Early Clinical Study of UTAA09 Injection in the Treatment of Relapsed/Refractory
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- AE
研究概览
简要总结
Main purpose:
To evaluate the safety of UTAA09 injection in the treatment of relapsed/refractory (R/R) autoimmune disease (AID).
Secondary purpose:
To evaluate the pharmacokinetic (PK) profile of UTAA09 injection in patients with R/R AID.
To evaluate the pharmacodynamic (PD) characteristics of UTAA09 injection in patients with R/R AID.
To evaluate the initial efficacy of UTAA09 injection in the treatment of R/R AID subjects.
To evaluate the immunogenicity of UTAA09 injection in R/R AID subjects.
详细描述
This clinical trial was designed as a single-arm, open-label, single-center, investigator-initiated early-stage clinical study to evaluate the safety of UTAA09 injection in patients with relapsed/refractory AID. After signing the informed consent letter, qualified subjects were screened for infusion of UTAA09 injection, and their blood was collected before and after infusion for pharmacokinetics, pharmacodynamics, immunogenicity, safety and other evaluation. In addition to the baseline period, the therapeutic efficacy was evaluated at the frequency of 14d, 28d, 2m, 4m, 6m, 8m, 10m, 12m, 15m, 18m, 21m, 24m after cell transfusion until disease progression (PD), new anti-disease therapy, death, intolerable toxicity, investigator decision, or subject's voluntary withdrawal. Whichever comes first.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •(2) Expected survival time ≥3 months; (3) Subjects with recurrent/refractory autoimmune diseases who have failed standard treatment or lack effective treatment, Including but not limited to systemic lupus erythematosus, idiopathic inflammatory myopathy, systemic sclerosis, IGG4-associated diseases, primary Sjogren's syndrome, rheumatoid arthritis, connective tissue disease-associated interstitial lung disease, immune thrombocytopenia, primary biliary cholangitis, etc.
- •(3) Histological evidence of non-suppurative destructive cholangitis and small bile duct destruction.
- •(4) Liver and kidney function, cardiopulmonary function meet the following requirements:
- •Creatinine ≤1.5×ULN; (2) Electrocardiogram showed no clinically significant abnormal bands;
- •Blood oxygen saturation >91% in non-oxygen state;
- •Total bilirubin ≤2×ULN; ALT and AST≤2.5 x ULN; ALT and AST abnormalities due to disease, such as liver infiltration or bile duct obstruction, were determined to be less than 5×ULN. If Gilbert syndrome is diagnosed, the total bilirubin index can be relaxed to ≤3.0×ULN and the direct bilirubin ≤1.5×ULN.
- •(5) no serious mental disorders; (6) Can understand this test and have signed the informed consent.
排除标准
- •Malignant tumors other than R/R AID disease in the 5 years prior to screening, except for adequately treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, local prostate cancer after radical surgery, and breast ductal carcinoma in situ after radical surgery;
- •Hepatitis B surface antigen (HBsAg) positive; Hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer detection is not within the normal reference value range; Hepatitis C virus (HCV) Antibody positive and peripheral blood hepatitis C virus (HCV) RNA positive; Human immunodeficiency virus (HIV) Antibody positive; Syphilis positive;
- •Serious heart disease, including but not limited to unstable angina, myocardial infarction or bypass or stent surgery (within 6 months prior to screening), congestive heart failure (NYHA classification ≥III), and severe arrhythmia;
- •Systemic diseases that are deemed unstable by researchers: including but not limited to severe liver, kidney, or metabolic diseases that require drug treatment;
- •Active or uncontrollable infections (except mild genitourinary and upper respiratory tract infections) that require systemic treatment within 7 days prior to administration;
- •Pregnant or lactating women, and female subjects who plan pregnancy within 2 years after cell transfusion or male subjects whose partners plan pregnancy within 2 years after cell transfusion;
- •Patients who received CAR-T therapy or other gene-modified cell therapy before screening;
- •Participated in other clinical studies 1 month before screening;
- •Evidence of central nervous system invasion during subject screening;
- •Mental patients with depression or suicidal thoughts;
- •Those who received live vaccine within 28 days prior to screening;
- •Situations considered unsuitable for inclusion by other researchers.
结局指标
主要结局
AE
时间窗: 3 months after cell reinfusion or disease progression/recurrence or start of new anti-disease therapy (whichever occurs first)
Type, frequency, and severity of adverse events (AEs) and laboratory abnormalities according to the Common Adverse Event Evaluation Standard NCI CTCAE version 5.0
次要结局
- Disease remission/response/improvement rates(3 months after cell reinfusion or disease progression/recurrence or start of new anti-disease therapy (whichever occurs first))
- anti-CAR antibody(3 months after cell reinfusion or disease progression/recurrence or start of new anti-disease therapy (whichever occurs first))
- Cmax(3 months after cell reinfusion or disease progression/recurrence or start of new anti-disease therapy (whichever occurs first))
- CD19-positive cells(3 months after cell reinfusion or disease progression/recurrence or start of new anti-disease therapy (whichever occurs first))