EUCTR2019-000667-24-GB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, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
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
入选标准
- •Subjects must meet all of the following criteria to be included in the study:
- •1) Subjects with documented diagnosis of classical Fabry disease as defined by <5% a-Gal A activity in either plasma or leukocytes and one or more of the following symptomatic characteristics of classical Fabry disease: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma.
- •For subjects who do not have a documented diagnostic a-Gal A activity level, a blood sample should be taken to measure a-Gal A activity levels (in plasma and/or leukocytes).
- •For those subjects who are on ERT, this blood draw must be taken at least 13 days after their last ERT infusion (trough).
- •i. If the subject’s level of a-Gal A activity is > 5% and the subject is on ERT, this level of enzyme activity may be due to residual a-Gal A activity from the last ERT infusion. In this case, the diagnosis of classical Fabry disease may be confirmed if the following three criteria are fulfilled:
- •a. two or more of the following documented symptomatic characteristics of classical Fabry: cornea verticillata, acroparesthesia, anhidrosis, angiokeratoma. If there is documented clustered periumbilicial angiokeratoma, this symptom alone is sufficient as it is a pathognomonic sign of classical Fabry disease;
- •b. a mutation that is indicative of classical Fabry (i.e. listed in a database, such as http://dbfgp.org); and
- •c. the a-Gal A activity at trough is below the lower limit of the normal range of the assay.
- •2. Subjects who are on ERT (14 days [± 3 days] regimen); or are ERT-naïve; or are ERT-pseudo-naïve (defined as not having received ERT treatment in the 6 months prior to consent).
- •3. 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 enrollment).
- •4. Male subjects = 18 years of age
- •5. Sexually mature subjects must agree to use a condom and refrain from sperm donation from the time of ST-920 administration until a minimum of 3 consecutive semen samples are negative for rAAV2/6 after administration of ST-920 and a minimum of 90 days after ST-920 administration
- •6. Signed, written informed consent of the subject
- •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 30
- •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 antibody response 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 = 60 ml/min/1.73m^2
- •6. New York Heart Association Class III or higher
- •7. Active infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) or an infection with tuberculosis (TB)
- •8. History of liver disease such as secondary steatosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, cholangitis or biliary disease within 6 months of informed consent; except for Gilbert’s syndrome
- •9. Abnormal circulating AFP
- •10. For subjects receiving ERT, recent or continued hypersensitivity response to ERT treatment within 6 months prior to consent, as manifested by significant infusion reaction to ERT in the opinion of the Site Investigator and Medical Monitor
- •11. One or more of the following: i. Albumin = 3.5 g/dL ii. Total bilirubin > upper limit of normal (ULN) and direct bilirubin = 0.5 mg/dL iii. Alkaline phosphatase (ALP) > 2.0 x ULN iv. Alanine aminotransferase (ALT) > 1.5 x ULN
- •12. Current or history of systemic (IV or oral) immunomodulatory agent or steroid use in the past 6 months (topical treatment is allowed, e.g. asthma or eczema). Occasional use of systemic steroid may be allowed after discussion with the Medical Monitor.
- •13. Contraindication to use of corticosteroids for immunosuppression 14. History of malignancy except for non-melanoma skin cancer
- •15. History of alcohol or substance abuse
- •16. Participation in prior investigational interventional drug or medical device study within the last 3 months prior to consent (with the exception of implantable loop recorders as in the RaILRoAD trial)
- •17. Prior treatment with a gene therapy product
- •18. Known hypersensitivity to components of ST-920 formulation
- •19. Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study
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