IMPACT Study: Intervention Monitoring for Pancreatitis in an Adolescent Undergoing Clinical Treatment
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Time Between Acute Pancreatitis Episodes While on Ivacaftor
研究概览
简要总结
The goal of this observational study is to learn about the long-term effects of Ivacaftor in an adolescent without a cystic fibrosis-associated CFTR mutation who is taking Ivacaftor to treat chronic pancreatitis. The main question it aims to answer is:
Does Ivacaftor reduce the frequency or severity of acute pancreatitis episodes in this adolescent compared to their historical medical data?
The participant, who is already taking Ivacaftor as part of their medical care, will be monitored over the course of 12 months. Flare frequency, hospitalizations, and wellness data will be tracked through caregiver reports, lab data, and physiological data collected from a wearable health monitoring device (Apple Watch).
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The subject has been diagnosed with recurrent acute pancreatitis or chronic pancreatitis and a clinical decision has been made to treat the subject with one or more FDA-approved therapies, which may be being used off-label) to treat or prevent pancreatitis symptoms.
排除标准
- •Participant has a known cystic fibrosis-associated CFTR mutation that would qualify them for standard Ivacaftor use.
- •Participant is unable to tolerate Ivacaftor due to prior adverse reactions.
- •Participant has severe liver dysfunction or baseline liver enzyme elevations that contraindicate Ivacaftor use, as determined by the treating physician.
- •Participant or parent/guardian is unwilling to comply with study procedures, including regular follow-up and data sharing.
- •Any other medical or psychological condition, in the judgment of the treating physician or counselor, that would make participation unsafe or not in the best interest of the participant.
结局指标
主要结局
Time Between Acute Pancreatitis Episodes While on Ivacaftor
时间窗: 12 months
The number of days between clinically confirmed acute pancreatitis attacks during the 12-month Ivacaftor treatment period, compared to historical pre-treatment intervals.
次要结局
未报告次要终点