NCT07714512招募中1 期
A Phase I/II Study on the Safety and Efficacy of CD38 Monoclonal Antibody in the Treatment of Refractory Severe Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China1 个研究点 分布在 1 个国家目标入组 32 人开始时间: 2026年6月30日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Incidence of adverse events
研究概览
简要总结
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB) <90 g/L at enrollment. If the phase I results demonstrate an acceptable safety profile and allow determination of the maximum tolerated dose (MTD), the phase II part will be initiated directly to evaluate the efficacy of isatuximab.
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
- •Diagnosed with primary acquired aplastic anemia according to the 2024 British Society for Haematology guideline, Guidelines for the Diagnosis and Management of Adult Aplastic Anaemia, and the Chinese Guideline for the Diagnosis and Treatment of Aplastic Anemia (2022 edition) issued by the Hematology Branch of the Chinese Medical Association.
- •Previously diagnosed with severe aplastic anemia (SAA) or very severe aplastic anemia (VSAA), with no response or relapse after receiving anti-thymocyte/anti-lymphocyte globulin (ATG/ALG) in combination with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin receptor agonist (TPO-RA) therapy for at least 4 months.
- •Hemoglobin <90 g/L or platelet count <30×10^9/L
- •Unsuitable for or unwilling to undergo hematopoietic stem cell transplantation, with no better available treatment options.
- •Age ≥18 years, regardless of gender.
- •Eastern Cooperative Oncology Group (ECOG) performance status score ≤2
- •Willing and able to comply with the requirements for this study and written informed consent.
排除标准
- •Diagnosed with congenital bone marrow failure syndromes
- •Bone marrow reticulin fibrosis grade ≥2
- •Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or active hemolysis
- •Subjects with clonal cytogenetic abnormalities characteristic of myelodysplastic syndromes, except +8, del(20q), and -Y
- •Active bacterial, viral, or fungal infection within 2 weeks before the first dose of the investigational drug, excluding common cold and onychomycosis, or any other serious infection. Any anti-infective treatment course for infection must have been completed at least 2 weeks before the first dose. Subjects with a history of HIV infection or positive HIV antibody during screening; positive Treponema pallidum antibody during screening; active tuberculosis, defined as chest imaging or other relevant examinations within 3 months before the first dose of the investigational drug or during screening suggesting active tuberculosis infection; or active hepatitis during screening, defined as hepatitis B surface antigen (HBsAg) positivity, or hepatitis B core antibody (HBcAb) positivity with hepatitis B virus (HBV) DNA ≥30 IU/mL, or hepatitis C virus (HCV) antibody positivity with HCV RNA positivity
- •Active bleeding in the gastrointestinal tract, respiratory tract, central nervous system, or other sites
- •A history of any clinically significant disease that, in the investigator's opinion, would pose a safety risk to the subject if participating in the study, or would affect the evaluation of efficacy or safety if the disease/condition worsens during the study, including but not limited to: a. cardiovascular diseases, such as a history of acute myocardial infarction or unstable angina within the past year, severe arrhythmia such as frequent multifocal premature ventricular contractions, ventricular tachycardia, or ventricular fibrillation, congestive heart failure, arterial or venous thrombosis, or New York Heart Association (NYHA) class III-IV cardiac function; b. a history of psychiatric disorders, severe cerebrovascular disease, or cognitive sequelae
- •Use of agents targeting B cells or plasma cells within 3 months before the first dose of the investigational drug or anticipated use during the clinical trial
- •Treatment with anti-lymphocyte globulin or anti-thymocyte globulin within 6 months before the first dose of the investigational drug
- •Treatment with tacrolimus, sirolimus, cyclophosphamide, anti-CD52 monoclonal antibody, or similar therapies within 4 weeks or 5 half-lives, whichever is shorter, before the first dose of the investigational drug
- •Planned participation in another clinical trial, or prior exposure to another investigational product before the first dose, with an interval of less than 4 weeks or 5 half-lives of the drug, whichever is shorter
- •Receipt of a live attenuated vaccine within 4 weeks before the first dose of the investigational drug or planned receipt during the study, or receipt of a COVID-19 vaccine within 7 days before dosing
- •Prior treatment targeting CD38
- •Women who are pregnant or breastfeeding, or who plan to become pregnant or breastfeed during the study
- •Patients considered to be ineligible for the study by the investigator for reasons other than the above
结局指标
主要结局
Incidence of adverse events
时间窗: Within 12 weeks post treatment
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 6 to assess the adverse event
Overall response rate
时间窗: Within 12 weeks post treatment
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
次要结局
未报告次要终点
研究者
Jun Shi
Director of the Red Blood Cell Diseases Center & Director of the Regenerative Medicine Clinic
Institute of Hematology & Blood Diseases Hospital, China
研究点 (1)
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