A Phase 2a Randomized, Double-blind, Placebo-controlled Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Physician-prescribed Trikafta® in People With Cystic Fibrosis Who Are Homozygous for the F508del Mutation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 22
- 主要终点
- To evaluate the incidence of treatment emergent adverse events [Safety and Tolerability] when SION-719 is administered to people with cystic fibrosis (CF) who are taking a standard stable dose of physician-prescribed Trikafta
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SION-719 when given to people with CF who are already taking Trikafta.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CF with F508del homozygous genotype based on documented CFTR genotype laboratory report.
- •Adherent to physician-prescribed Trikafta, as assessed by the Investigator, for at least 3 months prior to the Screening visit, taken at the recommended dose without modifications and on the regimen described in the current prescribing information.
- •Stable CF disease without pulmonary exacerbation within 28 days before Baseline or acute non-CF-related illness within 14 days before Baseline.
排除标准
- •Participant has clinically significant current or recurrent illness, other than CF
- •Participant has a history of malignancy, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of recurrence for at least 1 year.
研究组 & 干预措施
Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719
Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.
干预措施: Placebo-to-match SION-719 (Drug)
Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719
Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.
干预措施: Placebo-to-match SION-719 (Drug)
Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719
Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.
干预措施: SION-719 (Drug)
Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719
Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.
干预措施: SION-719 (Drug)
结局指标
主要结局
To evaluate the incidence of treatment emergent adverse events [Safety and Tolerability] when SION-719 is administered to people with cystic fibrosis (CF) who are taking a standard stable dose of physician-prescribed Trikafta
时间窗: Day 1 through day 57
Adverse events will be assessed by Common Terminology Criteria for Adverse Events (CTCAE), v. 5.0
次要结局
- Change from baseline in sweat chloride levels(Baseline to Day 15 in each treatment period)
- Concentration of SION-719 in plasma and change of concentration of SION-719 in plasma.(Day 1 through day 57)