EUCTR2019-000667-24-IT进行中（未招募）1 期
A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease - STAAR
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 48
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Subjects must meet all of the following criteria to be included in the study:
- •1. >=18 years of age
- •2. Signed, written informed consent
- •3. Diagnosis of Fabry disease
- •4. One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma
- •5. Subjects who are on ERT or are ERT-naïve or are ERT-pseudo-naïve (defined as not having received ERT treatment in the 6 months prior to consent). For subjects receiving ERT, ERT must have been administered at a stable dose for at least 6 months (defined as not having missed more than 3 doses of ERT during the 6 months prior to consent) and regimen (14 days ± 3 days for at least 3 months prior to screening); except for Cohort 3d (see inclusion criterion 8),who must have been on ERT for at least the past year prior to screening.
- •6. Male subjects must agree to use an effective form of contraception (e.g., sexual abstinence, documented vasectomy, condom) and refrain from sperm donation from the time of ST-920 administration until a minimum of 3 consecutive semen samples are negative for AAV2/6 after administration of ST-920 and a minimum of 90 days after ST-920 administration.
- •7. Female subjects from menarche until becoming post-menopausal or permanently sterile must have a negative serum pregnancy test at screening and must agree to use a highly effective contraception method from screening through the end of the study. Highly effective contraception in females includes:
- •combined (estrogen and progestogen containing) hormonal contraception (oral, intravaginal or transdermal)
- •progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable)
- •intrauterine device (IUD)
- •intrauterine hormone-releasing system (IUS)
- •bilateral tubal occlusion or vasectomized partner
- •sexual abstinence
- •FOR OTHERS INCLUSION CRITERIA PLEASE REFERS TO STUDY PROTOCOL
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 48
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects who meet any of the following criteria will be excluded from participating in the study:
- •1. Known to be unresponsive to ERT in the opinion of the Site Investigator and Medical Monitor (e.g., no documented substrate level decrease on ERT)
- •2. Current treatment with migalastat (Galafold™) or prior treatment within 3 months of informed consent
- •3. Positive neutralizing antibodies to AAV6
- •4. Intercurrent illness expected to impair evaluation of safety or efficacy during the observation period of the study in the opinion of the Site Investigator or Medical Monitor
- •5. eGFR <= 40 ml/min/1.73m2
- •6. Active infection with hepatitis A virus (HAV RNA positive), active or occult hepatitis B virus infection (positive HBV-DNA or anti-HBc positive), active infection with hepatitis C virus (HCV RNA positive), infection with the human immunodeficiency virus (HIV) as measured by quantitative polymerase chain reaction (qPCR) or active or latent infection with tuberculosis (TB) measured by quantiferon test
- •7. Breastfeeding at screening or planning to become pregnant (self or partner) or breastfeed during required period of contraception.
- •8. History of liver disease such as clinically significant steatosis, fibrosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, biliary disease within
- •6 months of informed consent; except for Gilbert's syndrome
- •9. Elevated circulating serum AFP
- •10. For subjects receiving ERT, recent or recurrent hypersensitivity reaction manifested by significant infusion reaction to ERT treatment within 6 months prior to consent, in the opinion of the Site Investigator and Medical Monitor
- •FOR OTHERS EXCLUSION CRITERIA PLEASE REFERS TO STUDY PROTOCOL
研究者
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