Autoimmune
Conditions
Brief summary
The purpose of this study is to determine the safety, tolerability and preliminary efficacy of NEUK203-215 , a healthy donor (HD) allogeneic CD19/BCMA-targeted CAR-NK cell product, in participants with severe, refractory autoimmune diseases. This study plans to conduct an interim data analysis when the cumulative enrollment reaches 50% of the total sample size. The objectives of this analysis are: (1) to summarize the safety and tolerability profile at the current dose level; (2) to preliminarily assess trends in clinical response endpoints and explore preliminary signals of drug efficacy; and (3) to preliminarily evaluate the pharmacokinetic and pharmacodynamic characteristics of the drug in humans. This analysis is exploratory in nature and is intended to accumulate preliminary clinical evidence for the drug in the target patient population.
Interventions
DRUGFludarabine
Specified dose on specified days
Specified dose on specified days
GENETICNEUK203-215 Injection
Specified dose on specified days
Sponsors
Neukio Biotherapeutics (Shanghai) Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* 1\) Has provided signed informed consent; 2) Is aged 18-65 years ; 3)Adequate functional reserve of vital organs 4) Has a confirmed diagnosis of systemic lupus erythematosus, diffuse systemic sclerosis, inflammatory myopathy, or autoimmune bullous dermatosis.
Exclusion criteria
* 1\) Requires dialysis treatment. 2) History of severe drug hypersensitivity. 3) Active infection requiring systemic therapy or suspected uncontrolled infection. 4\) Within 6 months before screening, any of the following cardiovascular events: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmia, any ventricular arrhythmia, or other clinically significant cardiac disease. 5\) Malignancy within the past 5 years. 6) Clinically significant chronic or intermittent bleeding within 60 days before the screening visit. 7\) Prior solid-organ (e.g., heart, lung, kidney, liver) or hematopoietic stem-cell/bone-marrow transplantation. 8\) At screening: positive HBsAg and/or HBcAb with HBV DNA detectable or above the lower limit of quantitation; positive HCV antibody with HCV RNA detectable or above the lower limit of quantitation; positive HIV antibody; positive syphilis test (except biologic false-positive results). 9\) Major surgery within 4 weeks before screening. 10) Live or live-attenuated vaccine received within 4 weeks before screening. 11) Uncontrolled concurrent medical conditions. 12) Documented history of neurologic or psychiatric disorders. 13) Any other factor that, in the investigator's judgment, could require premature withdrawal from the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse events | Up to 12 months post NEUK203-215 infusion |
| Existence/non existence of dose limiting toxicity (DLT) | Up to 12 months post NEUK203-215 infusion |
| serious adverse events (SAEs) | Up to 12 months post NEUK203-215 infusion |
Countries
China
Contacts
CONTACTHuji Xu
Outcome results
None listed