NL-OMON48684已完成2 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) - 401GSDIA01
ltragenyx Pharmaceutical, Inc.0 个研究点目标入组 1 人开始时间: 待定最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
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 (Section 9.2.1).
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
- •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
- •be a risk to the subject or would impede the study.
研究者
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