EUCTR2016-001446-25-NL进行中（未招募）1 期
AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH)
niversity of Pennsylvania0 个研究点目标入组 12 人开始时间: 2016年9月12日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
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
- •1. Age: 18 years or older;
- •2. Molecularly defined LDLR mutations in both LDLR alleles and clinical
- •presentation consistent with HoFH;
- •3. No cardiovascular event or cardiovascular intervention within 12 weeks of
- •enrollment;
- •4. A baseline serum AAV8 neutralizing antibody titer = 1:10.
- •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 12
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Unwilling to wash out of the following lipid lowering therapies for the pre-specified time period:
- •niacin > 250 mg/day: within 4 weeks of baseline
- •fibrates: within 4 weeks of baseline
- •Lomitapide: within 6 weeks of baseline
- •Mipomersen: within 24 weeks of baseline
- •2. Heart failure defined by the NYHA classification as functional Class III with history of hospitalization(s) within 12 weeks of the baseline visit or functional Class IV.
- •3. History within 12 weeks of the baseline visit of a myocardial infarction (MI), unstable angina leading to hospitalization, coronary artery bypass graft surgery (CABG), percutaneous coronary intervention (PCI), uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, carotid revascularization, endovascular procedure or surgical intervention.
- •4. Uncontrolled hypertension defined as: systolic blood pressure > 180 mmHg, diastolic blood pressure > 95 mmHg.
- •5. History of cirrhosis or chronic liver disease based on documented histological evaluation or non- invasive imaging or testing.
- •6. Documented diagnosis of any of the following liver diseases:
- •Nonalcoholic steatohepatitis (biopsy-proven)
- •Alcoholic liver disease
- •Autoimmune hepatitis
- •Liver cancer
- •Primary biliary cirrhosis
- •Primary sclerosing cholangitis
- •Wilson’s disease
- •Hemochromatosis
- •a1 anti-trypsin deficiency
- •7. Abnormal LFTs at screening (AST or ALT >2x upper limit of normal (ULN) and/or Total Bilirubin of
- •>1.5x ULN unless patient has unconjugated hyperbilirubinemia due to Gilbert’s syndrome).
- •8. Hepatitis B as defined by positive for HepB SAg, or Hep B Core Ab, and/or viral DNA
- •9. Chronic active Hepatitis C as defined by positive for HCV Ab and viral RNA.
- •10. History of alcohol abuse within 52 weeks.
- •11. Certain prohibited medications known to be potentially hepatotoxic, especially those that can induce microvesicular or macrovesicular steatosis. These include but are not limited to: acutane, amiodarone, HAART medications, heavy acetaminophen use (2g/day more than 3 times a week), isoniazid, methotrexate, tetracyclines, tamoxifen, valproate.
- •12. Current use of systemic corticosteroids or active tuberculosis, systemic fungal disease, or other chronic infection.
- •13. History of immunodeficiency diseases, including a positive HIV test result.
- •14. Chronic renal insufficiency defined as estimated GFR < 30 mL/min.
- •15. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer.
- •16. Previous organ transplantation.
- •17. Administration of an investigational drug within 12 weeks or 5 half-lives of the drug (whichever is longer) prior to the screening visit and until 52 weeks after receiving AAV8.TBG.hLDLR.
- •18. Any major surgical procedure occurring less than 3 months prior to the screening visit, or any planned future surgical procedure within 3 months of baseline.
- •19. Serious or unstable medical or psychological conditions that, in the opinion of the investigator, would compromise the subject’s safety or successful participation in the study.
- •20. A baseline serum AAV8 NAb titer > 1:10.
- •21. Any other medical condition or finding that would make it not in the subject’s best interest to participate in the study
- •22. Study staff member or any direct family member.
研究者
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