A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 34
- 主要终点
- Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation
研究概览
简要总结
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
详细描述
The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met.
The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participated in Study mRNA-3927-P
- •Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.
排除标准
- •Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
- •Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
- •History of liver and/or kidney transplant.
研究组 & 干预措施
mRNA-3927
Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation.
干预措施: mRNA-3927 (Biological)
结局指标
主要结局
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation
时间窗: Baseline through End of Study Visit (up to 8 years)
次要结局
- Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)(Baseline through End of Study Visit (up to 8 years))
- Annualized Frequency of Investigator-reported MDE-related Hospitalizations(Baseline through End of Study Visit (up to 8 years))
- Annualized Frequency of Investigator-reported PA-related Hospitalizations(Baseline through End of Study Visit (up to 8 years))
- Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters(Baseline through End of Study Visit (up to 8 years))