NCT05454774招募中早期 1 期
A Clinical Study of AAV Vector Expressing Human Coagulation Factor FVIII Gene Therapy for Hemophilia A
Institute of Hematology & Blood Diseases Hospital, China1 个研究点 分布在 1 个国家目标入组 8 人开始时间: 2022年7月19日最近更新:
适应症
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Incidence of dose limiting toxicity (DLT) events
研究概览
简要总结
This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) .
BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.
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
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent.
- •Males ≥ 18 years of age.
- •Have hemophilia A with ≤2 IU/dL (≤2 %) endogenous FVIII activity levels.
- •Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
- •Have had bleeding events and/or infusions with FVIII protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records.
- •Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration.
- •Have no FVIII inhibitor. (eg <0.6BU/ml Bethesda Units; or the patient's FVIII inhibitor titer was detected <0.6BU/ml in 2 consecutive times within 1-4 weeks using Bethesda method or Nijmegen method), or no prior medical history of FVIII inhibitor after 150 EDs of FVIII products; no clinical signs or symptoms of decreased response to FVIII products infusion.
- •Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after BBM002 infusion.
- •Compliance is good, patients and their families have the will of 'gene therapy' clinical trials.
排除标准
- •Being positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus-DNA (HBV-DNA). Being positive for hepatitis C virus antibody (HCV-Ab) or hepatitis C virus RNA (HCV-RNA).
- •Currently on antiviral therapy for hepatitis B or C.
- •Patients with coagulation disorders in addition to hemophilia A.
- •Use of any other systematic immunosuppressant other than glucocorticoids within 30 days prior to enrollment.
- •Patients with vaccination history within 30 days prior to screening.
- •Have potential liver diseases, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy or liver fibrosis (fibrosis stage ≥ 3); nodules or cysts were found by B ultrasound, or elevated alpha-fetoprotein was detected by laboratory tests. Subjects who are not eligible for the study if the abnormalities are clinically significant by researchers.
- •Patients with known planned major surgery schedule during the 52-week study period aren't eligible.
- •Have participated in a previous gene therapy research trial before screening, or in a clinical study with an investigational drug within 5 half-life of the investigational product, whichever is longer.
- •Have alcohol or drug dependence, or cannot stop drinking throughout the study. 10.Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation in the study.
结局指标
主要结局
Incidence of dose limiting toxicity (DLT) events
时间窗: 10 weeks
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) within 10 weeks after administration
The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
时间窗: 52weeks
To assess the safety of BBM 002 Injection by TEAEs and SAEs
次要结局
未报告次要终点
研究者
研究点 (1)
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