombocytopenia and determine the Phase II Recommended Dose (RP2D). The study will have the following sequential phases: Screening, Pre-Treatment (Cell Product Preparation \& Lymphodepleting Chemotherapy), Treatment and Follow-up, and Survival Follow-up. The total duration of the study is 2 years from Inaticabtagene Autoleucel Injection infusion.
- Conditions
- Autoimmune Thrombocytopenia
- Being tested
- Inaticabtagene autoleucel Injection
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inaticabtagene autoleucel InjectionDrug
Inaticabtagene autoleucel Injection, the autologous 2nd generation CD19-directed CAR-T cells, will be administered by vein. Before CAR-T infusion，patients will get a 3-4 days lymphodepletion therapy with fludarabine and cyclophosphamide.
| Study group | Role | What they receive |
|---|---|---|
| Single dose of Inaticabtagene Autoleucel Injection | Experimental | Subjects who meet the enrollment conditions will receive intravenous infusion of CAR-T cells after lymphodepletion. 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 ITP 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 (9)
Overall Remission Rate (ORR)
The proportion of subjects achieving CR or PR after treatment by Inaticabtagene Autoleucel Injection.CR is defined as platelet count ≥100×10^9/L. PR is defined as platelet count ≥30×10^9/L and at least doubling from baseline, without active bleeding.
Measured: Up to 6 months post-infusion
Proportion of subjects who achieved disease remission (DORIS)
The proportion of subjects to meet the following 2 criteria: 1.Clinical lupus erythematosus disease activity index (SLEDAI)=0. 2.Physician global assessment (PGA) is less than 0.5 (standard score is 0-3 points) without considering serological testing and low dose corticosteroids with a dose of ≤ 5mg/d (taking prednisone as an example) permitted. Antimalarial drugs, stable dose immunosuppressants, including biologics are allowed to be used.
Measured: Up to 6 months post-infusion
Proportion of subjects who achieved lupus hypoactivity state (LLDAS)
The proportion of subjects to meet the following 5 criteria:
- SLEDAI score ≤ 4, no activity of important organs, no hemolytic anemia or gastrointestinal activity;
- Compared with previous assessments, there is no new lupus activity;
- SELENA-SLEDAI physician overall assessment (PGA 0-3) ≤ 1 point;
- The current dose of prednisone is ≤ 7.5mg/d;
- Immunosuppressants and biologics with standard maintenance doses are well tolerated.
Measured: Up to 6 months post-infusion
Improvement in systemic lupus erythematosus scores (SELENA-SLEDAI)
0 to 4 is basically no disease activity;5 to 9 is light activity;10 to 14 is moderate activity;≥15 is considered heavy activity.
Measured: Up to 24 months post-infusion
Changes in MOS item short from health survey(SF-36)
Measured: Up to 24 months post-infusion
Changes in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
Measured: Up to 24 months post-infusion
Changes in serological markers
Changes in IgG, IgM, IgA, IgE levels compared to baseline.
Measured: Up to 24 months post-infusion
Changes in serological markers
Changes in SLE disease serological markers (anti ds-DNA antibody, anti nuclear antibody AG, complement C3, C4) levels compared to baseline.
Measured: Up to 24 months post-infusion
Reduction of Combination therapy evaluation
The duration of the patient's cessation or reduction of the combination therapy dose, as well as the degree of reduction in the combination therapy compared to baseline.
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 posted14 Feb 2025
- Study start1 Mar 2025
- Primary completion31 Mar 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune ThrombocytopeniaStudies 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
- Mengtao Li, Dr., Principal investigator, Peking Union Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06826430
- Sponsor's protocol ID
- HY001105
- Status last verified
- Aug 2025
- First submitted
- 6 Feb 2025
- Last updated
- 7 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label, Single-arm Phase I Clinical Study to Evaluate the Safety and Tolerability of Inaticabtagene Autoleucel Injection in Treatment of Refractory Systemic Lupus Erythematosus-related Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06826430
- Acronym / sponsor ID
- HY001105
- Conditions
- Autoimmune Thrombocytopenia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Juventas Cell Therapy Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Mar 2025 (actual)
- Primary completion
- 31 Mar 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 14 Feb 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 single-arm, open-label，phase I clinical study to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection in treatment of refractory systemic lupus erythematosus-related immune thrombocytopenia.
Detailed description
This is a single arm, open-label, non-randomized, dose-escalation, phase I study to determine the safety and tolerability of Inaticabtagene Autoleucel Injection in treatment of refractory systemic lupus erythematosus-related immune thrombocytopenia and determine the Phase II Recommended Dose (RP2D). The study will have the following sequential phases: Screening, Pre-Treatment (Cell Product Preparation \& Lymphodepleting Chemotherapy), Treatment and Follow-up, and Survival Follow-up. The total duration of the study is 2 years from Inaticabtagene Autoleucel Injection infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single dose of Inaticabtagene Autoleucel Injection | Experimental | Drug: Inaticabtagene autoleucel InjectionSubjects who meet the enrollment conditions will receive intravenous infusion of CAR-T cells after lymphodepletion. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Age range: 18-70 years (including 18 and 70 years), regardless of gender.
- Clinically diagnosed with Refractory Systemic Lupus Erythematosus-related Immune Thrombocytopenia according to the revised criteria of ACR in 1997 or EULAR/ACR in 2019. At least two consecutive blood routine tests showing platelet counts <50×10^9/L; Peripheral blood smear microscopy showed no significant abnormalities in the morphology of blood cells; The morphological characteristics of bone marrow cells are consistent with immune thrombocytopenia. Treated at least 1 course of MP shock therapy or high-dose steroids, combined with one or more immunosuppressive agents (including biologics) for at least 3 months but not achieving partial remission, or the efficacy cannot be maintained during the steroid reduction process.
- During the study period, the use of corticosteroids at a dose not exceeding 10mg prednisone or its equivalent, all immunosuppressants (excluding hydroxychloroquine) should be discontinued.
- Women of childbearing potential must have a negative blood pregnancy test 7 days prior to trial conditioning therapy; 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):
- Adequate organ function according to the following criteria:
Key exclusion criteria (17 listed)
- Severe active central nervous system (CNS) lupus, including seizures, psychosis, cerebrovascular accidents, or CNS vasculitis requiring therapeutic intervention within 60 days after baseline.
- Dialysis patients or creatinine clearance rate less than 30mL/min.
- Pregnancy or breastfeeding.
- Merge active infections (such as sepsis, bacteremia, mycosis, uncontrolled lung infections, and active tuberculosis).
- Hepatitis B surface antigen (HBsAg) and/or hepatitis B e antigen (HBeAg) are positive; Hepatitis B e antibody (HBe Ab) and/or hepatitis B core antibody (HBcAb) are positive, and the number of HBV-DNA copies is greater than the measurable lower limit; Hepatitis C (HCV) antibody positive; positive for human immunodeficiency virus (HIV) antibodies; positive for syphilis antibody (TP Ab);
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 InjectionInaticabtagene autoleucel Injection, the autologous 2nd generation CD19-directed CAR-T cells, will be administered by vein. Before CAR-T infusion，patients will get a 3-4 days lymphodepletion therapy with fludarabine and cyclophosphamide. | Drug | Single dose of 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 ITP patients will be evaluated by comprehensively assessing the Overall Response Rate (ORR) and the incidence of adverse events (AEs).
9 secondary outcomes
Overall Remission Rate (ORR)
Time frame: Up to 6 months post-infusion
Proportion of subjects who achieved disease remission (DORIS)
Time frame: Up to 6 months post-infusion
Proportion of subjects who achieved lupus hypoactivity state (LLDAS)
Time frame: Up to 6 months post-infusion
Improvement in systemic lupus erythematosus scores (SELENA-SLEDAI)
Time frame: Up to 24 months post-infusion
Changes in MOS item short from health survey(SF-36)
Time frame: Up to 24 months post-infusion
Changes in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
Time frame: Up to 24 months post-infusion
Changes in serological markers
Time frame: Up to 24 months post-infusion
Changes in serological markers
Time frame: Up to 24 months post-infusion
Reduction of Combination therapy evaluation
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 7 Aug 2025.
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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.
wenqiu Huang, Dr. contact
Mengtao Li, Dr. principal investigator
Peking Union Medical College
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 Inaticabtagene autoleucel Injection in people with Autoimmune Thrombocytopenia, 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 13 inclusion and 17 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 7 Aug 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?
Contact Junyin Yu (+86 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 7 Aug 2025.