Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 5
- 主要终点
- Concentration (ng/mL)
研究概览
简要总结
This clinical trial is examining the action and effects of several new drugs in the treatment of cystic fibrosis in children. In addition, several genetic factors are examined. The hope is that the ability to determine prior to treatment those individuals who will or will not respond to existing therapies will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen. Understanding the way these drugs work in the body and the best way to study them is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).
详细描述
Understanding variation in genetic response to pharmacological treatments and personalized CFTR modulator response is crucial to the optimization of the use of these novel compounds; expansion to all patients who might benefit from them; and development of predictive biomarkers. In addition, the ability to determine prior to treatment those individuals who will or will not respond to existing therapies will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen. Understanding the way these drugs work in the body and the best way to study them and the downstream effects is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).
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
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •documentation of CF diagnosis per CFF diagnostic criteria and known CFTR genotype
- •age 2 years and older
- •ability to provide written informed consent and/or assent (by subject and/or legal guardian)
- •on a stable dose of triple combination CFTR modulator therapy for at least two weeks prior to Visit 1
- •clinically stable lung disease, defined as no documented acute decrease in FEV1 > 10%, OR use of additional antibiotics (intravenous [IV] or oral [PO]) within 4 weeks prior to screening
排除标准
- •recent significant unintentional weight loss, as determined by the investigator, in the 4 weeks prior to screening
- •pregnant or breastfeeding female
- •history of alcohol or substance abuse in the 6 months prior to screening
- •participation in a study involving an investigational intervention within 28 days (or 5 half-lives, whichever is longer) prior to screening
- •in the opinion of the Investigator, medical or psychiatric illness, or other conditions that would interfere with participation
研究组 & 干预措施
Single Arm
Participants may be enrolled in an observational one-visit study for association of concentration with side effects. Participants may proceed to a single arm study if they have side effects to assess the feasibility of adjusting dose to maintain concentrations within an estimated effective range. Once within the range, dosing is no longer adjusted. Side effects will be evaluated as described in the protocol.
干预措施: therapeutic drug monitoring (Other)
Single Arm
Participants may be enrolled in an observational one-visit study for association of concentration with side effects. Participants may proceed to a single arm study if they have side effects to assess the feasibility of adjusting dose to maintain concentrations within an estimated effective range. Once within the range, dosing is no longer adjusted. Side effects will be evaluated as described in the protocol.
干预措施: Elexacaftor / Ivacaftor / Tezacaftor (Drug)
结局指标
主要结局
Concentration (ng/mL)
时间窗: One time assessment for observational part of the study, up to 6 times (6 months or more) for the therapeutic drug monitoring pilot and feasibility study.
Drug concentration of CFTR modulators
次要结局
- Participant Mental and Neuropsychological Health(From enrollment to the end of treatment at 6-12 months.)
- Investigators will evaluate the feasibility of reducing dose to manage Neuropsychological Side Effects (NPSE).(From enrollment to study conclusion at 6-12 months (after all visits are completed).)
- Response to dosing adjustments(From enrollment to study conclusion at 6-12 months, after all visits are completed.)
研究者
Jennifer Guimbellot
Principal Investigator - MD
Arkansas Children's Hospital Research Institute