A Long-Term Follow-up Study to Evaluate the Safety and Efficacy 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)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 6
- 主要终点
- Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs
研究概览
简要总结
The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.
详细描述
Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02. Participants will be followed in study 401GSDIA02 for at least 4 years, and up to 6 years after administration of DTX401.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Received DTX401 in study 401GSDIA
- •Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed.
- •Willing and able to comply with all scheduled study visits, procedures, and requirements.
排除标准
- •Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
- •Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.
研究组 & 干预措施
DTX401 Cohort 1
Participants received a single intravenous (IV) dose of 2.0 × 10^12 GC/kg DTX401(pariglasgene brecaparvovec) with a reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.
干预措施: No intervention (Other)
DTX401 Cohort 2
Participants received a single IV dose of 6.0 × 10^12 GC/kg DTX401(pariglasgene brecaparvovec) with a reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.
干预措施: No intervention (Other)
DTX401 Cohort 3
Participants received a single IV dose of 6.0 × 10^12 GC/kg DTX401(pariglasgene brecaparvovec) with an optimized reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.
干预措施: No intervention (Other)
DTX401 Cohort 4
Participants received a single IV dose of 6.0 × 10^12 GC/kg DTX401(pariglasgene brecaparvovec) with a prophylactic steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.
干预措施: No intervention (Other)
结局指标
主要结局
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs
时间窗: From Baseline (Week 52 of 401GSDIA01/Visit 1 of 401GSDIA02) Up to Week 329
An adverse event (AE) is defined as any untoward medical occurrence, regardless of its causal relationship to study product. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. A serious TEAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/Incapacity; congenital anomaly/birth defect not present at screening; other important medical events. Severity of events were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). As DTX401 was administered as part of Study 401GSDIA01, all AEs in Study 401GSDIA02 were considered TEAEs.
次要结局
- Change From Baseline in Time to First Hypoglycemic Event During a Controlled Fasting Challenge Over Time(Baseline (Week 0 of 401GSDIA01), Weeks 52 (Visit 1 of 401GSDIA02), 78, 104, 130, 156, 182, 208, 234, 260, Last Visit (Up to Week 329))