NCT03728322Unknown早期 1 期
A Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations
Allife Medical Science and Technology Co., Ltd.0 个研究点目标入组 12 人开始时间: 2019年1月1日最近更新:
适应症
试验速览
- 阶段
- 早期 1 期
- 发起方
- 入组人数
- 12
- 主要终点
- Occurrence of treatment related adverse events as assessed by CTCAE v4.0
研究概览
简要总结
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
详细描述
The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
研究设计
- 研究类型
- Interventional
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects ≥ 2 and ≤ 60 years of age
- •Subjects was confirmed the transfusion-dependent β-thalassemia
- •Adequate organ function, as defined by:
- •Serum creatinine ≤ 1.5 mg/dl ； Serum ALT/AST）≤2.5×ULN；ALB≥25g/L； Serum total bilirubin < 1.5x ULN Left ventricular ejection fraction≥50%
- •Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases
- •Subjects survival was expected≥6 months
- •Adult patients were willing to use reliable contraceptives (such as condoms) and not to donate sperm throughout the study period and within three months of discharge
- •Subjects and the guardians able to undergo post-physical therapy/rehabilitation
排除标准
- •Subjects allergic to macromolecular biological agents such as antibodies or cytokines
- •Subjects receipt of any investigational clinical trials within 3 months.
- •Subjects previous treatment with any hematopoietic stem cell transplantation or other organ transplantation
- •Uncontrolled bleeding symptoms
- •Severe cardiovascular disease is known, including any of the following:
- •Myocardial infarction or thrombosis has occurred in the past six months Subjects with unstable angina pectoris Subjects with Class III/IV cardiovascular disability according to the New York Heart Association Classification
- •Subjects have one kinds of tumors within 5 years
- •Active hepatitis B (HBV DNA>1000copy/mL), hepatitis C or HIV infection.
- •Subjects have an infectious diseases that cannot be controlled within 4 weeks
- •subjects have severe central nervous system disease or epilepsy
- •Subjects are Suffering from mental illness; Patients with alcohol dependence, drug abuse, drug addiction, and medical, psychological or social conditions that may interfere with research or have an impact on the evaluation of research results
- •Women in pregnancy (positive urine/blood pregnancy test) or lactation
- •Subjects who have other conditions that were not appropriate for the group determined by the researchers.
结局指标
主要结局
Occurrence of treatment related adverse events as assessed by CTCAE v4.0
时间窗: 1 year
Defined as \>= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment
次要结局
未报告次要终点
研究者
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