Prospective Single-Arm Clinical Trial of Golidocitinib Monotherapy for Hemophagocytic Lymphohistiocytosis.
概览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 28
- 试验地点
- 1
- 主要终点
- Evaluation of treatment response
概览
简要总结
This study aims to investigate the efficacy and safety of Golidocitinib(GO) monotherapy in the treatment of hemophagocytic lymphohistiocytosis.
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
- 14 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Researchers evaluate patients with newly diagnosed or refractory/relapsed HLH disease.
- •Definite diagnosis of HLH: molecular diagnosis consistent with pHLH, or at least 5 of the 8 criteria in the HLH-2004 diagnostic criteria.
- •Patients who are currently unsuitable or unable to undergo allo-HSCT.
- •Researchers estimate that the expected survival period exceeds one month.
- •Patients must be at least 14 years of age and no older than 70 years of age, with no gender restrictions.
- •Before the study began, TB was ≤10 times the upper limit of normal; Cr was ≤1.5 times the normal value.
- •Serum HIV antigen or antibody negative。
- •HCV antibody negative, or HCV antibody positive but HCV RNA negative.
- •.HBsAg and HBcAb are both negative. If either of the above is positive, peripheral blood hepatitis B virus DNA titer testing is required, with a titer of less than 1×10³ copies/ml.
- •Echocardiography showed LVEF ≥ 50%.
排除标准
- •Heart function above grade II(including II) (NYHA).
- •Pregnancy or lactating Women and reproductive-age patients who refused to use appropriate contraceptive measures during this trial.
- •Individuals who are allergic to GO or have a severe allergic constitution.
- •Active bleeding of the internal organs.
- •uncontrollable infection.
- •Severe mental illness.
- •History of non-melanoma skin cancer.
- •Unable to comply during the trial and/or follow-up phase. Participate in other clinical research at the same time.
研究组 & 干预措施
HLH patients
Initial diagnosis or presence of refractory/relapsed HLH disease or confirmed diagnosis of HLH.
干预措施: Golidocitinib (Drug)
结局指标
主要结局
Evaluation of treatment response
时间窗: Weeks 2, 4, 6, and 8 after the start of treatment
A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, triglyceride, blood cell count, hemophagocytosis, and level of consciousness (if CNS HLH is present). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; without blood transfusion: Neutrophils \<500/ml must increase by 100% and be \>500/ml; neutrophils 500-2000/ml must increase by 100% and return to normal.; and for patients with ALT \>400 U/L, response was defined as an ALT decrease of at least 50%. No response is defined as not meeting the criteria for partial response.
次要结局
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0(30 days after the last dose is administered.)
研究者
Zhao Wang
Doctor
Beijing Friendship Hospital