GSDIa Disease Monitoring Program
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 140
- 试验地点
- 38
- 主要终点
- Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
研究概览
简要总结
The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.
详细描述
The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the 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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient who had:
- •DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or
- •Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)
- •Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.
排除标准
- •Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
研究组 & 干预措施
Group 2: DTX401 in Post-Marketing Setting
Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting
干预措施: No Intervention (Other)
Group 1: DTX401 in Prior Clinical Study
Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
干预措施: No Intervention (Other)
结局指标
主要结局
Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
时间窗: 10 Years
Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner
时间窗: 10 Years
Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies
时间窗: 10 Years
Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator
时间窗: 10 Years
Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)
时间窗: 1 Year
Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)
时间窗: 1 Year
次要结局
- Percent Change Over Time from Baseline in Total Cornstarch Intake(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Frequency of Cornstarch Intake(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch)(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Daytime and Nighttime Percentage of Glucose Values During the 2 Weeks Leading Up to the Assessment Visits(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Total Number of Hypoglycemic Events on Continuous Glucose Monitoring (CGM) During the 2 Weeks Leading Up to the Assessment Visits(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Total Duration of Hypoglycemic Events on CGM During the 2 Weeks Leading Up to the Assessment Visits(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Real-World Overnight Fasting Tolerance(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height(10 Years)
- Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight(10 Years)
- Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI)(10 Years)
- Number of Participants with Clinically Significant Laboratory Values(10 Years)
- Frequency of Major Clinical Events (MCEs)(10 Years)
- Frequency of MCEs from Review of Medical Records(10 Years)
- Change from Baseline in Patient Experience Clinical Interview(Baseline, Up to 10 Years)
- Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score(Baseline, Up to 1 Year)
- Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score(Baseline, Up to 1 Year)
- Change from Baseline Over Time in Annual Number of GSDIa Related Hospitalizations, Emergency Room Visits, Outpatient Visits, and Hospital Days(Baseline, Up to 10 Years)
- Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months(Baseline, Up to 10 Years)
- Change from Baseline in Work/School Productivity Rating(Baseline, Up to 10 Years)
- Change from Baseline in Employment(Baseline, Up to 10 Years)