NCT04038710已完成不适用
An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- pulmonary function
研究概览
简要总结
This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access. Pulmonary health, intestinal health, and the overall health of individuals will be tracked for a year to see how effective triple combination therapy is in these people with severe cystic fibrosis disease.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Cystic Fibrosis
- •Ability to reproducibly perform spirometry (according to ATS criteria)
- •Physician decision to treat with TCT through the EAP program
- •Ability to understand and sign a written informed consent or assent and comply with the requirements of the study
排除标准
- •History of hypersensitivity to VX 445, VX 659, tezacaftor and/or ivacaftor
- •Any acute lower respiratory symptoms treated with oral, inhaled or IV antibiotics or systemic corticosteroids within the 2 weeks prior to Visit
- •Major or traumatic surgery within 12 weeks prior to Visit
- •Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit
- •Use of an investigational agent within 28 days prior to Visit
- •History of lung or liver transplantation or listing for organ transplantation.
研究组 & 干预措施
Patients with severe disease
Patients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program.
干预措施: Triple combination therapy (Drug)
结局指标
主要结局
pulmonary function
时间窗: For a year post initiation of therapy.
FEV1 values.
次要结局
- CFQ-R score(For a year following the initiation of triple combination therapy.)
研究者
研究点 (1)
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