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临床试验/EUCTR2016-001446-25-IT
EUCTR2016-001446-25-IT进行中（未招募）1 期

AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH)

REGENXBIO Inc.0 个研究点目标入组 12 人开始时间: 2021年5月6日最近更新:
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适应症

试验速览

阶段
1 期
状态
进行中（未招募）
入组人数
12
研究概览入排标准研究者记录与标识符相似试验

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. Male or female = 18 years of age.
  • •2. Untreated and/or treated LDL-C levels and clinical presentation consistent with the diagnosis of homozygous FH
  • •3. Molecularly defined LDLR mutations at both LDLR alleles.
  • •4. Concurrent allowed lipid lowering medication must be stable for = 4 weeks before the baseline visit and must remain stable until 18 weeks after vector administration. (or 4 weeks post steroid termination). These include but are not limited to: statins, ezetimibe, bile acid sequestrants, PCSK9 inhibitors, and LDL and/or plasma apheresis. Subjects on other lipid- lowering medications are eligible for the study but must wash out of these medications for the pre-specified time period.
  • •5. Females of childbearing potential must have a negative pregnancy test at screening and baseline visits and be willing to have additional pregnancy tests during the study.
  • •6. Sexually active subjects (both female and male) must be willing to use a medically accepted method of contraception from screening visit until 6 months after vector administration
  • •7. A baseline serum AAV8 NAb titer = 1:10.
  • •8. Subjects must be able to comprehend and be willing to provide a signed institutional review board/ethics committee (IRB/EC) approved Informed Consent Form. (ICF).
  • •9. Subjects must be willing to comply with all study-related procedures and be available for the duration of the study.
  • •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 6 weeks of baseline
  • •fibrates: within 4 weeks of baseline
  • •lomitapide: within 8 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. Uncontrolled diabetes defined as HbA1c > 8.5% or an average fasting glucose = 160 mg/dl..
  • •6. Known hypersensitivity to prednisone.
  • •7. History of cirrhosis or chronic liver disease based on documented histological evaluation or non- invasive imaging or testing.
  • •8. 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
  • •9. Abnormal liver function tests (LFTs) at screening (AST or ALT > 2 x upper limit of normal (ULN) and/or Total Bilirubin of >1.5x ULN unless patient has unconjugated hyperbilirubinemia due to Gilbert’s syndrome).
  • •10. Hepatitis B as defined by positive for HepB SAg, or Hep B Core Ab, and/or viral DNA
  • •11. Chronic active Hepatitis C as defined by positive for HCV Ab and viral RNA.
  • •12. History of chronic alcohol abuse within 52 weeks of the screening visit.
  • •13. Certain prohibited medications known to be potentially hepatotoxic, especially those that can induce microvesicular or macrovesicular steatosis. These include but are not limited to: Accutane (isotretinoin), amiodarone, HAART medications, heavy acetaminophen use (2 g/day more than 3 times a week), isoniazid, methotrexate, tetracyclines, tamoxifen, valproate.
  • •14. Active tuberculosis, systemic fungal disease, or other chronic infection.
  • •15. History of immunodeficiency diseases, including a positive HIV test result.
  • •16. Chronic renal insufficiency defined as estimated GFR < 30 mL/min/1.73m2.
  • •17. 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.
  • •18. Previous organ transplantation.
  • •19. 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. Subjects are not prohibited from receiving investigational drugs after 52 weeks.
  • •20. 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.
  • •21. 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.
  • •22. Any other medical condition or finding that would make it not in the subject’s best interest to participate in the stu

研究者

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