Personalized Theratyping Trial
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- spirometry
研究概览
简要总结
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.
详细描述
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs. Symdeko (Tezacaftor/Ivacaftor), Orkambi (Ivacaftor and Lumacaftor), correctors of CFTR misfolding and Kalydeco (Ivacaftor), a potentiator of abnormal CFTR gating, will be explored as a treatment for patients with other CF mutations than those currently approved. Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, or Orkambi, depending on the in vitro response pattern. Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy. Patients with a mutation equivalent to wild type will be given Ivacaftor. If the patient is 6-12 years old, we will only study Orkambi or ivacaftor as symdeko is not yet FDA approved in this patient population.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CF
- •Age ≥6 y.o.
- •CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators
- •Informed Consent/Assent
- •Stable CF pulmonary regimen
排除标准
- •Exacerbation requiring antibiotic or steroids for >28 days before trial entry
- •Ongoing participation in a CFTR modulator study
- •Active smoking in the past 6 months
- •History of solid organ transplant
- •Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)
- •Any condition that precludes the patient from participation in the opinion of the investigator
- •Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators
研究组 & 干预措施
Symdeko
Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern
干预措施: Symdeko (Drug)
Ivacaftor
Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy.. Patients with a mutation equivalent to wild type will be given Ivacaftor.
干预措施: Symdeko (Drug)
Orkambi
Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern
干预措施: Symdeko (Drug)
结局指标
主要结局
spirometry
时间窗: 32 weeks
change in lung function as measured via spirometry
次要结局
未报告次要终点
研究者
George Solomon
Principal Investigator
University of Alabama at Birmingham