EUCTR2016-003023-30-NL进行中（未招募）1 期
A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV)Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
ltragenyx Pharmaceutical, Inc.0 个研究点目标入组 12 人开始时间: 2018年8月1日最近更新:
适应症
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. Willing and able to provide written informed consent.
- •2. Males and females =18 years of age.
- •3. Documented GSDIa with confirmation by molecular testing.
- •4. Documented history of =1 hypoglycemic event with glucose <60 mg/dL (<3.33 mmol/L).
- •5. Subject’s GSDIa disease is stable as evidenced by no hospitalization for severe hypoglycemia during the 4-week period preceding the screening visit.
- •6. Hematology and coagulation panel results are within the normal range or, if outside the normal range, are deemed not clinically significant in the opinion of the investigator.
- •7. No known allergic reaction to any component of DTX401.
- •8. Willing and able to comply with study procedures and requirements, including periodic inpatient hospitalization, frequent blood collections, and 24-hour urine collection.
- •9. Males and females of childbearing potential must be willing to use effective contraception at the time of administration of DTX401 and for 52 weeks following administration of DTX401 to prevent the potential transmission of the AAV vector.
- •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 8
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •1. Screening or Baseline (Day 0) glucose level <60 mg/dL (<3.33 mmol/L); subjects may be
- •rescreened after glucose is controlled and stable, at the discretion of the investigator.
- •2. Liver transplant, including hepatocyte cell therapy/transplant.
- •3. Presence of liver adenoma >5 cm in size.
- •4. Presence of liver adenoma >3 cm and =5 cm in size that has a documented annual growth rate of =0.5 cm per year.
- •5. Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase > the upper limit of normal (ULN), total bilirubin >1.5 × ULN, or alkaline phosphatase >2.5 × ULN. Liver function tests may be repeated during the screening period at the investigator’s discretion.
- •6. Serum creatinine >2.0 mg/dL.
- •7. Triglycerides =1000 mg/dL at the time of the screening visit.
- •8. Presence of active, or history of treatment for, hepatitis B virus or hepatitis C virus infection.
- •9. History of human immunodeficiency virus infection AND any of the following: CD4+ cell count <350 cells/mm3, change in antiretroviral therapy regimen within 6 months prior to
- •Day 0, or viral load >200 copies/mL, on 2 separate occasions, as measured by polymerase chain reaction.
- •10. History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer treated with watchful waiting or surgically removed nonmelanoma skin cancer.
- •11. Active infection (viral or bacterial).
- •12. Anti-AAV8 neutralizing antibody titer =1:5.
- •13. Participation (current or previous) in another gene transfer study.
- •14. Participation in another investigational product study within 3 months of Screening.
- •15. Has a positive serum pregnancy test at Screening (females of childbearing potential only), a positive urine pregnancy test at Baseline (Day 0; females of childbearing potential only), or is nursing.
- •16. Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.
研究者
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